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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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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Kinetics of Donor Red Blood Cell Survival in Sickle Cell Disease

This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT).

Locations

3 United States sites

Age

> 6 Years

Genotypes

HbSS

Phase

Phase 1

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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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Building Adaptive Coping and Knowledge to Improve Daily Life (Back2Life): A Pilot Feasibility Clinical Trial for Youth With Chronic Sickle Cell Pain

The purpose of this study is to find out how teenagers with chronic pain and sickle cell disease respond to a new training program called Back2Life and get their feedback about how to modify the program to best fit their needs.

Locations

3 United States sites

Age

10 to 18 Years

Phase

N/A

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A Phase 4, Multicenter, Open-label Study to Evaluate the Treatment Effect of Voxelotor on Physical Activity in Adolescents and Adults With Sickle Cell Disease

This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.

Locations

4 United States sites

Age

12 to 55 Years

Phase

Phase 4

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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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Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) Part 3

The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world implementation of stroke prevention guidelines (STOP Protocol) in which transcranial Doppler (TCD), a measure of cerebral blood vessel velocity, is used to screen for stroke risk in children ages 2-16 with sickle cell anemia (SCA).

Locations

15 United States sites

Age

2 to 7 Years

Phase

N/A

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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease.

Locations

16 United States sites

5 Nigeria sites

4 Egypt sites

3 Italy sites

3 Kenya sites

3 United Kingdom sites

2 Oman sites

2 Ghana sites

2 Saudi Arabia sites

Age

2 to 14 Years

Phase

Phase 3

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