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Ongoing Clinical Trials for Sickle Cell Disease

There are currently 19 clinical trials ongoing across Europe for patients with sickle cell disease (also known as sickle cell anemia, drepanocytosis). These studies are testing various treatments including gene therapies, medications to reduce painful crises, and drugs to improve hemoglobin levels. Trials are being conducted in multiple countries including France, Spain, Italy, Germany, Belgium, Netherlands, Greece, Ireland, and Finland.

Clinical trial locations

Long-term Safety and Efficacy Study of Lovotibeglogene Autotemcel Gene Therapy for Patients with Sickle Cell Disease

This study evaluates a one-time gene therapy treatment called lovotibeglogene autotemcel (also known as lovocel, bb1111, or LentiGlobin BB305). The treatment involves collecting a patient’s own blood stem cells, modifying them outside the body using a lentiviral vector to help correct the genetic defect, and then returning them to the patient.

Main inclusion criteria: Participants must have sickle cell disease and must have previously received treatment with this drug product in a prior clinical study sponsored by bluebird bio. Written informed consent must be provided by the patient or their parent/legal guardian. Both male and female patients of all eligible ages may participate.

Main exclusion criteria: Patients without sickle cell disease, those not within the specified age range, individuals unwilling to follow study procedures, those with interfering medical conditions, pregnant or breastfeeding women, recent participants in other clinical trials, and those with allergies to the study medication cannot participate.

Main focus: The primary goal is to evaluate the long-term safety and effectiveness of this gene therapy over a 15-year follow-up period. The study monitors immune-related issues, blood disorders, neurological conditions, and vaso-occlusive events (painful episodes caused by blocked blood flow).

Investigational drug: Lovocel is a gene therapy using the patient’s own modified blood stem cells to produce healthier red blood cells that can reduce symptoms and complications.

Long-term safety study of oral etavopivat tablets in children, adolescents and adults with sickle cell disease or thalassaemia who completed previous etavopivat treatment

This study examines the long-term safety of etavopivat tablets (200 mg) taken by mouth in participants who previously completed treatment with this medication in other studies and showed clinical benefits.

Main inclusion criteria: Participants must have completed a treatment period in another etavopivat study and received clinical benefits from previous treatment. If there were dose reductions or interruptions, participants need to restart treatment before entering this study. Patients may continue taking other medications such as hydroxyurea, crizanlizumab, or L-glutamine if they have been on stable doses. Both male and female patients, including children, adolescents, and adults, are eligible.

Main exclusion criteria: Those who have not previously participated in etavopivat studies, individuals under 2 years of age, pregnant or breastfeeding women, patients with severe liver or kidney problems, uncontrolled high blood pressure, active infections, severe allergic reactions to medications, those taking certain interacting medications, participants in other concurrent trials, those who had major surgery within 30 days, patients with significant heart conditions, and those with unstable medical conditions cannot participate.

Main focus: The study monitors long-term safety by tracking side effects, hospital stays, and changes in hemoglobin levels (a protein that carries oxygen in the blood) over time until November 2029.

Investigational drug: Etavopivat is a medication that modifies how red blood cells function by activating the pyruvate kinase-R enzyme, potentially improving cellular metabolism and reducing sickling of red blood cells.

Study on How Well Etavopivat Works for Adolescents and Adults with Sickle Cell Disease

This double-blind study evaluates whether etavopivat (200 mg tablets) is more effective than placebo in treating adolescents and adults with sickle cell disease.

Main inclusion criteria: Participants must be at least 12 years old at consent signing, have confirmed sickle cell disease documented through lab tests, have experienced 2-15 vaso-occlusive crises in the past year that are documented in medical records, and have hemoglobin levels between 5.0 and 10.0 g/dL at screening. Both males and females are eligible.

Main exclusion criteria: Patients without sickle cell disease, those not meeting the age requirements, individuals unable to provide consent, and those considered part of vulnerable groups requiring extra protection cannot participate.

Main focus: The primary outcome is the number of vaso-occlusive crisis events requiring medical contact. Secondary outcomes include changes in hemoglobin levels, fatigue scores, and physical performance measured by the 6-minute walking test.

Investigational drug: Etavopivat works by activating enzymes that help red blood cells maintain normal shape and function, reducing the sickling process and improving oxygen-carrying capacity.

Study on Hydroxycarbamide Tablets for Children with Sickle Cell Disease

This study investigates how hydroxycarbamide (SIKLOS PAEDIATRIC 50mg dispersible tablets) behaves in the body when given twice daily to children aged 9 months to 11 years for up to 12 months.

