HAEMATOPOIETIC STEM CELL GENE THERAPY

Phase 1

Active, not recruiting

• Conditions: Wiskott-Aldrich Syndrom; MedDRA version: 20.0Level: PTClassification code 10061598Term: ImmunodeficiencySystem Organ Class: 10021428 - Immune system disorders; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2009-017346-32-IT
• Lead Sponsor: Fondazione Telethon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-04-26
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: Male
• Target Recruitment: 8

Inclusion Criteria

1) Diagnosis of WAS defined by genetic mutation and at least one of the following criteria: -severe WAS mutation -absent WASP expression -severe clinical score (Zhu clinical score >= 3); 2) No HLA-identical sibling donor; and 3.1) Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months; or 3.2) Patients of > 5 years of age who are not candidate to unrelated allogeneic transplant based on clinical conditions and 4) Parental/guardian/patient signed informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from study admission: 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified