Sickle cell disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 17 Jun 2026 04:02:20 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Sickle cell disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Methoxyflurane versus oral oxycodone hydrochloride and morphine sulfate for pain relief in sickle cell disease vaso‑occlusive crises https://clinicaltrials.eu/trial/methoxyflurane-versus-oral-oxycodone-hydrochloride-and-morphine-sulfate-for-pain-relief-in-sickle-cell-disease-vaso-occlusive-crises/ Fri, 05 Jun 2026 10:55:57 +0000 https://clinicaltrials.eu/trial/methoxyflurane-versus-oral-oxycodone-hydrochloride-and-morphine-sulfate-for-pain-relief-in-sickle-cell-disease-vaso-occlusive-crises/ The study involves people with sickle cell disease who experience a painful episode called a vaso-occlusive crisis. The investigation compares a fast‑acting inhaled pain medicine, methoxyflurane, with standard oral opioid pills such as morphine sulfate or oxycodone hydrochloride. The purpose is to see which approach provides quicker and more effective relief of pain when patients first arrive at the emergency department.

When a participant comes to the emergency department with a crisis, they receive either the inhaled medication or an oral opioid pill. Their pain level is recorded every ten minutes for the first hour using a line‑marking tool called the Visual Analog Scale. After one hour, the patient rates their overall satisfaction on a numbered rating system known as the Likert scale, and the attending nurse also records a satisfaction rating. Throughout the stay, the amount of any additional pain medicines given, the length of the hospital visit, and basic safety checks are noted, after which the patient is discharged.

]]> A study to test how etavopivat works and its safety in children with sickle cell disease https://clinicaltrials.eu/trial/a-study-to-test-how-etavopivat-works-and-its-safety-in-children-with-sickle-cell-disease/ Thu, 30 Apr 2026 13:08:48 +0000 https://clinicaltrials.eu/trial/a-study-to-test-how-etavopivat-works-and-its-safety-in-children-with-sickle-cell-disease/ This study is looking at sickle cell disease, a condition that affects the shape of red blood cells and can cause various health problems. The study will test a medicine called etavopivat in children and young people from 6 months to under 18 years of age. Etavopivat is given by mouth in the form of granules. The medicine is being tested to see how it works in the body and whether it is safe for children with sickle cell disease.

The purpose of the study is to understand how the body handles etavopivat and to check for any unwanted effects during a 24-week treatment period. During the study, doctors will measure how much medicine is in the blood after a single dose and during regular treatment. They will also monitor for any side effects and record how often treatment needs to be stopped or changed. The study will look at whether the medicine helps increase the level of a substance in the blood called hemoglobin, which carries oxygen throughout the body. Doctors will also track painful episodes called vaso-occlusive crises, which are common in sickle cell disease when blood flow becomes blocked.

Participants will be divided into different groups based on their age, starting with older children and then including younger ones. Some children in the study may already be taking other treatments for sickle cell disease, such as hydroxyurea, crizanlizumab, or L-glutamine, and can continue these medicines if their dose has been stable. The study will also measure changes in tiredness levels and check blood flow in the brain using a test called transcranial Doppler ultrasonography in children over 2 years old. After the first 24 weeks, there will be an extension period where the medicine can continue to be given while doctors keep checking for safety.

]]> Study of lidocaine to reduce opioid use in patients with sickle cell disease experiencing severe pain crisis https://clinicaltrials.eu/trial/study-of-lidocaine-to-reduce-opioid-use-in-patients-with-sickle-cell-disease-experiencing-severe-pain-crisis/ Thu, 30 Apr 2026 13:08:21 +0000 https://clinicaltrials.eu/trial/study-of-lidocaine-to-reduce-opioid-use-in-patients-with-sickle-cell-disease-experiencing-severe-pain-crisis/ This study involves people with Sickle Cell Disease who are experiencing a painful condition called vaso-occlusive crisis, which happens when blood vessels become blocked and cause severe pain in different parts of the body. Some patients may also develop acute chest syndrome, which causes breathing problems and chest pain along with changes visible on chest imaging. The study will compare two treatment approaches for managing pain during these severe episodes. One group will receive lidocaine hydrochloride along with standard pain treatment, while the other group will receive sodium chloride along with standard pain treatment. Both medications will be given as injections. Standard pain treatment typically includes strong pain medications called opioids, such as morphine or oxycodone, which are given through a vein.