Main inclusion criteria: Both parents or a legal representative must provide written informed consent. Children must have HbSS or HbSβ0 types of sickle cell disease, be between 9 months and 11 years old, have no previous treatment with hydroxycarbamide, have specific laboratory values (Absolute Neutrophil Count ≥1.0×109/L, Platelets ≥75×109/L, Hemoglobin >5.5 g/dL), and be covered by social security. For children over 18 months, a Transcranial Doppler test must show low stroke risk.

Main exclusion criteria: Children younger than 9 months or older than 11 years, those without sickle cell disease, participants unable to take medication twice daily, and those not part of the selected vulnerable population cannot participate.

Main focus: The study monitors how the medication is processed in the body by measuring drug concentration at 1, 3, 6, 9, and 12 months, along with changes in blood parameters and tracking any sickle cell disease events or adverse effects.

Investigational drug: Hydroxycarbamide increases fetal hemoglobin levels, reducing painful episodes and complications by inhibiting ribonucleotide reductase at the molecular level.

Study on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease

This Phase 2 study compares different doses of tebapivat (also called AG-946) versus placebo to determine its effect on anemia in people with sickle cell disease over a 12-week double-blind period, with an optional open-label extension where all receive tebapivat.

Main inclusion criteria: Participants must be at least 16 years old (18 in France), have confirmed sickle cell disease, have hemoglobin levels between 5.5 and 10.5 g/dL measured at least twice during screening. If taking hydroxyurea, the dose must be stable for at least 90 days before randomization, or if recently stopped, a 90-day washout period is required. Women of childbearing age and men with partners who can become pregnant must use effective birth control.

Main exclusion criteria: Those with sickle cell types other than HbSS or HbSβ0 thalassemia, recent blood transfusion recipients (within 90 days), pregnant or breastfeeding women, patients with severe kidney or active liver disease, uncontrolled high blood pressure, those on unstable hydroxyurea doses, current participants in other trials, those with recent vaso-occlusive crises or hospitalizations (within 30 days), and individuals with substance abuse history or serious mental health conditions cannot participate.

Main focus: The primary outcome measures whether hemoglobin increases by at least 1.0 g/dL from baseline between weeks 10-12. The study also tracks blood markers, pain crises, fatigue levels, and conducts cognitive and physical function tests.

Investigational drug: Tebapivat is a pyruvate kinase activator that may increase energy metabolism in red blood cells and potentially reduce sickling.

Study on the Safety and Effects of BMS-986470 for Patients with Sickle Cell Disease

This early-stage study evaluates BMS-986470 (film-coated tablets) to determine if it is safe and well-tolerated in both healthy individuals and those with sickle cell disease, while finding the optimal dose for future research.

Main inclusion criteria: Participants must be at least 18 years old with documented sickle cell disease (HbSS, HbSβ0-thalassemia, or HbSβ+-thalassemia genotypes), have experienced at least 4 vaso-occlusive crises in the past 12 months or at least 2 in the past 6 months, have tried and failed or cannot tolerate approved therapies, and meet specific blood test requirements including hemoglobin levels between 5.5-12 g/dL for males or 5.5-10.6 g/dL for females, absolute neutrophil count ≥1500/μL, platelet count ≥100,000/μL, and absolute reticulocyte count requirements.

Main exclusion criteria: Those without sickle cell disease, individuals outside the specified age range, and those considered part of vulnerable populations cannot participate.

Main focus: The study monitors safety and tolerability through regular assessments while observing effects on different types of hemoglobin (including fetal and sickle forms) and other markers related to the condition.

Investigational drug: BMS-986470 is an investigational drug targeting specific pathways to improve the health and function of red blood cells, though it is not yet approved for general medical use.

Study on Tocilizumab for Treating Acute Chest Syndrome in Children and Adults with Sickle Cell Disease

This study evaluates whether tocilizumab, given as a single intravenous infusion, can help patients recover more quickly from acute chest syndrome (characterized by fever and breathing difficulties) compared to placebo.

Main inclusion criteria: Patients must have sickle cell disease of any genotype (SS, SC, S/β0, and S/β+), be at least 2 years old, be hospitalized for acute chest syndrome (defined as having fever and/or acute respiratory symptoms with a new lung issue on chest imaging), and require supplemental oxygen of at least 2 liters per minute or respiratory support for less than 48 hours. Girls or women of childbearing age must have negative pregnancy tests. Written informed consent from patient or legal representatives is required, along with health insurance coverage and agreement to use effective birth control for up to 6 months after treatment.

Main exclusion criteria: Patients without sickle cell disease, those not experiencing acute chest syndrome, and those not using supplemental oxygen or respiratory support cannot participate.

