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Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

NCT03275051 · Status: ACTIVE_NOT_RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2022-11-23

No results posted yet for this study

Summary

OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).

Conditions

  • Beta Thalassaemia

Interventions

OTHER

Safety and Efficacy assessments

Safety and efficacy assessment of OTL-300 in subjects with transfusion dependent beta-thalassemia will be performed.

Sponsors & Collaborators

  • Telethon Institute for Gene Therapy (OSR-TIGET)

    collaborator UNKNOWN

  • IRCCS San Raffaele

    lead OTHER

Principal Investigators

  • Orchard Clinical Trials · Ospedale San Raffaele- Telething Institute for Gene Therapy (SR-TIGET)

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-10-04
Primary Completion
2025-12-01
Completion
2026-06-01

Countries

  • Italy

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 NCT03275051 on ClinicalTrials.gov