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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A Phase 1b Sequential Open Label Dose-Ranging Study of Safety, Pharmacokinetics, and Preliminary Activity of Benserazide in Subjects With Beta Thalassemia Intermedia

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.

Locations

2 United States sites

1 Canada site

Age

18 to 65 Years

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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Peer-to-Peer (iP2P) Mentoring Program for Youth With Sickle Cell Disease: A Pilot Feasibility Randomized Controlled Trial

The iPeer2Peer Sickle Cell Disease (SCD) study matches youth (12-18 years of age) with SCD to a mentor (trained young adult) who has learned to manage their SCD well, transitioned to adult care, and can support youth participants emotionally and socially.

Locations

3 Canada sites

1 United States site

Age

12 to 18 Years

Phase

N/A

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Folic Acid Supplementation in Children With Sickle-Cell Disease: A Randomized Double-Blind Cross-Over Trial

Folic acid supplementation (1mg/d) is the standard recommendation for Canadian children with Sickle cell disease (SCD), even though it can provide up to six times the recommended intake amount for healthy children.

Locations

1 Canada site

Age

2 to 19 Years

Phase

N/A

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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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A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects with severe sickle cell disease (SCD).

Locations

8 United States sites

1 Belgium site

1 Canada site

1 Germany site

1 Italy site

1 United Kingdom site

Age

12 to 35 Years

Phase

Phase 1/Phase 2

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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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Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Locations

4 United States sites

2 Canada sites

Age

2 to 25 Years

Genotypes

SS, SC

Phase

Phase 2

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iCanCope With Sickle Cell Disease: A Mobile Pain Management Intervention for Adolescents

The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease.

Locations

3 United States sites

1 Canada site

Age

12 to 18 Years

Phase

N/A

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A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-MAID)

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor.

Locations

1 Canada site

Age

1 to 19 Years

Genotypes

HbSS, HbSC

Phase

Phase 2

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