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A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2023-06-18

Study results available
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Summary

This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (\<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

Conditions

Interventions

GENETIC

LentiGlobin BB305 Drug Product

LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Himal Lal Thakar, MD · Genetix Biotherapeutics Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-08-08
Primary Completion
2022-03-31
Completion
2022-03-31

Countries

  • United States
  • France
  • Germany
  • Italy
  • Thailand
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02906202 on ClinicalTrials.gov