Phase 2 N=8 Treatment

Gene Therapy for Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome (WAS)

Bottom Line

View on ClinicalTrials.gov: NCT01515462 ↗

Enrolled (actual)

Serious AEs

100.0%

Results posted

Apr 2025

Primary outcome: Primary: Safety of Reduced Conditioning Regimen — 8 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: TLT003 (Genetic)
Age: Pediatric, Adult, Older Adult
Sex: All
Sponsor: Fondazione Telethon
Primary completion: Oct 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Safety of Reduced Conditioning Regimen	8	—
PRIMARY Safety of Lentivirus Gene Transfer Into HSC	8	—
PRIMARY Sustained Engraftment of Genetically Corrected Haematopoietic Stem Cells in Peripheral Blood and/or in Bone Marrow	8	—
PRIMARY Presence of Detectable Vector-derived WASP	8	—
PRIMARY Improved T-cell Functions	8	—
PRIMARY Antigen-specific Responses to Vaccination	7	—
PRIMARY Improved Platelet Count and MPV Normalization	8	—
PRIMARY Overall Survival	8	—
SECONDARY Lack of Immune Response to Transgene	8	—
SECONDARY Reduced Frequency of Severe Infections	—	—
SECONDARY Reduced Bruising and Bleeding Episodes	—	—
SECONDARY Reduced Autoimmunity Phenomena and Eczema	—	—
SECONDARY Improved Quality of Life	—	—
SECONDARY Multilineage Engraftment of Genetically Corrected Cells	—	—
SECONDARY Overall Safety of the Treatment	—	—

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.

Eligibility Criteria

Inclusion Criteria

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 Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01515462). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.