X-Linked severe combined Immunodeficiency (SCID-X1) MedDRA version: 9.1 Level: LLT Classification code 10010099 Term: Combined immunodeficiency
Conditions
Interventions
Product Name: pSRS11.EFS.IL2RG.pre* retroviral vector
Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector
Pharmaceutical Form: Solution for blood fraction modification
Product Name: pSRS11.EFS.IL
Sponsors
Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1 a) No HLA identical (A,B,C,DR,DQ) family donor b) No HLA identical unrelated donor available within 3 mths of diagnosis or c) 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 (clinical genetics laboratory, GOSH) 3. Parental/guardian voluntary consent Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. No available molecular diagnosis confirming SCID-X1
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. Treatment of SCID-X1 patients by somatic gene therapy when HLA-matched family or unrelated bone marrow donors are unavailable. 2. Successful ex vivo transduction of CD34+ haematopoietic cells from SCID-X1 patients by ex vivo gammaretrovirus-mediated gene transfer. 3. Evaluation of immunological and functional reconstitution in progeny of engrafted cells. 4. Longitudinal evaluation of clinical effect in terms of augmented immunity. 5. Evaluation of the functional performance of novel SIN gammaretroviral configuration. 6. Evaluation of the molecular characteristics of vector integration. 7. Evaluation of safety. ;Secondary Objective: ;Primary end point(s): Immunological reconstitution | — |
Countries
United Kingdom
Outcome results
None listed