Status:
UNKNOWN
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
Lead Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Conditions:
X-linked Severe Combined Immunodeficiency
Eligibility:
MALE
Up to 16 years
Phase:
NA
Brief Summary
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys. This trial aims to treat SCID-X1 patients using gene t...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- 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.
- Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes) and confirmed by DNA sequencing
- Parental/guardian voluntary consent
- Boys between the ages of 0 and 16
Exclusion
Key Trial Info
Start Date :
April 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2018
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT01175239
Start Date
April 1 2011
End Date
December 1 2018
Last Update
August 1 2017
Active Locations (1)
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1
Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom, WC1N 3JH