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Gene Therapy for Transfusion Dependent Beta-thalassemia

NCT02453477 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2019-06-28

No results posted yet for this study

Summary

This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia

Conditions

Interventions

GENETIC

Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene

Autologous CD34+ cell enriched fraction containing hematopoietic stem cells (HSC) transduced with the GLOBE lentiviral vector encoding for the human beta-globin gene resuspended in their final formulation medium. The target dose in the transduced product is 5x10\^6 cells/Kg CD34+ cells, with a minimum dose of 2 x 10\^6/Kg and a maximum dose of 20 x 10\^6/Kg, depending on the yield of cells. The product will be injected intraosseously.

Sponsors & Collaborators

  • Fondazione Telethon

    collaborator OTHER

  • Orchard Therapeutics

    collaborator INDUSTRY

  • IRCCS San Raffaele

    lead OTHER

Principal Investigators

  • Alessandro Aiuti, MD, PhD · Ospedale San Raffaele

  • Fabio Ciceri, MD · Ospedale San Raffaele

  • Sarah Marktel, MD · Ospedale San Raffaele

  • Maria Domenica Cappellini, MD · IRCCS Policlinico Foundation

  • Giuliana Ferrari, PhD · Telethon Institute of Gene Therapy, Ospedale San Raffaele

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Max Age
64 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-31
Primary Completion
2019-08-31
Completion
2019-08-31

Countries

  • Italy

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 NCT02453477 on ClinicalTrials.gov