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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Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial

The trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) is designed to further knowledge on efficacy and safety of the therapy.

Locations

10 United States sites

Age

3 to 21 Years

Phase

Phase 3

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An Open-label, Expanded Access Protocol for Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

The intent of this open-label, multicenter Expanded Access Program (EAP) is to provide early access to treatment with voxelotor prior to market authorization for pediatric patients age 4 to 11 years with sickle cell disease (SCD) who have no alternative treatment options and are ineligible to participate in clinical trials of voxelotor.

Locations

12 United States sites

Age

4 to 11 Years

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Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies).

Locations

1 United States site

Age

2 to 30 Years

Phase

Early Phase 1

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Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease

This study seeks to utilize an innovative approach of a single session problem-solving intervention to address psychosocial factors affecting patient outcomes within the pediatric sickle cell population.

Locations

1 United States site

Age

> 7 Years

Genotypes

SS

Phase

N/A

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A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects With Sickle Cell Disease

A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease .

Locations

10 United States sites

4 Tunisia sites

3 Lebanon sites

1 Greece site

1 Italy site

1 Netherlands site

1 Oman site

Age

18 to 65 Years

Genotypes

HbSS

Phase

Phase 2

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A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Subjects With Sickle Cell Disease

The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease.

Locations

1 United States site

Age

18 to 70 Years

Phase

Phase 1

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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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A Comparison of Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease

The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment.

Locations

6 United States sites

Age

> 18 Years

Genotypes

SS, SC

Phase

Phase 3

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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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Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Alliance for Research (STAR) Trial

This study aims to enroll 58 pre-adolescent (<10 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor.

Locations

11 United States sites

Age

2 to 10 Years

Phase

Phase 2

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