The purpose of this study is to find out whether adding lidocaine to the usual pain management can reduce the total amount of opioid pain medication needed during a severe vaso-occlusive crisis. The study will measure the total amount of opioid medication used from the time patients join the study until they leave the intensive care unit, where they receive care for their condition.

Patients who take part will be admitted to the intensive care unit for treatment of their vaso-occlusive crisis or acute chest syndrome. They will receive either lidocaine or sodium chloride in addition to their standard pain treatment for up to three days. During their stay in the intensive care unit, doctors will monitor their pain levels, how much pain medication they need, and how long it takes for their crisis to improve. The study will also track how long patients stay in the intensive care unit and in the hospital overall, whether any complications develop, and their quality of life after 28 days. Researchers will look at whether patients need additional hospital visits or emergency room care within the first month after joining the study.

]]> Comparing morphine alone to morphine and lidocaine for pain relief in sickle cell anemia patients with vaso-occlusive crisis https://clinicaltrials.eu/trial/comparing-morphine-alone-to-morphine-and-lidocaine-for-pain-relief-in-sickle-cell-anemia-patients-with-vaso-occlusive-crisis/ Wed, 29 Apr 2026 15:08:23 +0000 https://clinicaltrials.eu/trial/comparing-morphine-alone-to-morphine-and-lidocaine-for-pain-relief-in-sickle-cell-anemia-patients-with-vaso-occlusive-crisis/ This study involves patients with sickle cell anaemia who experience a painful complication called a vaso-occlusive crisis, which occurs when blood flow is blocked in blood vessels, causing severe pain. The study will compare two different pain treatment approaches. One group of patients will receive morphine hydrochloride alone, which is a strong pain medication, while the other group will receive a combination of morphine hydrochloride and lidocaine hydrochloride, which is a local numbing medication. Both treatments will be given through a vein using a method called patient-controlled analgesia, which allows patients to control their own pain medication doses within safe limits set by doctors.

The purpose of the study is to find out if adding lidocaine hydrochloride to morphine hydrochloride can reduce the total amount of morphine needed to control pain during a vaso-occlusive crisis. The study is designed so that neither the patients nor the doctors will know which treatment each patient is receiving during the trial, which helps ensure fair results. Patients will be randomly assigned to receive either the combination treatment or morphine alone.

During the study, researchers will measure how much morphine each patient uses, track changes in pain levels using a pain rating scale, monitor certain substances in the blood that may indicate how the body is responding to treatment, and watch for any unwanted effects that morphine might cause. The study will help doctors understand whether combining these two medications provides better pain relief with less morphine compared to using morphine by itself.

]]> A study testing gene therapy with Dream01 and imatinib for patients with severe sickle cell disease to improve red blood cell production https://clinicaltrials.eu/trial/a-study-testing-gene-therapy-with-dream01-and-imatinib-for-patients-with-severe-sickle-cell-disease-to-improve-red-blood-cell-production/ Wed, 29 Apr 2026 15:08:02 +0000 https://clinicaltrials.eu/trial/a-study-testing-gene-therapy-with-dream01-and-imatinib-for-patients-with-severe-sickle-cell-disease-to-improve-red-blood-cell-production/ This study involves sickle cell disease, a serious inherited blood disorder that affects red blood cells and can cause severe pain episodes, organ damage, and other complications. The study will test a treatment called DREAM01, which is a type of gene therapy. Gene therapy is a treatment that works by modifying a patient’s own cells to correct the genetic problem causing the disease. In this approach, special blood-forming cells called CD34+ cells are collected from the patient, modified in a laboratory using a lentiviral vector (a tool that delivers corrected genetic material into the cells), and then given back to the patient through an intravenous infusion, which means the cells are returned into the bloodstream through a vein. Some patients may also receive imatinib before the gene therapy if they have severe inflammation detected during the initial evaluation phase.

The purpose of this study is to evaluate whether this gene therapy treatment is safe and effective for patients with sickle cell disease, both with and without prior imatinib treatment. The study will examine whether the treatment can restore normal red blood cell production by correcting the genetic defect that causes the abnormal hemoglobin (the protein in red blood cells that carries oxygen) found in sickle cell disease. The modified cells are designed to produce a corrected form of hemoglobin and also include a component that specifically targets the defective hemoglobin to reduce its production.

