Status:
ACTIVE_NOT_RECRUITING
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
Lead Sponsor:
Genethon
Conditions:
X-Linked Chronic Granulomatous Disease
Eligibility:
MALE
6+ years
Phase:
PHASE1
PHASE2
Brief Summary
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is caused by an error in a gene that makes part of the immune system. The basic defect lies in special...
Eligibility Criteria
Inclusion
- Male X-CGD patients
- Molecular diagnosis confirmed by DNA sequencing
- At least one prior ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
- No HLA-matched donor available after 3 months search unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable by the investigator
Exclusion
- Contraindication for leukapheresis
- Contraindication for administration of conditioning medication
- Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
Key Trial Info
Start Date :
June 24 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2032
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT01855685
Start Date
June 24 2013
End Date
September 1 2032
Last Update
April 6 2023
Active Locations (2)
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1
University College London Hospital (UCLH)
London, United Kingdom, NW1 2PG
2
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom