Clinical Trial Finder

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 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

Locations

16 United States sites

3 Canada sites

Age

18 Years - 50 Years

Phase

Phase 1/Phase 2

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

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat 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

49 United States sites

5 France sites

5 Spain sites

4 Greece sites

4 Italy sites

4 United Kingdom sites

3 Canada sites

2 Lebanon sites

2 Germany sites

Age

12 Years - 65 Years

Phase

Phase 2/Phase 3

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Desmopressin as a Therapy for Nocturnal Enuresis in Pediatric Patients With Sickle Cell Disease

This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.

Locations

1 United States site

Age

8 Years - 21 Years

Genotypes

Hemoglobin SS

Phase

Phase 4

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A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Subjects With Sickle Cell Disease

The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of dosing, and to identify a range of doses that are safe and pharmacologically active in participants with sickle cell disease.

Locations

8 United States sites

2 Spain sites

1 Denmark site

Age

18 Years - 70 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

4 United States sites

1 Canada site

Age

> 18 Years

Phase

Phase 1/Phase 2

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A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

This study is evaluating a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Locations

4 United States sites

1 Italy site

Age

2 Years - 40 Years

Genotypes

HbSS, HbSC

Phase

Phase 1/Phase 2

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Sickle Cell Disease and CardiovAscular Risk – Red Cell Exchange Trial (SCD-CARRE)

The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces the total number of episodes of clinical worsening of SCD requiring acute health care encounters (non-elective infusion center/ER/hospital visits) or resulting in death over 12 months as compared with standard of care.

Locations

18 United States sites

1 United Kingdom site

1 France site

Age

> 18 Years

Phase

Phase 3

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Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Advocacy and Research Alliance (STAR) Trial

This study aims to enroll 58 pre-adolescent (<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor.

Locations

11 United States sites

Age

2 Years - 13 Years

Genotypes

HbSS

Phase

Phase 2

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The Use Of Intranasal (IN) Ketamine For Pain Control In Patients With Sickle Cell Disease And Vaso-occlusive Episode (VOE) In The Pediatric Emergency Department

This will be a descriptive cohort study of intranasal ketamine as the initial analgesic for children with sickle cell disease who present to the pediatric emergency department with vaso-occlusive crisis and are awaiting intravenous line placement.

Locations

2 United States sites

Age

3 Years - 25 Years

Genotypes

Hemoglobin SS

Phase

Phase 3

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Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant (ASCENT)

The ASCENT Trial is a single arm, multi-center, phase II study.

Locations

9 United States sites

Age

< 20 Years

Phase

Phase 2

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