Phase 2
Completed N=8
Gene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Source: ClinicalTrials.gov NCT01515462 ↗
Enrolled (actual)
8
Serious AEs
100.0%
Results posted
Apr 2025
Primary outcomePrimary: Safety of Reduced Conditioning Regimen — 8 Participants
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.
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 |
— | — |
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.
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. Informational only — not medical advice.