Ongoing Clinical Trials for Sickle Cell Anaemia
Currently, there are 10 ongoing clinical trials investigating new treatments for sickle cell anaemia across Europe. These studies focus on advanced gene therapies using CRISPR technology, medications to prevent painful crises, and drugs to stabilize red blood cells. Trials are being conducted in several European countries including France, Germany, Italy, Belgium, Spain, Netherlands, and Finland.
Clinical trial locations
- Belgium
- Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
- A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials
- Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells
- Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises
- Finland
- France
- Evaluating Morphine Effectiveness Based on Kidney Function in Patients with Sickle Cell Disease During Vaso-occlusive Crisis
- Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease
- A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials
- Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises
- Study on Voxelotor for Reducing Hemolysis in Patients with Sickle Cell Disease
- Germany
- Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
- Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
- Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor
- A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials
- Italy
- Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
- Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
- Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor
- Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease
- A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials
- Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells
- Netherlands
- Spain
Long-term Safety Study of Exagamglogene Autotemcel for Patients with Sickle Cell Disease or Transfusion-Dependent Thalassemia
This study focuses on the long-term safety monitoring of patients who have already received CTX001, an advanced gene therapy using CRISPR-Cas9 technology. The treatment modifies a patient’s own blood-forming stem cells to restore natural production of fetal hemoglobin, which can help improve symptoms.
Main inclusion criteria: Participants must have already received a CTX001 infusion in a previous study. The trial is open to both male and female patients across age groups including older children, teenagers, and adults. Patients or their legal representatives must provide informed consent.
Main exclusion criteria: Patients who have not received CTX001 treatment previously cannot participate. The study excludes vulnerable populations who may require special protection or care.
Study focus: The primary goal is to evaluate the long-term safety of CTX001 by monitoring patients for new cancers, blood disorders, and other health issues over an extended period until 2040. Researchers will track changes in hemoglobin levels and genetic modifications in blood and bone marrow cells.
Investigational treatment: CTX001 is a one-time gene therapy that uses CRISPR-Cas9 gene editing to modify the patient’s own hematopoietic stem cells, delivered through intravenous infusion.
Study on the Effects of Exagamglogene Autotemcel for Patients with Transfusion-Dependent Beta-Thalassemia or Severe Sickle Cell Disease
This trial evaluates CTX001 therapy, which involves collecting a patient’s own blood stem cells, modifying them using CRISPR-Cas9 technology, and returning them to the patient to help produce more fetal hemoglobin and reduce symptoms.
Main inclusion criteria: Participants must have severe sickle cell disease with at least two vaso-occlusive crisis events per year for the past two years. Patients should have experienced failure or intolerance to hydroxyurea treatment and must be eligible for autologous stem cell transplant. Both adolescents and adults are eligible.
Main exclusion criteria: Patients not dependent on blood transfusions or without severe disease cannot participate. The study excludes patients outside the specified age range and those from vulnerable populations.
Study focus: The primary objective is to evaluate the effectiveness and safety of a single dose of CTX001. Researchers will monitor changes in fetal hemoglobin levels starting 60 days after the last red blood cell transfusion and continuing for up to 12 months.
Investigational treatment: CTX001 is administered as a single intravenous infusion. Supporting medications include filgrastim, busulfan, and plerixafor, which help prepare the body for treatment by mobilizing and collecting stem cells.
Study on the Safety and Effectiveness of CTX001 for Children with Severe Sickle Cell Disease Using Exagamglogene Autotemcel, Busulfan, and Plerixafor
This pediatric study tests CTX001 gene therapy specifically in children with severe disease, using the same CRISPR-Cas9 technology to modify stem cells and improve red blood cell production.
Main inclusion criteria: Children must have a diagnosis of severe disease with at least two severe vaso-occlusive crises per year for the previous two years. They must have experienced hydroxyurea treatment failure unless intolerant, and must be suitable for autologous stem cell transplant.
Main exclusion criteria: Children who do not meet the age requirements or do not have severe disease cannot participate. Pregnant or breastfeeding patients are excluded, as are those unable to provide consent or with certain severe organ damage.
Study focus: The primary goal is to achieve at least 12 consecutive months without severe vaso-occlusive crises, beginning 60 days after the last red blood cell transfusion. The study will monitor participants for up to 24 months to assess reduction in painful episodes and hospitalizations.
Investigational treatment: CTX001 is delivered as a single intravenous infusion. Plerixafor helps mobilize stem cells for collection, while busulfan prepares the body to receive the modified cells.
