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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ESCORT-HU Extension: European Sickle Cell Disease Cohort – Hydroxyurea – Extension

As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed.

Locations

61 France site

5 Germany sites

4 French Guiana sites

4 Italy sites

2 Guadeloupe sites

2 Martinique sites

1 Greece site

Age

> 2 Years

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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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A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or Study CTX001-121 (NCT03745287).

Locations

5 United States sites

1 Italy site

1 Germany site

Age

> 18 Years

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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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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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Register Sichelzellkrankheit Der GPOH

Sickle cell disease is one of the most common hereditary diseases.

Locations

1 Germany site

Age

< 100 Years

Genotypes

HbSS, HbSC

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Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

This is a multi-center, long-term safety and efficacy follow-up study for subjects with hemoglobinopathies (β-thalassemia or severe sickle cell disease) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.

Locations

6 United States sites

2 Germany sites

1 Australia site

1 France site

1 Italy site

1 United Kingdom site

1 Thailand site

Age

< 50 Years

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