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Gene Therapy for Wiskott-Aldrich Syndrome

NCT01515462 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2025-04-04

Study results available
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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.

Conditions

  • Wiskott-Aldrich Syndrome (WAS)

Interventions

GENETIC

TLT003

TLT003 is an autologous CD34+ cells collected from bone marrow and/or peripheral blood and transduced with a lentiviral vector encoding Wiskott-Aldrich syndrome (WAS) protein

Sponsors & Collaborators

  • Ospedale San Raffaele

    collaborator OTHER

  • Fondazione Telethon

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-04-20
Primary Completion
2023-10-04
Completion
2023-10-04

Countries

  • Italy

Study Locations

More Related Trials

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 NCT01515462 on ClinicalTrials.gov