- enrolling by invitation
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
This study is checking on people with sickle cell disease or a type of thalassaemia called transfusion-dependent beta-thalassaemia. They all previously received a special treatment called EDIT-301. We want to see how safe and effective this treatment has been for them over a long time.
United States · Canada - enrolling by invitation
SCRIPT: Sickle Cell Risk in Pregnancy Tool
This study looks at a new tool to predict pregnancy risks for women with Sickle Cell Disease (SCD). It aims to identify who might benefit most from extra care, helping doctors give the right support while avoiding unnecessary treatments for others.
United States · Canada - 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, 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 - 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 - 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 - 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 - 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 - 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 - 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 - 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 - recruitingPHASE2
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
This study is testing a new medicine, Anumigilimab, for adults with sickle cell disease. Researchers want to see if it's safe. Participants will receive the medicine or a dummy medicine for over a year to check for side effects and how well it's tolerated. It's a key step in understanding this potential new treatment.
United States · Belgium · 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 - 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 - 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 - 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 - 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 - 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 - 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)
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 - recruitingNA
High-flow Oxygen for Vaso-occlusive Pain Crisis
This study looks at whether a special way of giving oxygen, called high-flow nasal oxygen (HFNO), can help people with sickle cell disease who are having a pain crisis. We want to see if it reduces pain and stops serious lung problems.
France - recruiting
Lung Ultrasound in Pediatric Acute Chest Syndrome
This study looks at how lung ultrasound can help children with sickle cell disease who develop a serious lung problem called Acute Chest Syndrome (ACS). Researchers want to see if ultrasound scans can predict how long children might need help with breathing and how serious their condition is.
France - 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 - recruiting
Sickle-cell Disease Registry of the GPOH
This study is gathering information on people with sickle cell disease in Germany, Austria, and Switzerland. It aims to understand how the disease affects people, how it's treated, and improve care, especially for newborns. This will help doctors better manage the condition.
Germany - recruitingPHASE2
A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
This study is testing a new type of stem cell transplant for people with sickle cell disease. It aims to see if this new transplant, using cells from a partially matched family member, is as safe and effective as the current best transplant option.
Austria · Germany - 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 - recruitingPHASE2
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
This study looks into a new way of doing stem cell transplants for children aged 1 to 19 with sickle cell disease. It aims to make this life-changing treatment available to more children, especially if their donor's blood type doesn't perfectly match theirs.
Canada - 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 - active not recruitingPHASE2
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
This study is for children under 13 with sickle cell disease who have a healthy sibling donor. It looks at a special type of bone marrow transplant to see if it's safe and effective, aiming to improve long-term health and quality of life for these children.
United States · Canada - 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 - 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 - recruiting
SCORE - Sickle cell outreach, resources & engagement
This study aims to design local support centres, called 'sickle hubs', for people with Sickle Cell Disease in Liverpool. It will gather ideas from patients, carers, and experts to create better, joined-up care and support for this condition.
United Kingdom · England - recruitingNA
Adapting the FACETS Program to Sickle Cell Disease
This study is adapting a program called FACETS to help adults with sickle cell disease manage their tiredness (fatigue). The program combines talking therapies and ways to save energy, aiming to improve how they feel day-to-day.
France