Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1
- Conditions
- X-Linked severe combined Immunodeficiency (SCID-X1)MedDRA version: 9.1Level: LLTClassification code 10010099Term: Combined immunodeficiency
- Registration Number
- EUCTR2007-000684-16-GB
- Lead Sponsor
- Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 1
1a)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
1.No available molecular diagnosis confirming SCID-X1
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: 1.Treatment of SCID-X1 patients by somatic gene therapy when HLA-matched family or unrelated bone marrow donors are unavailable.<br>2.Successful ex vivo transduction of CD34+ haematopoietic cells from SCID-X1 patients by ex vivo gammaretrovirus-mediated gene transfer.<br>3.Evaluation of immunological and functional reconstitution in progeny of engrafted cells.<br>4.Longitudinal evaluation of clinical effect in terms of augmented immunity. <br>5.Evaluation of the functional performance of novel SIN gammaretroviral configuration.<br>6.Evaluation of the molecular characteristics of vector integration.<br>7.Evaluation of safety.<br>;Secondary Objective: ;Primary end point(s): Immunological reconstitution<br>
- Secondary Outcome Measures
Name Time Method