During the study, patients will undergo several evaluations to monitor the effectiveness and safety of the treatment. This includes measuring various blood parameters related to anemia and the breakdown of red blood cells, checking the function of different organs that may be affected by the disease such as the brain, heart, kidneys, liver, and lungs, and assessing iron levels in the body. The study will also track the frequency of painful episodes that require hospitalization, the need for blood transfusions, and changes in the patient’s physical abilities and quality of life. Patients will be followed for an extended period after receiving the treatment to carefully monitor outcomes and any potential side effects. The treatment involves a preparation phase where patients receive conditioning therapy to prepare their body for the infusion of the modified cells.

]]> Study on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-the-effects-of-tebapivat-and-ag-946-in-patients-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:06:14 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-tebapivat-and-ag-946-in-patients-with-sickle-cell-disease/ This clinical trial is studying Tebapivat for the treatment of Sickle Cell Disease (SCD). Sickle Cell Disease is a group of inherited blood disorders where red blood cells become crescent or sickle-shaped instead of round, which can lead to blocked blood flow, pain, infections, and anemia (low red blood cell count). The purpose of this study is to compare different doses of Tebapivat versus placebo to determine its effect on anemia in people with Sickle Cell Disease.

The study is designed to find the optimal dose of Tebapivat by measuring its impact on hemoglobin levels (the protein in red blood cells that carries oxygen) and other markers of blood health. Participants will take either Tebapivat or placebo for 12 weeks during the initial double-blind period. During this time, researchers will monitor changes in hemoglobin concentration, markers of red blood cell breakdown (hemolysis), and how participants feel regarding fatigue and pain.

After the initial 12-week period, participants may continue in an open-label extension period where all participants receive Tebapivat. Throughout the study, participants will undergo various assessments including blood tests, questionnaires about pain and quality of life, and tests measuring physical and cognitive function. The study will also evaluate the safety of Tebapivat by monitoring any side effects that occur during treatment.

]]> Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-rilzabrutinib-for-patients-aged-10-65-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:05:00 +0000 https://clinicaltrials.eu/trial/study-on-rilzabrutinib-for-patients-aged-10-65-with-sickle-cell-disease/ This clinical trial is focused on studying the effects of a medication called rilzabrutinib in patients with sickle-cell disease. Sickle-cell disease is a blood disorder that can cause episodes of pain known as vaso-occlusive crises. The purpose of the study is to evaluate how well rilzabrutinib can prevent these painful episodes in patients aged 10 to 65 years.

Participants in the study will be randomly assigned to receive either rilzabrutinib or a placebo, which is a substance with no active medication. The study will last for 52 weeks, during which time the safety and effectiveness of rilzabrutinib will be closely monitored. The study aims to see if rilzabrutinib can reduce the number of painful episodes and improve the overall health of patients with sickle-cell disease.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. The study will also track the time it takes for the first painful episode to occur, the number of visits to the doctor due to complications, and any changes in fatigue levels. The goal is to gather comprehensive information on how rilzabrutinib affects patients with sickle-cell disease and to ensure the treatment is safe and effective.

]]> Study on the Safety and Effects of BMS-986470 for Patients with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-bms-986470-for-patients-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:04:58 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-bms-986470-for-patients-with-sickle-cell-disease/ This clinical trial is focused on studying Sickle Cell Disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The study will evaluate a new treatment called BMS-986470, which is taken as a film-coated tablet. The purpose of the study is to determine if BMS-986470 is safe and well-tolerated when given to both healthy individuals and those with sickle cell disease. Additionally, the study aims to find the best dose of BMS-986470 for future research in participants with sickle cell disease.

The study will involve participants taking the medication and being monitored for any side effects or adverse events. Some participants will receive a placebo, which is a substance with no active medication. The study will also look at how the body processes the drug and its effects on different types of hemoglobin, including fetal and sickle forms. Researchers will check if there are improvements in other markers related to sickle cell disease.

Participants will be involved in the study for a period of time, during which they will take the medication and undergo various tests and assessments. The study will help gather important information about the safety and potential benefits of BMS-986470 for people with sickle cell disease, contributing to the development of new treatment options for this condition.

