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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Preliminary Feasibility and Efficacy of The Balance Program to Reduce the Impact of Pain on Daily Functioning in Pediatric Sickle Cell Disease

Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain.

Locations

1 United States site

Age

8 to 17 Years

Genotypes

HbSS, HbSC

Phase

N/A

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An Open-Label Extension Study of Voxelotor Administered Orally to Pediatric Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Open-label extension study of voxelotor for pediatric participants ages 4 to 18 years old with Sickle Cell Disease who have participated in voxelotor clinical trials.

Locations

7 United States sites

3 United Kingdom sites

2 Lebanon sites

Age

4 to 18 Years

Phase

Phase 3

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Near-infrared Spectroscopy in Sickle Cell Pediatric Patients During Pain Crisis and After Recovery

Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease.

Locations

1 United States site

Age

6 to 21 Years

Genotypes

SS, SC

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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 Pilot Study of Acupuncture for the Treatment of Pain in Sickle Cell Disease

Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry.

Locations

1 United States site

Age

8 to 22 Years

Genotypes

HbSS, HbSC

Phase

N/A

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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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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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