Main focus: The primary goal is evaluating the time to successfully stop using supplemental oxygen and respiratory support, defined as maintaining oxygen saturation of 95% or higher without oxygen for 24 hours and breathing independently for 48 hours.

Investigational drug: Tocilizumab is an immunosuppressive drug administered intravenously that blocks a specific protein causing inflammation, potentially reducing the need for oxygen and breathing support by targeting the inflammatory immune response.

Study of GBT021601 for Adults and Children with Sickle Cell Disease

This multi-part study evaluates osivelotor (GBT021601) tablets taken orally in adults, adolescents, and children to understand changes in hemoglobin levels and effects on vaso-occlusive crises over periods up to 48 weeks.

Main inclusion criteria: Participants must have sickle cell disease with HbSS or HbSB genotypes confirmed by lab tests, have experienced 2-10 vaso-occlusive crises in the past year requiring healthcare visits, have hemoglobin levels between 5.5 and 10.5 g/dL during screening, and if taking hydroxyurea or L-glutamine, must be on stable doses for at least 90 days with evidence of insufficient pain control. Different age cohorts are included: adults and adolescents 12 years or older (Part B), and pediatric cohorts ranging from 6 months to under 18 years (Part C).

Main exclusion criteria: Those without sickle cell disease, individuals outside specified age ranges, those unwilling to follow study procedures, patients with interfering medical conditions or taking conflicting medications, pregnant or breastfeeding women, recent clinical trial participants, and those with allergic reactions to the study medication cannot participate.

Main focus: Part A measures hemoglobin changes over 12 weeks in adults. Part B compares hemoglobin response and vaso-occlusive crisis rates between osivelotor (150 mg once daily) and placebo over 48 weeks in participants aged 12 and older. Part C assesses how the body processes osivelotor in pediatric participants divided into age cohorts.

Investigational drug: Osivelotor is a hemoglobin modulator that works by activating enzymes to help red blood cells maintain normal shape and function, potentially reducing sickling and frequency of vaso-occlusive crises.

Study of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease

This study evaluates how mitapivat tablets affect kidney function by measuring the albumin creatinine ratio (a kidney health measure) in patients with both sickle cell disease and nephropathy (kidney disease).

Main inclusion criteria: Participants must be at least 16 years old (18 in France), females must have started menstruating, have confirmed HbSS or HbS/β0-thalassemia sickle cell disease, have hemoglobin levels between 5.5 and 10.5 g/dL during screening, have two urine tests showing albumin creatinine ratio between 100 and 2000 mg/g during screening with at least one over 100 mg/g in the last 24 weeks, and if taking hydroxyurea or ACE inhibitors/ARB therapy, must be on stable doses for at least 90 days. Women of childbearing potential must agree to use two forms of birth control until 28 days after the last dose.

Main exclusion criteria: Patients without both sickle cell disease and nephropathy, those outside specified age ranges, and those considered part of vulnerable populations cannot participate.

Main focus: The primary outcome measures changes in albumin creatinine ratio from baseline to six months. Secondary outcomes include changes in estimated glomerular filtration rate and frequency of emergency room visits and hospitalizations, with follow-up continuing until June 2028.

Investigational drug: Mitapivat is a pyruvate kinase activator administered orally in tablet form that plays a role in energy production in red blood cells, potentially improving kidney function in patients with these conditions.

Study on Improving Treatment for Sickle Cell Disease Patients Using Hydroxycarbamide

This study compares two different methods of monitoring patients taking hydroxyurea (available as HYDREA 500 mg capsules or Siklos film-coated tablets in various dosages) to optimize treatment management.

Main inclusion criteria: Participants must be between 2 and 35 years old, able to understand study goals and provide signed consent (with parental/legal guardian consent for minors), have HbSS sickle cell disease, have been hospitalized for a vaso-occlusive crisis in the last 3 months and need treatment adjustment, be covered by health insurance, and have been informed of prior medical examination results. Women of childbearing age (15 or older) must have negative pregnancy tests and agree to effective birth control during the study and for 182 days after. Men who can have children must also agree to birth control during the study and for 92 days after.

Main exclusion criteria: The specific exclusion criteria are not fully detailed in the provided information.

Main focus: The study observes how quickly patients reach stable treatment levels and monitors various parameters such as painful episodes, hospitalizations, side effects, blood counts, and fetal hemoglobin percentage, with expected completion by August 7, 2025.

Investigational drug: Hydroxyurea, classified as an antineoplastic agent though used here for blood disorder benefits, is administered orally and works by increasing fetal hemoglobin production through inhibiting ribonucleotide reductase, helping reduce sickling and painful crises.

