A Single Arm, Open Label Clinical Study of Hematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS)

Fondazione Telethon Ets, Fondazione Telethon Ets1 site in 1 country9 target enrollmentStarted: October 28, 2024Last updated: December 11, 2024

Trial Snapshot

Phase: Phase 3
Status: Recruiting
Sponsor
Enrollment: 9
Locations: 1
Primary Endpoint: Evaluation of the clinical efficacy: • annualized rate of severe infections from 6 to 18 months after gene therapy (GT) compared with 1 year prior to GT; • annualized rate of moderate and severe bleeding episodes up to 1 year after GT compared with 1 year prior to GT.

Study Overview Eligibility Criteria Outcomes Investigators Sites Records & Identifiers Similar Trials

Study Overview

Brief Summary

• To evaluate the clinical efficacy of the cryopreserved formulation of OTL-103 at 12 months for bleeding events and from 6 to 18 months for severe infections

Eligibility Criteria

Ages: 0 years to 64 years (0-17 Years, 18-64 Years)
Sex: Male
Accepts Healthy Volunteers: No

Inclusion Criteria

•Diagnosis of WAS defined by genetic mutation and at least one of the following criteria: severe WASP mutation; absent WASP expression; severe clinical score (Zhu clinical score ≥ 3); • No human leukocyte antigen (HLA)-identical related donor available.

Exclusion Criteria

•End-organ dysfunction, severe active infection not responsive to treatment, or other severe disease or clinical condition which, in the judgment of the investigator, would make the subject inappropriate for entry into this study. • Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome (detected via medical history check during screening). Subjects with a prior successfully treated malignancy and a sufficient follow-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the Medical Monitor. • Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukemia, or other serious hematological disorders. • Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin. • Previous gene therapy. • Documented human immunodeficiency virus (HIV) infection (positive HIV ribonucleic acid and/or anti-p24 antibodies).

Outcomes

Primary Outcomes

Evaluation of the clinical efficacy: • annualized rate of severe infections from 6 to 18 months after gene therapy (GT) compared with 1 year prior to GT; • annualized rate of moderate and severe bleeding episodes up to 1 year after GT compared with 1 year prior to GT.

Secondary Outcomes

• Evaluation of the overall survival at 12, 24 and 36 months • Evaluation of the safety of treatment: 1) safety and tolerability as measured by adverse event (AE) reporting; 2) absence of malignancy or abnormal clonal proliferation (ACP) development due to insertional oncogenesis; 3) absence of replication-competent lentivirus (RCL). • Evaluation of the engraftment at 6 months • Evaluation of biological correlates of efficacy at 12 months, 2 years and 3 years

Investigators

Sponsor

Fondazione Telethon Ets, Fondazione Telethon Ets

Sponsor Class

Patient organisation/association, Patient organisation/association

Responsible Party

Principal Investigator

Elena Tomasetto

Scientific

Fondazione Telethon Ets

Study Sites (1)

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