Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

NCT04628585 · tracked via the Priya Life Science France tracker

Sponsor

Genetix Biotherapeutics Inc

Phase

Observational

Started

2020-10-21

Last updated

2025-03-20

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

Safety and efficacy assessments

Safety and efficacy assessments: Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Study summary

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. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Eligibility

Sex

ALL

Min age

2 Years

Max age

53 Years

Healthy volunteers

Inclusion Criteria: * Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s) * Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study Exclusion Criteria: * There are no exclusion criteria for this study

Primary outcome measure(s)

Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) — Through 15 years post-drug product infusion
Number of subjects with new or worsening hematologic disorders — Through 15 years post-drug product infusion
Number of subjects with new or worsening neurologic disorders — Through 15 years post-drug product infusion
Number of subjects with malignancies — Through 15 years post-drug product infusion

Trial sites (16)

Facility	City	Region
University of Alabama	Birmingham	Alabama
UCSF Benioff Children's Hospital Oakland	Oakland	California
Children's Healthcare of Atlanta	Atlanta	Georgia
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
Warren Grant Magnuson Clinical Center	Bethesda	Maryland
University of Minnesota Masonic Children's Hospital	Minneapolis	Minnesota
Hackensack University Medical Center	Hackensack	New Jersey
Cohen Children's Medical Center	New Hyde Park	New York
Columbia University Medical Center	New York	New York
The University of North Carolina	Chapel Hill	North Carolina
Duke University Medical Center	Durham	North Carolina
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
Medical University of South Carolina	Charleston	South Carolina
Baylor College of Medicine	Houston	Texas
Virginia Commonwealth University	Richmond	Virginia
Hospital Necker	Paris	France

More Genetix Biotherapeutics Inc. trials in France

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Active, not recruiting

Other trials for the same condition

Comparison of Thrombin Generation, Platelet Activation and Aggregation Profiles in Patients With Sickle Cell Disease Depending on Their SS or SC Genotype (DrepAgreg) Starting soon

Morphine Clearance and Glomerular Filtration in Sickle Cell Patients in Crisis in Intensive Care Recruiting

Inflammation, Platelets and Sickle Cell Disease Active, not recruiting

Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE) Active, not recruiting

Long-term Endurance Training in Sickle Cell Disease Patients: Impact on Clinical Profile, Physical Fitness, and Quality of Life. Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04628585 on ClinicalTrials.gov ↗ ← All trials in France