]]> Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-how-well-etavopivat-works-for-adolescents-and-adults-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:03:57 +0000 https://clinicaltrials.eu/trial/study-on-how-well-etavopivat-works-for-adolescents-and-adults-with-sickle-cell-disease/ This clinical trial is focused on studying sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study will evaluate the effectiveness and safety of a treatment called etavopivat, also known by its code name FT-4202. Participants in the study will receive either etavopivat or a placebo, which is a substance with no active medication.

The purpose of the study is to determine if etavopivat is more effective than a placebo in treating adolescents and adults with sickle cell disease. Participants will take part in a series of assessments over the course of the study, which will last for a period of time. These assessments will help researchers understand how well the treatment works and its safety profile. The study will involve regular check-ups and monitoring to track any changes in the participants’ health and symptoms.

Throughout the study, researchers will pay close attention to the number of painful episodes known as vaso-occlusive crises, which are common in sickle cell disease. They will also monitor changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen, and other health indicators. The goal is to see if etavopivat can reduce these painful episodes and improve overall health outcomes for those living with sickle cell disease.

]]> Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide https://clinicaltrials.eu/trial/study-on-improving-treatment-for-sickle-cell-disease-patients-using-hydroxycarbamide/ Wed, 29 Apr 2026 15:01:42 +0000 https://clinicaltrials.eu/trial/study-on-improving-treatment-for-sickle-cell-disease-patients-using-hydroxycarbamide/ This clinical trial is focused on studying the management of patients with sickle cell disease, a genetic condition that affects the shape and function of red blood cells. The treatment being investigated is hydroxyurea, a medication that helps to reduce the frequency of painful episodes and other complications associated with sickle cell disease. The study involves different forms of hydroxyurea, including HYDREA 500 mg capsules and Siklos film-coated tablets in various dosages.

The purpose of the study is to optimize the treatment of sickle cell disease by comparing two different methods of monitoring patients who are taking hydroxyurea. Participants will be divided into two groups, each receiving a different approach to therapeutic follow-up. The study will observe how quickly patients reach a stable treatment level and will monitor various health parameters, such as the number of painful episodes, hospitalizations, and any side effects related to the medication.

Throughout the study, participants will take hydroxyurea orally, and their progress will be tracked over a period of time. The study aims to provide insights into the best ways to manage sickle cell disease with hydroxyurea, potentially improving the quality of life for those affected by this condition. The trial is expected to continue until 2025, with the goal of gathering comprehensive data on the effectiveness and safety of hydroxyurea in treating sickle cell disease.

]]> Study of GBT021601 for Adults and Children with Sickle Cell Disease https://clinicaltrials.eu/trial/study-of-gbt021601-for-adults-and-children-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:01:33 +0000 https://clinicaltrials.eu/trial/study-of-gbt021601-for-adults-and-children-with-sickle-cell-disease/ This clinical trial is focused on studying Sickle Cell Disease, a genetic condition that affects the shape and function of red blood cells. The study will evaluate a treatment called Osivelotor, which is taken orally in the form of a tablet. The purpose of the study is to understand how Osivelotor affects patients with sickle cell disease, particularly looking at changes in hemoglobin levels, which is a protein in red blood cells that carries oxygen throughout the body.

The study is divided into different parts. In one part, adult participants will receive osivelotor to see how it changes their hemoglobin levels over a period of time. Another part of the study will compare the effects of osivelotor to a placebo in both adult and adolescent participants, focusing on hemoglobin response and the frequency of painful episodes known as vaso-occlusive crises. Additionally, there is an open-label part of the study that will look at how osivelotor is processed in the bodies of pediatric participants, which means the participants and researchers know what treatment is being given.

Participants in the study will take the medication for a maximum of 48 weeks, and the effects will be monitored through regular check-ups and tests. The study aims to provide valuable information on the safety and effectiveness of osivelotor for treating sickle cell disease, potentially leading to improved management of this condition in the future.

]]> Study of Mitapivat for Adults with Sickle Cell Disease https://clinicaltrials.eu/trial/study-of-mitapivat-for-adults-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:01:10 +0000 https://clinicaltrials.eu/trial/study-of-mitapivat-for-adults-with-sickle-cell-disease/ This clinical trial is focused on studying sickle cell disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The treatment being tested in this study is a medication called mitapivat, also known by its code name AG-348. The purpose of the study is to evaluate the safety and effectiveness of mitapivat in treating sickle cell disease.

