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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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 to 21 Years

Genotypes

SS, SC

Phase

Phase 4

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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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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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A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.

Locations

9 Brazil sites

9 United States sites

6 United Kingdom sites

5 Italy sites

5 Belgium sites

5 Germany sites

4 Spain sites

3 Netherlands sites

3 India sites

3 Greece sites

3 Colombia sites

3 France sites

2 Turkey sites

2 Canada sites

2 Panama sites

2 Lebanon sites

1 South Africa site

1 Jordan site

1 Ghana site

1 Finland site

1 Oman site

Age

> 12 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 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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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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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

15 United States sites

Age

> 18 Years

Phase

Phase 3

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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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