Clinical trial • Phase III • Immunology|Rare Disease

Etuvetidigene autotemcel for Wiskott-Aldrich syndrome

Phase III trial of Etuvetidigene autotemcel for Wiskott-Aldrich syndrome. open-label, none/not specified-controlled. 9 participants.

Overview

Trial Therapeutic Area: Immunology|Rare Disease
Trial Disease: Wiskott-Aldrich syndrome
Trial Stage: Phase III
Drug Modality: Cell therapy|Monoclonal antibody|Small molecule|Peptide/protein/enzyme
Paediatric Trial: Yes
Orphan Drug: Yes

open-label, none/not specified-controlled Phase III trial across 1 site in Italy.

Recruits 9 paediatric patients.

Vulnerable Population: Includes minors. Study documents include a parent informed consent form (Parent ICF Main Study), an assent form for ages 12-17 (Assent 12-17 ICF), and an adult ICF. Consent from parent/guardian is required for minors; assent is obtained from adolescents (12-17). Materials available in Italian, English and Spanish (information sheets / ICFs listed).

{"criterion_text":"- 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);\n- No human leukocyte antigen (HLA)-identical related donor available."}

{"criterion_text":"- 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.\n- 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.\n- Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukemia, or other serious hematological disorders.\n- Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin.\n- Previous gene therapy.\n- Documented human immunodeficiency virus (HIV) infection (positive HIV ribonucleic acid and/or anti-p24 antibodies)."}

{"endpoint_text":"- annualized rate of severe infections from 6 to 18 months after gene therapy (GT) compared with 1 year prior to GT\n- annualized rate of moderate and severe bleeding episodes up to 1 year after GT compared with 1 year prior to GT","definition_or_measurement_approach":"Annualized rates compared to the 1-year period prior to gene therapy; severe infections measured from 6 to 18 months after GT; bleeding episodes measured up to 1 year after GT."}

{"endpoint_text":"- Evaluation of the overall survival at 12, 24 and 36 months\n- 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).\n- Evaluation of the engraftment at 6 months\n- Evaluation of biological correlates of efficacy at 12 months, 2 years and 3 years","definition_or_measurement_approach":"Overall survival assessed at 12, 24 and 36 months; safety measured by AE reporting and specific assessments for malignancy/ACP and RCL; engraftment evaluated at 6 months; biological correlates assessed at specified timepoints (12 months, 2 and 3 years)."}

Planned Sample Size: 9
Recruitment Window Months: 107
Consent Approach: Informed consent obtained from adult participants; for minors parental/guardian informed consent is required and an assent form is used for adolescents aged 12-17. Study documentation listed in CTIS includes Parent ICF, Assent 12-17 ICF, Adult ICF and information sheets in Italian, English and Spanish.

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