Evaluating Morphine Effectiveness Based on Kidney Function in Patients with Sickle Cell Disease During Vaso-occlusive Crisis
This study examines how kidney function affects morphine’s effectiveness in managing severe pain during vaso-occlusive crises, which occur when sickle-shaped red blood cells block blood vessels.
Main inclusion criteria: Patients must be 18 years or older with confirmed homozygous disease (types SS, SC, S-beta+ or S-beta0). They must be admitted to a continuous care unit or intensive care unit with vaso-occlusive crisis or acute chest syndrome and receiving morphine patient-controlled analgesia.
Main exclusion criteria: Patients under 18 or over 75 years old cannot participate. Pregnant or breastfeeding women, patients with kidney disease, and those with previous allergic reactions to morphine or iohexol are excluded.
Study focus: Researchers will measure kidney function using iohexol clearance and evaluate how this affects morphine processing in the body. The goal is to determine when standard morphine doses might not provide adequate pain relief, potentially leading to more personalized pain management strategies.
Investigational treatment: Morphine hydrochloride (1 mg/ml solution) is administered through patient-controlled analgesia. Iohexol is used as a contrast agent to accurately measure kidney function.
Study on the Effectiveness and Safety of Exa-cel for Adolescents and Adults with Severe Sickle Cell Disease
This trial specifically studies Exa-cel (exagamglogene autotemcel) in patients with the βS/βC genotype, a specific type of disease. The treatment uses CRISPR-Cas9 gene editing to restore natural fetal hemoglobin production.
Main inclusion criteria: Participants must have documented βS/βC genotype with severe disease, defined by at least two yearly events including acute pain crises, acute chest syndrome, priapism, or splenic sequestration over the past two years. They must have a performance status of 80% or higher and be eligible for autologous stem cell transplant.
Main exclusion criteria: Individuals without the specific βS/βC genotype or who are not adolescents or adults cannot participate. Vulnerable populations are excluded.
Study focus: The study evaluates the absence of severe vaso-occlusive crises for at least 12 consecutive months beginning 60 days after the last red blood cell transfusion. Participants will be monitored for adverse events and health improvements. Long-term follow-up continues for 15 years after treatment.
Investigational treatment: Exa-cel is administered as a single intravenous infusion. Mozobil mobilizes stem cells, and busulfan serves as conditioning treatment to prepare the body for the modified cells.
Study on the Effects of Mitapivat on Brain Blood Flow and Oxygen Use in Patients with Sickle Cell Anemia
This study investigates how mitapivat, taken as an oral tablet, affects brain oxygen use and blood flow. The medication works by activating an enzyme in red blood cells to help them function better.
Main inclusion criteria: Participants must be 18 years or older with documented genotypes HbSS or HbSβ0-thalassemia and hemoglobin levels of 10.5 g/dL or less. If taking hydroxyurea, the dose must be stable for at least 90 days. Female participants of childbearing potential must use effective contraception.
Main exclusion criteria: Patients with conditions that might interfere with the study, those who are pregnant or breastfeeding, and patients who have participated in other recent clinical trials may not be eligible.
Study focus: Researchers will use MRI scans at three and twelve months to assess the effects of mitapivat on brain oxygen metabolism and blood flow. The study aims to determine whether mitapivat can improve brain function and circulation.
Investigational treatment: Mitapivat is taken orally in tablet form. It is classified as a pyruvate kinase activator, helping red blood cells produce energy more effectively.
A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials
This continuation study allows patients who benefited from crizanlizumab in previous trials to continue receiving treatment. Crizanlizumab prevents blood cells from sticking together and blocking vessels.
Main inclusion criteria: Participants must be adults (18 years or older) currently participating in and benefiting from a Novartis study receiving crizanlizumab. They must have completed all requirements of their current study and shown good attendance at scheduled visits.
Main exclusion criteria: Patients not currently receiving crizanlizumab in a Novartis-sponsored study or without confirmed disease diagnosis cannot participate. Pregnant or breastfeeding women and patients with severe allergic reactions to crizanlizumab are excluded.
Study focus: The study continues monitoring patients receiving regular crizanlizumab infusions, tracking safety and treatment-related effects. The trial is scheduled to run until June 2031.
Investigational treatment: Crizanlizumab (SEG101) is administered through intravenous infusion every few weeks at a maximum dose of 7.5 mg per kilogram of body weight. It is a monoclonal antibody that binds to P-selectin to prevent blood cell adhesion.
