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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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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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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Long-term Follow-up of Subjects With Sickle Cell Disease 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 sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.

Locations

2 United States sites

1 France site

Age

2 to 53 Years

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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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SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.

Locations

1 United States site

Age

> 11 Years

Phase

N/A

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A Phase 1/2 Randomized, Double-blind, Placebo-controlled, Multicenter, Ascending Dose, Safety and PK/PD Study of SHP655 (rADAMTS13) in Sickle Cell Disease at Baseline Health and During Acute Vaso-Occlusive Crisis

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of SHP655 in participants with baseline health sickle cell disease (SCD) and SCD with acute vaso-occlusive crisis (VOC).

Locations

14 United States sites

Age

18 to 65 Years

Genotypes

HbSS

Phase

Phase 1/Phase 2

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

This study aims to enroll 58 pre-adolescent (<10 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor.

Locations

11 United States sites

Age

2 to 10 Years

Phase

Phase 2

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LCI-HEM-SCD-ST3P-UP-001: A Comparative Effectiveness Study of Peer Mentoring [PM] Versus Structured Transition Education Based Intervention [STE] for the Management of Care Transitions in Emerging Adults With Sickle Cell Disease (SCD)

This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on decreasing the number of acute care visits per year, improving patient-reported outcomes, and reducing healthcare utilization among emerging adults with sickle cell disease (EA-SCD) .

Locations

14 United States sites

Age

> 16 Years

Phase

N/A

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