Participants in the study will take mitapivat in the form of a tablet. The study will be conducted over a period of time, during which participants will receive the medication and be monitored for any changes in their condition. The study will assess how well the medication works in reducing the sickling of red blood cells, which is a key problem in sickle cell disease. Additionally, the study will look at the safety of the medication by observing any side effects or changes in health indicators such as vital signs and heart function.

Throughout the study, participants will undergo various assessments to track their health and response to the treatment. These assessments will include checking blood levels, evaluating the flexibility of red blood cells, and monitoring overall health and quality of life. The study aims to provide valuable information on how mitapivat can help manage sickle cell disease and improve the lives of those affected by this condition.

]]> Study on Hydroxycarbamide Tablets for Children with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-hydroxycarbamide-tablets-for-children-with-sickle-cell-disease/ Wed, 29 Apr 2026 15:00:26 +0000 https://clinicaltrials.eu/trial/study-on-hydroxycarbamide-tablets-for-children-with-sickle-cell-disease/ This clinical trial is focused on studying Sickle Cell Disease in children. The treatment being tested is called Hydroxycarbamide, which is provided in the form of SIKLOS PAEDIATRIC 50mg dispersible tablets. The purpose of the study is to understand how this medication behaves in the body when given twice a day to children aged 9 months to 11 years.

During the study, children will receive the medication for up to 12 months. The study will monitor how the medication is absorbed and processed in the body over time. This includes checking the levels of the medication in the blood at different times and observing any changes in the children’s health. The study will also look at how the medication affects certain blood components, such as hemoglobin, which is important for carrying oxygen in the blood.

Throughout the study, researchers will keep track of any health events related to Sickle Cell Disease and any side effects that might occur. The study aims to gather information on how well children tolerate the medication and how easy it is for them and their caregivers to use the treatment. This information will help in understanding the safety and effectiveness of the medication for children with Sickle Cell Disease.

]]> Study of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease https://clinicaltrials.eu/trial/study-of-mitapivat-for-patients-with-sickle-cell-disease-and-kidney-disease/ Wed, 29 Apr 2026 14:58:35 +0000 https://clinicaltrials.eu/trial/study-of-mitapivat-for-patients-with-sickle-cell-disease-and-kidney-disease/ This clinical trial is focused on studying the effects of a medication called Mitapivat on patients with Sickle Cell Disease and Nephropathy, which is a type of kidney disease. Sickle Cell Disease is a genetic condition that affects the shape of red blood cells, leading to various health complications. The purpose of the study is to evaluate how Mitapivat affects the albumin creatinine ratio, a measure used to assess kidney function, in these patients.

Participants in the study will receive Mitapivat in the form of a tablet. The study will be conducted over a period of time, during which participants will take the medication and attend regular check-ups to monitor their health and the effects of the treatment. Some participants may receive a placebo instead of the actual medication. The study aims to observe changes in kidney function and other health indicators over the course of the treatment.

The trial will also track other health outcomes, such as the frequency of emergency room visits and hospitalizations, as well as any side effects experienced by participants. The goal is to determine whether Mitapivat can provide a beneficial effect on kidney function and overall health in patients with Sickle Cell Disease and Nephropathy.

]]> Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment https://clinicaltrials.eu/trial/long-term-safety-study-of-etavopivat-for-adults-adolescents-and-children-with-sickle-cell-disease-or-thalassemia/ Wed, 29 Apr 2026 14:57:44 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-etavopivat-for-adults-adolescents-and-children-with-sickle-cell-disease-or-thalassemia/ This clinical trial focuses on studying long-term treatment of sickle cell disease and thalassemia, which are inherited blood disorders that affect how red blood cells function in the body. The study uses etavopivat (also known as FT-4202), which is given as a 200 mg tablet taken by mouth. These conditions can cause severe pain, fatigue, and other serious health problems due to abnormal red blood cells.

The purpose of this research is to understand how safe and effective etavopivat is when used for an extended period in adults, teenagers, and children who have previously completed treatment with this medication in other studies. The medication will be given to participants who have shown improvement during their previous treatment with etavopivat.

During the study, patients may continue taking other medications they were already using for their condition, such as hydroxyurea, crizanlizumab, or L-glutamine, as long as their doses have remained stable. The study will monitor various aspects of the participants’ health, including any side effects, hospital stays, and changes in their blood cell measurements over time.

