A Study Evaluating the Safety & Efficacy of LentiGlobin BB305 Drug Product

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

Sickle Cell Disease Clinical Trial

Official title:
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral β-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)

See also
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Recruiting	`NCT03937817` - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
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Completed	`NCT04134299` - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease	N/A
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Recruiting	`NCT04754711` - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition	N/A
Completed	`NCT04388241` - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD	N/A
Recruiting	`NCT05431088` - A Phase 2/3 Study in Adult and Pediatric Participants With SCD	Phase 2/Phase 3
Completed	`NCT01158794` - Genes Influencing Iron Overload State
Recruiting	`NCT03027258` - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome	N/A
Withdrawn	`NCT02960503` - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease	Phase 1/Phase 2
Withdrawn	`NCT02630394` - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease	Phase 1
Completed	`NCT02565082` - Evaluation of the Hemostatic Potential in Sickle Cell Disease Patients	N/A
Completed	`NCT02567695` - A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects	Phase 1

NCT number	NCT02151526
Study type	Interventional
Source	bluebird bio
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	June 7, 2013
Completion date	February 26, 2019

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms