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A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

NCT01745120 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2019-05-08

Study results available
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Summary

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product \[autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene\].

Conditions

  • β-thalassemia Major

Interventions

GENETIC

LentiGlobin BB305 Drug Product

Transplant of autologous hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector.

Sponsors & Collaborators

  • Genetix Biotherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Mohammed Asmal, MD · Genetix Biotherapeutics Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-08-31
Primary Completion
2018-02-08
Completion
2018-02-21

Countries

  • United States
  • Australia
  • Thailand

Study Locations

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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 NCT01745120 on ClinicalTrials.gov