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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Stroke Prevention in Young Adults With Sickle Cell Anemia

Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide.

Locations

1 United States site

1 Nigeria site

Age

16 to 26 Years

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Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease.

Locations

1 United States site

Age

10 to 25 Years

Phase

Phase 2

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Stroke Registry for Children and Young Adults With Sickle Cell Disease in Nigeria – The Afolabi Stroke Registry

Sickle Cell Anemia (SCA) occurs in 300,000 newborns per year in the world, with 150,000 affected births in Nigeria, alone.

Locations

2 Nigeria sites

1 United States site

Age

5 to 26 Years

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Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia

The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients .

Locations

4 United States sites

Age

5 to 50 Years

Genotypes

HbSS

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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 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 will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Locations

3 United States sites

Age

2 to 40 Years

Genotypes

HbSS, HbSC

Phase

Phase 1/Phase 2

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Reduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation for Patients With Severe Sickle Cell Disease

This study is being done to test a transplant method that may have fewer side effects (or less toxic, less harmful) than conventional high dose chemotherapy conditioning-based transplants for children and young adults with Sickle Cell Disease (SCD).

Locations

1 United States site

Age

2 to 25 Years

Phase

Phase 2

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Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers and to use surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD.

Locations

1 United States site

Age

> 13 Years

Genotypes

HbSS, HbSC

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An Open-Label Extension Study of Voxelotor Administered Orally to Pediatric Participants With Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials

Open-label extension study of voxelotor for pediatric participants ages 4 to 18 years old with Sickle Cell Disease who have participated in voxelotor clinical trials.

Locations

7 United States sites

3 United Kingdom sites

2 Lebanon sites

Age

4 to 18 Years

Phase

Phase 3

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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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