Study on Rilzabrutinib for Patients Aged 10-65 with Sickle Cell Disease

This 52-week double-blind study evaluates how well rilzabrutinib (coated tablets taken orally) can prevent painful vaso-occlusive crises in patients with sickle cell disease compared to placebo.

Main inclusion criteria: Participants must be at least 10 years old (at least 18 in France), have confirmed sickle cell disease diagnosis, have experienced 2-10 documented vaso-occlusive crises within 12 months before screening, and either not be taking hydroxyurea/L-glutamine or have been on stable doses for at least 3 months before screening. ECOG performance status must be grade 2 or lower for adults, or Karnofsky score 50% or more for adolescents. For participants aged 10 to under 18 years, parent or legal guardian must provide written informed consent.

Main exclusion criteria: Patients with sickle cell types other than HbSS or HbSβ0 thalassemia, recent blood transfusion recipients (within 90 days), pregnant or breastfeeding women, those with severe kidney or active liver disease, uncontrolled high blood pressure, unstable hydroxyurea doses, current participants in other trials, those with recent vaso-occlusive crises or hospitalizations requiring medical care (within 30 days), and individuals with substance abuse history or serious mental health conditions cannot participate.

Main focus: The study tracks the frequency and severity of painful episodes while monitoring safety through regular health assessments, blood tests, questionnaires about fatigue and pain, and recording symptoms in patient diaries throughout the 52-week period.

Investigational drug: Rilzabrutinib is a Bruton’s tyrosine kinase inhibitor that targets specific parts of the immune system potentially contributing to blood vessel blockages, aiming to keep blood flowing smoothly and reduce painful crises.

Summary

The 19 ongoing clinical trials for sickle cell disease represent a diverse range of therapeutic approaches being investigated across Europe. France stands out as the most active country with participation in 16 trials, followed by Spain and Italy with 9 trials each, and Germany with 5 trials. Several trials are being conducted simultaneously across multiple countries, reflecting strong European collaboration in sickle cell disease research.

A significant focus of current research involves pyruvate kinase activators, with multiple trials testing etavopivat, mitapivat, and tebapivat. These medications aim to improve red blood cell metabolism and reduce sickling. Etavopivat appears in several studies, including long-term safety evaluations and efficacy trials comparing different doses. Gene therapy is also being explored, with one trial examining the long-term effects of lovotibeglogene autotemcel over 15 years.

Another notable trend is the investigation of medications targeting the inflammatory and adhesion processes underlying vaso-occlusive crises. Crizanlizumab, a P-selectin inhibitor, appears in multiple trials, including studies for patients who previously participated in other crizanlizumab research. Rilzabrutinib, crovalimab, and tocilizumab are similarly being tested for their potential to prevent or treat painful episodes.

Several trials specifically target pediatric populations, including children as young as 6 months to 2 years, demonstrating a commitment to developing treatments suitable for younger patients. Some studies focus on specific complications such as acute chest syndrome or kidney disease associated with sickle cell disease.

The trials encompass various study phases, from early-stage safety evaluations to later-phase efficacy studies, and include both short-term assessments and long-term follow-up extending up to 15 years. This comprehensive approach should provide valuable data on both immediate benefits and long-term safety of emerging treatments for sickle cell disease.

Ongoing Clinical Trials on Sickle cell disease

  • Study of GBT021601 for Adults and Children with Sickle Cell Disease

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany

  • Study on the Effects of Tebapivat and AG-946 in Patients with Sickle Cell Disease

    Not recruiting

    1
    Investigated diseases:
    Belgium France Ireland The Netherlands

  • Study of Mitapivat for Adults with Sickle Cell Disease

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Ireland

  • Study on Crizanlizumab for Adolescents and Adults with Sickle Cell Disease Experiencing Vaso-Occlusive Crises

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Finland France The Netherlands Spain

  • A Phase 4 Study of Crizanlizumab Treatment for Patients with Sickle Cell Disease Who Previously Participated in Novartis Clinical Trials

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany Italy Spain

  • Study on the Effectiveness of Decitabine and Tetrahydrouridine in Patients with Sickle Cell Disease Not Eligible for Hydroxyurea Treatment

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Greece Italy Spain

  • Study on the Effects of Etavopivat for Patients Aged 12-65 with Sickle Cell Disease

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Greece Italy Spain

  • Study on the Effect of Famotidine in Reducing P-selectin Levels in Children with Sickle Cell Disease

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on the Safety and Effects of Crovalimab for Treating Vaso-Occlusive Episodes in Sickle Cell Disease Patients

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    France Italy The Netherlands Spain

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