This study evaluates the safety and efficacy of transferring the WAS gene into hematopoietic stem/progenitor cells to treat Wiskott-Aldrich Syndrome. The product is called TLT003 (previously GSK2696275, previously OTL-103). The Phase 1/2 trial enrolled patients with a genetically confirmed diagnosis who also had a severe WAS mutation, absent WASP expression or a severe clinical score (Zhu score of 3 or higher), and who had no HLA-identical sibling donor and no matched unrelated donor.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. 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 2. No HLA-identical sibling donor 3. 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. 4. Parental/guardian/patient signed informed consent. Exclusion Criteria: 1. Patients positive for HIV-infection. 2. Patients affected by neoplasia. 3. Patients with cytogenetic alterations typical of MDS/AML. 4. 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. 5. Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months. 6. Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.