Status:

ACTIVE_NOT_RECRUITING

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

Lead Sponsor:

IRCCS San Raffaele

Collaborating Sponsors:

Telethon Institute for Gene Therapy (OSR-TIGET)

Conditions:

Beta Thalassaemia

Eligibility:

All Genders

3+ years

Phase:

NA

Brief 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 t...

Eligibility Criteria

Inclusion

  • Subjects who have completed study TIGET-BTHAL i.e. who have received treatment and been followed for two years post treatment with OTL-300.
  • For adults; capable of giving signed informed consent. For children; informed assent and/or consent in writing signed by the subject and/or parent(s) / legal representative (according to local regulations and age of the subject).

Exclusion

  • None

Key Trial Info

Start Date :

October 4 2017

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 1 2026

Estimated Enrollment :

9 Patients enrolled

Trial Details

Trial ID

NCT03275051

Start Date

October 4 2017

End Date

June 1 2026

Last Update

November 23 2022

Active Locations (1)

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Page 1 of 1 (1 locations)

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Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

Milan, Italy, 20132