CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 8 enrolled
Drug / intervention
TLT003genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01515462
NCT01515462Phase 2Completed
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A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

Fondazione Telethon·interventional·Posted Jan 24, 2012·Updated Apr 4, 2025

In Brief

A Phase 2 clinical trial evaluating TLT003 for Wiskott-Aldrich Syndrome (WAS). Completed, enrolled 8 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsWiskott-Aldrich Syndrome (WAS)
CountriesItaly
CollaboratorsOspedale San Raffaele

Timeline

Phase 2CompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedJan 24, 2012
Enrollment StartApr 20, 2010
Primary CompletionOct 4, 2023
TodayJul 2, 2026
Enrollment to primary: 13.5 yearsPosted 14.4 years ago

Interventions

TLT003genetic

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