Clinical Trials

Hundreds of clinical trials are rolled out around the world monthly, and many of them are designed to specifically uncover and manage the medical needs of people with sickle cell disease and trait. We keep an updated list of these global studies here, so you don’t have to go searching for them. There might be active study recruitment and enrollment happening at a site near you. Explore the list below to see the different types of studies, and use the navigation options on the left to get as specific as you would like.

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An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).

Locations

9 United States sites

Age

12 to 65 Years

Phase

Phase 2/Phase 3

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An Open-label Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia) Who Participated in Study IMR-SCD-102

This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102).

Locations

4 United Kingdom sites

3 United States sites

Age

> 18 Years

Phase

Phase 2

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Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) Part 3

The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world implementation of stroke prevention guidelines (STOP Protocol) in which transcranial Doppler (TCD), a measure of cerebral blood vessel velocity, is used to screen for stroke risk in children ages 2-16 with sickle cell anemia (SCA).

Locations

15 United States sites

Age

2 to 7 Years

Phase

N/A

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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.

Locations

16 United States sites

5 Nigeria sites

4 Egypt sites

3 Italy sites

3 Kenya sites

3 United Kingdom sites

2 Oman sites

2 Ghana sites

2 Saudi Arabia sites

Age

2 to 14 Years

Phase

Phase 3

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Outcomes Mandate National Integration With Cannabis as Medicine for Prevention and Treatment of COVID-19

This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.

Locations

17 United States sites

Age

> 7 Years

Phase

Phase 2

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A Randomized, Double-Blind, Placebo-Controlled Pilot Study of the Safety and Efficacy of Deferoxamine Intradermal Delivery Patch (DIDP) in Chronic Sickle Cell Leg Ulcers

Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).

Locations

4 United States sites

Age

> 18 Years

Genotypes

SC

Phase

Phase 1/Phase 2

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A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.

Locations

9 Brazil sites

9 United States sites

6 United Kingdom sites

5 Italy sites

5 Belgium sites

5 Germany sites

4 Spain sites

3 Netherlands sites

3 India sites

3 Greece sites

3 Colombia sites

3 France sites

2 Turkey sites

2 Canada sites

2 Panama sites

2 Lebanon sites

1 South Africa site

1 Jordan site

1 Ghana site

1 Finland site

1 Oman site

Age

> 12 Years

Phase

Phase 3

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Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation (BMT CTN 1702)

The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.

Locations

43 United States sites

Phase

N/A

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A Randomized, Placebo-controlled, Double Blind, Single Ascending and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of FT-4202 in Healthy Volunteers and Sickle Cell Disease Patients

FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias.

Locations

19 United States sites

Age

12 to 65 Years

Phase

Phase 1

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PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.

Locations

1 United States site

Age

3 to 39 Years

Phase

Phase 1/Phase 2

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