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

Trial Source

There is no location for this trial

Location data is sourced from multiple external providers and UKCTG is not responsible for and cannot guarantee the accuracy of data.

Health Conditions
  • X-linked Severe Combined Immunodeficiency
Primary Contact Details
Recruiting
Recruitment Status
NCT01175239
Primary Trial ID Number
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
This is available on the Clinicaltrials.gov website
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
Trial Location(s)
GSK Investigational Site
London
WC1N 3JH
Trial Contact(s)
Primary Trial Contact
Adrian Thrasher, Professor
a.thrasher@ich.ucl.ac.uk
Other Trial Contacts
Sorry, this information is not available
Countries Recruiting
United Kingdom
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

Sep 2013

Trial Start Date
Date Not Available
Trial End Date
Date Not Available
Date Assigned

29 Jul 2010

Last Updated

01 Mar 2012