Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
Launched by GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST · Aug 3, 2010
Trial Information
Current as of June 14, 2025
Unknown status
Keywords
ClinConnect Summary
No description provided
Gender
MALE
Eligibility criteria
- Inclusion Criteria:
- • 1. No HLA identical (A,B,C,DR,DQ) family donor and no HLA identical unrelated donor available within 3 months of diagnosis or patients whose underlying clinical problems and prognosis would be significantly compromised by chemotherapy conditioning (including persisting pneumonitis, protracted diarrhoea requiring parental nutrition, ongoing visceral viral infection (herpes viruses, HSV,VZV,CMV, EBV or adenovirus), systemic BCG infection, virus-induced lymphoproliferation.
- • 2. Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes) and confirmed by DNA sequencing
- • 3. Parental/guardian voluntary consent
- • 4. Boys between the ages of 0 and 16
About Great Ormond Street Hospital For Children Nhs Foundation Trust
Great Ormond Street Hospital for Children NHS Foundation Trust is a leading pediatric healthcare institution in the UK, renowned for its commitment to advancing child health through innovative research and clinical trials. As a prominent sponsor of clinical studies, the Trust focuses on developing and evaluating new treatments and therapies for a wide range of pediatric conditions, leveraging its extensive expertise and state-of-the-art facilities. With a multidisciplinary team of specialists and a patient-centered approach, Great Ormond Street Hospital aims to improve outcomes for children and contribute to the global body of medical knowledge.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
London, , United Kingdom
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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