]]> Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises https://clinicaltrials.eu/trial/study-on-crizanlizumab-for-adolescents-and-adults-with-sickle-cell-disease-experiencing-vaso-occlusive-crises/ Wed, 29 Apr 2026 14:30:32 +0000 https://clinicaltrials.eu/trial/study-on-crizanlizumab-for-adolescents-and-adults-with-sickle-cell-disease-experiencing-vaso-occlusive-crises/ This clinical trial is focused on studying sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study is testing a treatment called crizanlizumab, which is also known by the code name SEG101. This treatment is a type of medication known as a monoclonal antibody, which is designed to target specific proteins in the body. The trial will compare two different doses of crizanlizumab to a placebo, with some participants also receiving standard treatments like hydroxyurea or hydroxycarbamide.

The purpose of the study is to evaluate how effective and safe crizanlizumab is in reducing the number of painful episodes, known as vaso-occlusive crises, that lead to healthcare visits in people with sickle cell disease. Participants in the study will receive either crizanlizumab or a placebo through an intravenous infusion, which means the medication is given directly into a vein. The study will last for about a year, during which time participants will have regular check-ups to monitor their health and the effects of the treatment.

Throughout the study, researchers will collect information on how often participants experience vaso-occlusive crises, how long these episodes last, and any other health changes that occur. This information will help determine if crizanlizumab is a beneficial treatment option for managing sickle cell disease. The study aims to provide valuable insights into improving care for individuals living with this condition.

]]> A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials https://clinicaltrials.eu/trial/study-for-patients-with-sickle-cell-disease-using-crizanlizumab-after-completing-a-previous-novartis-study/ Wed, 29 Apr 2026 14:30:28 +0000 https://clinicaltrials.eu/trial/study-for-patients-with-sickle-cell-disease-using-crizanlizumab-after-completing-a-previous-novartis-study/ This study focuses on Sickle Cell Disease, a condition where red blood cells become crescent-shaped and can block blood vessels, causing pain and other complications. The study will use crizanlizumab, which is given through an intravenous infusion (delivered directly into a vein). This medication is designed to help prevent blood cells from sticking together and forming blockages in blood vessels.

The purpose of this research is to continue providing crizanlizumab treatment to patients with Sickle Cell Disease who have previously participated in other studies of this medication. The treatment will be administered through an intravenous line every few weeks, with each dose calculated based on the patient’s weight. The maximum dose will be 7.5 milligrams per kilogram of body weight.

During the study, which may last up to 120 months, patients will receive regular infusions of crizanlizumab. The medical team will monitor the patients’ health and track any side effects that may occur during the treatment. This monitoring will help ensure patient safety throughout the study period.

]]> Long-term Safety and Efficacy Study of Lovotibeglogene Autotemcel Gene Therapy for Patients with Sickle Cell Disease https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-lovotibeglogene-autotemcel-gene-therapy-for-patients-with-sickle-cell-disease/ Wed, 29 Apr 2026 14:28:00 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-lovotibeglogene-autotemcel-gene-therapy-for-patients-with-sickle-cell-disease/ This clinical trial focuses on sickle cell disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study is examining a treatment called lovotibeglogene autotemcel, also known as bb1111 or LentiGlobin BB305 Drug Product for Sickle Cell Disease. This treatment involves a type of gene therapy where a patient’s own blood stem cells are modified outside the body using a special tool called a lentiviral vector to help correct the genetic defect causing the disease.

The purpose of the study is to evaluate the long-term safety and effectiveness of this treatment in individuals with sickle cell disease. Participants in the study will receive the treatment and then be monitored over an extended period to observe any changes in their health. The study will track various health outcomes, including the occurrence of immune-related issues, blood disorders, and any new or worsening neurological conditions. Additionally, the study will assess the treatment’s impact on the frequency and severity of painful episodes known as vaso-occlusive events, which are common in sickle cell disease.

Participants will be followed for up to 15 years to gather comprehensive data on the treatment’s effects. This long-term follow-up will help researchers understand how well the treatment works over time and identify any potential long-term side effects. The study aims to provide valuable insights into the potential benefits and risks of using gene therapy to treat sickle cell disease, ultimately contributing to improved care for individuals living with this condition.