Study of Exagamglogene Autotemcel (CTX001) for Treatment of Severe Sickle Cell Disease Using Modified Stem Cells
This trial evaluates CTX001 gene therapy in patients with severe disease. The patient’s own stem cells are collected, modified using CRISPR-Cas9 technology in a laboratory, and returned to produce healthier red blood cells.
Main inclusion criteria: Participants must have confirmed severe disease with at least two severe pain crises per year in the past two years. They must be considered suitable for stem cell transplant and must be adolescents or adults (12 years or older).
Main exclusion criteria: Patients with prior stem cell transplantation, active hepatitis or HIV infections, significant heart, lung, liver or kidney disease, current pregnancy or breastfeeding, or active cancer cannot participate. Those unable to provide informed consent or with severe organ damage are also excluded.
Study focus: The primary goal is to achieve at least 12 consecutive months without severe vaso-occlusive crises, starting 60 days after the last red blood cell transfusion. Monitoring continues for 24 months to assess reduction in painful episodes and hospitalizations.
Investigational treatment: CTX001 is delivered as a single intravenous infusion of CRISPR-Cas9 modified CD34+ stem cells. Plerixafor helps mobilize stem cells for collection, and busulfan prepares the body for treatment.
Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises
This trial compares two different doses of crizanlizumab to a placebo in reducing painful episodes that lead to healthcare visits. Some participants may also receive standard treatments like hydroxyurea.
Main inclusion criteria: Participants must be 12 years or older with confirmed disease and at least two vaso-occlusive crises requiring healthcare visits in the past 12 months. Patients taking hydroxyurea must have been on a stable dose for at least 3 months. Laboratory values must meet specific requirements for neutrophil count, platelet count, hemoglobin, kidney function, and liver function.
Main exclusion criteria: Patients outside the specified age range or not part of the trial groups cannot participate. Gender is not an exclusion criterion. Vulnerable populations may not be eligible.
Study focus: The study evaluates crizanlizumab’s effectiveness in reducing the frequency and severity of vaso-occlusive crises over approximately one year. Researchers will monitor how often participants experience painful episodes, how long they last, and any other health changes.
Investigational treatment: Crizanlizumab or placebo is administered through intravenous infusion at doses of either 7.5 mg/kg or 5.0 mg/kg. Hydroxyurea may be used as background therapy for some participants.
Study on Voxelotor for Reducing Hemolysis in Patients with Sickle Cell Disease
This trial tests voxelotor, an oral medication designed to reduce the breakdown of red blood cells by stabilizing hemoglobin and increasing its oxygen-carrying capacity.
Main inclusion criteria: Participants must be 18 years or older with SS or S-β0 major type disease and hemoglobin levels less than 9 g/dL. If receiving treatments like hydroxyurea, EPO, ACE inhibitors, ARBs, glutamine, or crizanlizumab, doses must be stable for at least 3 months. Female participants must have negative pregnancy tests and use effective birth control.
Main exclusion criteria: The specific exclusion criteria are not detailed in the available information, but typical exclusions would include conditions that might interfere with study participation or medication safety.
Study focus: The primary assessment measures the reduction of hemolysis by evaluating plasma hemoglobin levels over 48 weeks. Secondary assessments include measuring blood volumes, brain blood flow, cognitive performance, kidney function, blood viscosity, and red blood cell properties.
Investigational treatment: Voxelotor (Oxbryta) is taken orally as 500 mg film-coated tablets. It works by binding to hemoglobin, increasing its affinity for oxygen, thereby stabilizing red blood cells and reducing their breakdown.
Summary
The current landscape of clinical trials for sickle cell anaemia in Europe demonstrates a strong focus on innovative gene therapies using CRISPR-Cas9 technology. Six of the ten trials involve CTX001 or Exa-cel, advanced treatments that modify patients’ own stem cells to produce healthier red blood cells. This concentration reflects the promising potential of gene editing as a transformative treatment approach.
France and Italy lead in trial availability, each hosting five studies, followed by Belgium and Germany with four trials each. Spain, Netherlands, and Finland have more limited participation with one or two trials each. This geographic distribution may influence treatment access for patients across Europe.
Three trials focus on crizanlizumab, a monoclonal antibody that prevents blood cell adhesion and reduces painful crises. Other approaches include mitapivat for improving brain blood flow, voxelotor for stabilizing red blood cells, and a study on optimizing morphine pain management based on kidney function.
Most trials target adolescents and adults, with one specifically designed for pediatric patients. The studies generally require participants to have severe disease with frequent vaso-occlusive crises, though eligibility criteria vary regarding prior treatments and specific genetic types. Patients interested in participating should consult with their healthcare providers to determine which trials might be appropriate for their specific situation.
