Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) | Recruiting
Gene Therapy for X-linked Severe Co... | Recruiting
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

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Medical Conditions
  • X-linked Severe Combined Immunodeficiency
Primary Contact Details
Recruitment Status
Recruiting
Trial source and source ID number
NCT01175239

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This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
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 therapy to replace the defective gene.
Research Details
    Sorry, this information is not available
Phase
Phase 1/Phase 2
Study Design
Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Study Type
Interventional
Intervention
Genetic : Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre

Study Arm Groups : Single infusion of autologous CD34+ cells

Intervention Type
See Interventions above
Primary Outcome Measures
    Immunological reconstitution; 1-18 months post-infusion,then annually
Secondary Outcome Measures
    Incidence of adverse reactions; from consent until 5 years post-infusion of gene-modified cells; Molecular characterisation of gene transfer; until 5 years post-infusion of gene-modified cells; Normalisation of nutritional status, growth, and development; until 5 years post-infusion of gene-modified cells
Publication(s)
Sorry, this information is not available
Result Reports
Check availability of results on the Clinicaltrials.gov website
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Gender
Male
Age Range
N/A - 16 Years
Who Can Participate
Patients
Number of Participants
10
Participant Inclusion 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
Participant Exclusion Criteria
This is in the inclusion criteria above
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Trial Location(s)
London
WC1N 3JH
Trial Contact(s)
Primary Trial Contact
Adrian Thrasher, Professor
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
United Kingdom
This information is designed to help you decide whether this trial is of interest. In some cases it is provided as a link to more detailed patient information or it may still be awaited from the organisation running the trial. Please look again shortly if the information you need is not here or, if named, contact the researcher named above.
Scientific Title
Gene Therapy for SCID-X1 Using a Self-inactivating (SIN) Gammaretroviral Vector
EudraCT Number
Not available for this trial
Funder(s)
    Sorry, this information is not available
Other Study ID Numbers
06MI10
Sponsor(s)
Great Ormond Street Hospital for Children NHS Foundation Trust
Key Dates

Recruitment Start Date

Apr 2011

Recruitment End Date

Dec 2018

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date added to source

29 Jul 2010

Date updated in source

11 Sep 2015