]]> Study on the Effectiveness of Decitabine and Tetrahydrouridine in Patients with Sickle Cell Disease Not Eligible for Hydroxyurea Treatment https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-decitabine-and-tetrahydrouridine-in-patients-with-sickle-cell-disease-not-eligible-for-hydroxyurea-treatment/ Wed, 29 Apr 2026 14:27:53 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-of-decitabine-and-tetrahydrouridine-in-patients-with-sickle-cell-disease-not-eligible-for-hydroxyurea-treatment/ This clinical trial is focused on studying the effects of a new treatment for sickle cell disease, a condition that affects the red blood cells and can cause pain and other serious health problems. The treatment being tested is a combination of two substances, decitabine and tetrahydrouridine, which are taken as a capsule. The study will compare this treatment to a placebo to see how well it works in improving hemoglobin levels in patients who cannot use another medication called hydroxycarbamide.

The purpose of the study is to evaluate the effectiveness and safety of the decitabine-tetrahydrouridine combination. Participants will take the medication orally over a period of time, and their health will be monitored regularly. The study will last for several weeks, during which changes in hemoglobin and other health markers will be observed. The goal is to determine if the new treatment can help improve the condition of people with sickle cell disease.

Throughout the study, participants will undergo various assessments to track their progress. These assessments will include measuring the concentration of the medication in the blood and observing any changes in the number of painful episodes or other complications related to sickle cell disease. The study aims to provide valuable information on whether this new treatment can offer a better option for managing sickle cell disease in patients who are not eligible for existing treatments.

]]> Study on the Effects of Etavopivat for Patients Aged 12-65 with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-the-effects-of-etavopivat-for-patients-aged-12-65-with-sickle-cell-disease/ Wed, 29 Apr 2026 14:27:13 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-etavopivat-for-patients-aged-12-65-with-sickle-cell-disease/ This clinical trial is focused on studying sickle cell disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The study is testing a new treatment called Etavopivat, which is a type of medication known as a pyruvate kinase activator. This medication is being tested in two different doses, 100 mg and 200 mg, and will be compared to a placebo. The purpose of the study is to see if Etavopivat can help improve hemoglobin levels and reduce the number of painful episodes known as vaso-occlusive crises, which are common in people with sickle cell disease.

Participants in the study will be randomly assigned to receive either Etavopivat or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are not biased. The study will take place over a period of time, with regular check-ins to monitor the participants’ health and any changes in their condition. The main goal is to assess the effectiveness of Etavopivat in improving health outcomes for those with sickle cell disease.

Throughout the study, participants will have their hemoglobin levels checked and will be monitored for any changes in their condition. The study will also look at other health markers related to sickle cell disease, such as the number of red blood cells and certain proteins in the blood. By the end of the study, researchers hope to determine whether Etavopivat is a safe and effective treatment option for people living with sickle cell disease.

]]> Study on the Effect of Famotidine in Reducing P-selectin Levels in Children with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-the-effect-of-famotidine-in-reducing-p-selectin-levels-in-children-with-sickle-cell-disease/ Wed, 29 Apr 2026 14:24:59 +0000 https://clinicaltrials.eu/trial/study-on-the-effect-of-famotidine-in-reducing-p-selectin-levels-in-children-with-sickle-cell-disease/ This clinical trial is focused on studying the effects of a medication called Famotidine in children with Sickle Cell Disease. Sickle Cell Disease is a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study aims to explore whether Famotidine can help reduce the expression of a protein called P-selectin, which is involved in the inflammation and blockage of blood vessels, a common issue in Sickle Cell Disease.

Participants in the study will take Famotidine, which is administered as an oral suspension, for a period of 29 days. The study will monitor changes in the levels of P-selectin and other related molecules in the blood before and after the treatment. This will help researchers understand the potential benefits of Famotidine in managing Sickle Cell Disease symptoms. The study is designed as a pilot, meaning it is a preliminary investigation to gather initial data on the treatment’s effects.

Throughout the study, researchers will also observe any changes in blood values, such as hemoglobin and other markers, to assess the overall impact of Famotidine on the participants’ health. Additionally, the occurrence of any adverse events will be recorded to ensure the safety of the treatment. This trial is an important step in exploring new ways to improve the quality of life for children affected by Sickle Cell Disease.

