- authorised (with conditions)Phase II and Phase III (Integrated)
C5351004 - A Phase 2/3 Randomized, Multicenter Study of Osivelotor Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease
This study is testing a new oral medicine, osivelotor, for adults with sickle cell disease. It aims to see how well it works to increase healthy blood cells and reduce pain crises, and how safe it is compared to a dummy pill.
Germany · France - active not recruitingPHASE2, PHASE3
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This study is looking at a new medicine called mitapivat for sickle cell disease. It aims to find the right dose and see if it helps increase healthy red blood cells and reduce painful crises.
United States · Belgium · Brazil - active not recruitingPHASE2
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
This study is looking at a new medicine called IMR-687 for adults with sickle cell disease. It's for people who were already in a previous study of this medicine and now want to see how safe it is over a longer time.
United States · United Kingdom - recruitingPHASE3
RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease
This study is looking at a specific stem cell transplant for adults with severe sickle cell disease. It aims to see if this treatment is effective and good value for money, especially for those who haven't responded to other treatments.
United Kingdom - recruitingPHASE3
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
This study looks at a new drug, Rilzabrutinib, for people aged 10-65 with sickle cell disease. We'll see how well it works and if it's safe compared to a dummy pill to help reduce painful crises.
United States · Belgium · Brazil - active not recruitingPHASE3
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This study is testing a new tablet medicine called etavopivat for people aged 12 to 65 with sickle cell disease. It aims to see if the medicine can improve a patient's red blood cells and reduce painful sickle cell crises. Half the group will receive the new treatment, the other a placebo.
United States · Canada · Egypt - recruiting
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This study looks at how safe and effective a medicine called Xromi is for very young children (under 2) with sickle cell disease. It compares children taking Xromi to similar children who are not, to see how it helps and if there are any side effects.
Germany · United Kingdom - authorisedTherapeutic exploratory (Phase II)
Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease
This study is testing a new medicine called anumigilimab for adults with sickle cell disease. It's a key step to see if the medicine is safe and how the body reacts to it, compared to a dummy treatment. We're looking closely at any side effects and how healthy people are during the study.
Belgium · Netherlands · France - active not recruitingPHASE3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This study is testing a new gene therapy, CTX001, for children aged 2-11 with severe sickle cell disease. It's for those whose current medicine, hydroxyurea, isn't working well or can't be taken. The aim is to see if this new treatment is safe and effective.
United States · Germany · Italy - enrolling by invitationPHASE3
A Long-term Follow-up Study in Participants Who Received CTX001
This study is looking at the long-term safety and how well a treatment called CTX001 works for children and adults with serious blood conditions like beta-thalassemia and sickle cell disease. It's for people who have already had CTX001 in a previous study.
United States · Belgium · Canada - active not recruitingPHASE2
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
This study is testing a new medicine called tebapivat for sickle cell disease. Researchers want to see if it helps improve anaemia and if different doses have different effects, compared to a dummy medicine (placebo).
United States · Belgium · Canada - recruitingPHASE3
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
This study is testing a new medicine, Etavopivat, for sickle cell disease and thalassaemia. It looks at how safe and effective the treatment is over a long period for people already benefiting from it in previous studies. The goal is to improve oxygen delivery in the body.
United States · Canada · Egypt - recruitingPHASE2, PHASE3
A Phase 2/3 Study in Adult and Adolescent Participants With SCD
This study is looking at a new medicine called osivelotor for people with sickle cell disease. It aims to see if it's safe, how well it works, and what dose is best. Both adults and teenagers can take part.
United States · Brazil · India - ongoing, recruitingTherapeutic use (Phase IV)
FAMODREP : Interest of Famotidine in Reducing Endothelial Expression of P-selectin in Children With Sickle Cell Disease: Pilot Study, Single-center, Prospective, Non-comparative
This study looks at whether a medicine called famotidine can help children with sickle cell disease. Researchers want to see if famotidine can reduce a specific marker in the blood that is linked to this condition. It's a small, early study to learn more.
France - ongoing, recruitingTherapeutic exploratory (Phase II)
A RANDOMIZED DOUBLE-BLIND PHASE IIA STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB AS ADJUNCT TREATMENT IN PREVENTION OF VASO-OCCLUSIVE EPISODES (VOE) IN SICKLE CELL DISEASE (SCD)
This study is testing a new medicine, crovalimab, for adults with sickle cell disease. It aims to see if this medicine can help prevent painful crises, called vaso-occlusive episodes (VOEs), and other problems by working alongside their usual treatment.
Netherlands · Spain · France - ongoing, recruitingHuman Pharmacology (Phase I)- Other
A Phase IB, Randomized, Placebo-Controlled Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso‑Occlusive Episodes (VOE) in Patients With Sickle Cell Disease (SCD)
This study is testing a new medicine, crovalimab, for people with sickle cell disease who are having a pain crisis (called a vaso-occlusive episode). We want to see if it's safe, how it works in the body, and if it helps reduce pain and other problems.
France · Netherlands · Spain - ongoing, recruitingTherapeutic exploratory (Phase II)
A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease
This study is testing a new combination medicine for sickle cell disease. It aims to see if it can improve a patient's red blood cells and overall health over about a year, while also checking for any side effects.
France · Italy · Greece - ongoing, recruitingTherapeutic exploratory (Phase II)
HEMolyse and Organ damage imPROvement in sickle cell disease by VoxElotor. An open-label one stage phase II design.HEMOPROVE
This study, called HEMOPROVE, is looking at a new medicine called Oxbryta for adults with sickle cell disease. Researchers want to see if this medicine can help reduce damage to red blood cells and improve how organs work, specifically by lowering a harmful type of haemoglobin in the blood.
France - recruitingPHASE2
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
This study is looking at a new once-a-day medicine called etavopivat for young people with sickle cell disease. We want to see how safe it is, how long it stays in the body, and if it helps improve their health.
Canada · France · Kenya - authorisedTherapeutic confirmatory (Phase III)
Lidocaine for opioid sparing in vaso-occlusive crisis of Sickle Cell Disease
This study looks at whether a medicine called lidocaine can help reduce the amount of strong pain relief needed for sickle cell pain crises in adults. It compares lidocaine plus usual care to usual care alone.
France - authorisedPhase I and Phase II (Integrated)- Other
A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients with Sickle Cell Disease
This study is testing a new medicine called etavopivat for children and young people with sickle cell disease. It aims to see how safe the medicine is, how it moves through the body, and if it helps improve their health, especially by increasing healthy red blood cells.
France - recruitingPHASE3
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
This study looks at a new medicine called etavopivat for people with sickle cell disease. It aims to see if it reduces painful crises, organ damage, and tiredness, and improves exercise. Participants will get either the medicine or a dummy pill.
United States · Australia · Belgium