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 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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Randomized Trial Evaluating Lidocaine Intravenous in the Emergency Department For Sickle Cell Crisis – RELIEF-SCC

Sickle cell crisis continues to be a frequent presentation to emergency departments.

Locations

3 United States sites

Age

> 18 Years

Phase

Phase 2

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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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An Open-Label, Expanded Access Protocol for Patient 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 voxelotor prior to market authorization .

Locations

18 United States sites

Age

> 12 Years

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An Open Label Extension Study of Voxelotor (GBT440) Administered Orally to Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials .

Locations

24 United States sites

6 United Kingdom sites

4 Egypt sites

3 Kenya sites

3 Turkey sites

2 Lebanon sites

2 Netherlands sites

1 Canada site

1 France site

1 Oman site

1 Italy site

Age

> 12 Years

Phase

Phase 3

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A Phase 2,Multicenter,Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab,With or Without Hydroxyurea/Hydroxycarbamide,in Sequential,Descending Age Groups of Pediatric Sickle Cell Disease Patients With Vaso-Occlusive Crisis

The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years.

Locations

19 United States sites

5 Spain sites

4 Belgium sites

3 Brazil sites

3 Colombia sites

3 India sites

3 Italy sites

2 Canada sites

2 Germany sites

2 Lebanon sites

2 Turkey sites

2 United Kingdom sites

1 Oman site

1 Switzerland site

1 France site

Age

6 to 17 Years

Genotypes

HbSS, HbSC

Phase

Phase 2

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Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients With Symptomatic Sickle Cell Disease

This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD).

Locations

28 United States sites

Age

5 to 45 Years

Genotypes

SS

Phase

Phase 2

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A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Exploratory Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

This study consists of four parts, Parts A, B, C, and D.

Locations

11 United States sites

4 United Kingdom sites

2 Lebanon sites

Age

9 to 17 Years

Genotypes

HbSS

Phase

Phase 2

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A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults With Severe Sickle Cell Disease (BMT CTN #1503)

This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care.

Locations

35 United States sites

Age

15 to 40 Years

Genotypes

SS, SC

Phase

Phase 2

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1503 BMT CTN STRIDE Biorepository

The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)".

Locations

40 United States sites

Age

15 to 40 Years

Genotypes

SS, SC

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