|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
EUCTR |
|
Last refreshed on:
|
20 July 2020 |
|
Main ID: |
EUCTR2007-000684-16-GB |
|
Date of registration:
|
17/09/2009 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1
|
|
Scientific title:
|
Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector. - Gene therapy for SCID-X1 |
|
Date of first enrolment:
|
21/01/2010 |
|
Target sample size:
|
10 |
|
Recruitment status: |
Not Recruiting |
|
URL:
|
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-000684-16 |
|
Study type:
|
Interventional clinical trial of medicinal product |
|
Study design:
|
Controlled: no
Randomised:
Open:
Single blind:
Double blind:
Parallel group:
Cross over:
Other:
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
|
|
Phase:
|
Human pharmacology (Phase I): yes
Therapeutic exploratory (Phase II): yes
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): no
|
|
|
Countries of recruitment
|
|
United Kingdom
| | | | | | | |
|
Contacts
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
| |
|
Key inclusion & exclusion 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:
1. No available molecular diagnosis confirming SCID-X1
Age minimum:
Age maximum:
Gender:
Female: no
Male: yes
|
|
Health Condition(s) or Problem(s) studied
|
X-Linked severe combined Immunodeficiency (SCID-X1)
MedDRA version: 9.1
Level: LLT
Classification code 10010099
Term: Combined immunodeficiency
|
|
Intervention(s)
|
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.IL2RG.pre* retroviral vector transduced cells
Product Code: pSRS11.EFS.IL2RG.pre* retroviral vector transduce
Pharmaceutical Form: Solution for infusion
|
|
Primary Outcome(s)
|
|
Secondary Objective:
|
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.
|
Primary end point(s): Immunological reconstitution
|
|
Source(s) of Monetary Support
|
|
Ethics review
|
Status: Approved
Approval date: 11/05/2007
Contact:
|
|