Status:
COMPLETED
Gene Therapy for Wiskott-Aldrich Syndrome
Lead Sponsor:
Fondazione Telethon
Collaborating Sponsors:
Ospedale San Raffaele
Conditions:
Wiskott-Aldrich Syndrome (WAS)
Eligibility:
All Genders
Phase:
PHASE1
PHASE2
Brief Summary
This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.
Detailed Description
Wiskott-Aldrich Syndrome (WAS) is an X-linked primary immunodeficiency caused by mutations in the WAS gene which encodes the WAS protein (WASP), a cytoskeletal regulator which is expressed exclusively...
Eligibility Criteria
Inclusion
- Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
- Severe WAS mutation
- Absence of WASP expression
- Severe clinical score (Zhu clinical score ≥ 3
- No HLA-identical sibling donor
- Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months
- Patients of \> 5 years of age who are not candidate to unrelated allogeneic transplant based on clinical conditions.
- Parental/guardian/patient signed informed consent.
Exclusion
- Patients positive for HIV-infection.
- Patients affected by neoplasia.
- Patients with cytogenetic alterations typical of MDS/AML.
- Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study.
- Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months.
- Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.
Key Trial Info
Start Date :
April 20 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 4 2023
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT01515462
Start Date
April 20 2010
End Date
October 4 2023
Last Update
April 4 2025
Active Locations (1)
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1
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, Italy, 20132