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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Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD).

Locations

2 United States sites

Age

12 to 35 Years

Phase

Phase 1/Phase 2

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A Phase I/II Study of the Correction of a Single Nucleotide Mutation (Adenosine –> Thymine [A–>T]) in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease

This study is a first-in-human, single-arm, open-label Phase I/II study of GPH101 in approximately 15 participants, diagnosed with severe Sickle Cell Disease.

Locations

1 United States site

Age

12 to 40 Years

Phase

Phase 1/Phase 2

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An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).

Locations

9 United States sites

Age

12 to 65 Years

Phase

Phase 2/Phase 3

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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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Improving SCD Care Using Web-based Guidelines, Nurse Care Managers and Peer Mentors in Primary Care and Emergency Departments in Central North Carolina

The overall purpose of this proposed study is to improve management of vaso-occlusive episodes (VOEs) in adult EDs.

Locations

8 United States sites

Age

18 to 45 Years

Genotypes

SS, SC

Phase

N/A

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A Phase I Multicenter Study of Hematopoietic Stem Cell Transplant of ECT-001-Expanded Cord Blood With a Reduced Toxicity Conditioning Regimen in Patients With Severe Sickle Cell Disease

The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment, particularly in pediatric populations.

Locations

2 United States sites

Age

5 to 30 Years

Phase

Phase 1

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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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Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization- mESH Study

This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU).

Locations

8 United States sites

Age

15 to 45 Years

Genotypes

SS, SC

Phase

N/A

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