]]> Study on Crovalimab for Preventing Painful Episodes in Sickle Cell Disease Patients https://clinicaltrials.eu/trial/study-on-crovalimab-for-preventing-painful-episodes-in-sickle-cell-disease-patients/ Wed, 29 Apr 2026 14:23:55 +0000 https://clinicaltrials.eu/trial/study-on-crovalimab-for-preventing-painful-episodes-in-sickle-cell-disease-patients/ This clinical trial is focused on studying Sickle Cell Disease, a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study is evaluating a treatment called Crovalimab, which is being tested to see if it can help prevent painful episodes known as vaso-occlusive episodes (VOE) that occur in people with this disease. These episodes happen when the sickle-shaped cells block blood flow, causing pain and potential damage to organs.

The purpose of the study is to assess how effective and safe Crovalimab is compared to a placebo. Participants in the study will receive either Crovalimab or a placebo, which is a substance with no active medication, to compare the outcomes. Crovalimab is administered as a solution for injection or infusion, meaning it is given directly into the bloodstream through a vein or under the skin. The study will monitor participants over a period to observe any changes in the frequency and severity of their VOEs, as well as other health measures.

Throughout the study, researchers will collect information on how often participants experience VOEs, both at home and in medical facilities, and any changes in their overall health. The study will also look at other health indicators, such as changes in blood tests and the time it takes for a participant to experience their first VOE after starting the study. This information will help determine if Crovalimab is a beneficial treatment option for people with Sickle Cell Disease.

]]> Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-crovalimab-for-treating-vaso-occlusive-episodes-in-sickle-cell-disease-patients/ Wed, 29 Apr 2026 14:23:51 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-crovalimab-for-treating-vaso-occlusive-episodes-in-sickle-cell-disease-patients/ This clinical trial is focused on studying Sickle Cell Disease (SCD), a genetic condition that affects the red blood cells, causing them to become misshapen and leading to various health complications. The study is investigating a treatment called Crovalimab, which is administered as a solution for injection or infusion. The purpose of the study is to evaluate the safety of Crovalimab compared to a placebo in managing acute uncomplicated vaso-occlusive episodes, which are painful events that occur when blood flow is blocked in small vessels.

Participants in the study will receive either Crovalimab or a placebo. The study will monitor the safety of the treatment by observing any adverse events, changes in vital signs, and any reactions related to the infusion. Additionally, the study will measure the levels of Crovalimab in the blood over time and assess how the drug interacts with the body, including its effects on specific biological markers. The study aims to understand how quickly patients experience improvement from their vaso-occlusive episodes and to check for any antibodies that might develop against the drug.

The trial is designed to gather information over a period of time, with the estimated completion date set for October 2025. Participants will be closely monitored throughout the study to ensure their safety and to collect data on the effectiveness of Crovalimab in treating Sickle Cell Disease. This research is important for developing new treatments that can help manage the symptoms and complications associated with this condition.

]]> Study on Tocilizumab for Treating Acute Chest Syndrome in Children and Adults with Sickle Cell Disease https://clinicaltrials.eu/trial/study-on-tocilizumab-for-treating-acute-chest-syndrome-in-children-and-adults-with-sickle-cell-disease/ Wed, 29 Apr 2026 14:23:20 +0000 https://clinicaltrials.eu/trial/study-on-tocilizumab-for-treating-acute-chest-syndrome-in-children-and-adults-with-sickle-cell-disease/ This clinical trial is focused on studying the treatment of Sickle Cell Disease, a genetic condition that affects the red blood cells, leading to various health complications. The study is specifically looking at a condition called Acute Chest Syndrome, which can occur in people with Sickle Cell Disease and involves symptoms like fever and breathing difficulties. The treatment being tested is a medication called Tocilizumab, which is given as an intravenous infusion, meaning it is administered directly into the bloodstream through a vein. Tocilizumab is being compared to a placebo to see if it can help patients recover more quickly from the need for extra oxygen and breathing support.

The purpose of the study is to evaluate how effective Tocilizumab is in helping patients with Acute Chest Syndrome due to Sickle Cell Disease. Participants in the study will receive a single dose of Tocilizumab, with the amount based on their weight. The study will monitor how quickly patients can stop using supplemental oxygen and other forms of breathing assistance. This will help determine if Tocilizumab can improve recovery times for these patients.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how well the treatment works. The study aims to provide valuable insights into the potential benefits of Tocilizumab for people with Sickle Cell Disease experiencing Acute Chest Syndrome, ultimately contributing to better treatment options for this serious condition.

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