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Health Condition(s) or Problem
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X-linked Severe Combined Immunodeficiency
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Lay Summary
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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.
(from ClinicalTrials.gov)
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Who can enter the trial
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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
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Who cannot enter the trial
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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
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What will happen
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Genetic; Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre; Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre; Single infusion of autologous CD34+ cells
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Primary aim
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Immunological reconstitution
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Secondary Aim
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Incidence of adverse reactions; from consent until 5 years post-infusion of gene-modified cells; No; At each scheduled visit, adverse events that might have occurred since the previous visit or assessment will be elicited from the patient/parent/guardian.
The investigators will maintain a record of all adverse events/occurrences in patients participating in the clinical trial. This record will be noted in the patient's medical notes.
Adverse events that have a causal relationship to the IMP (ARs) and SAEs will be recorded on the AE reporting section of the CRF.; Molecular characterisation of gene transfer; until 5 years post-infusion of gene-modified cells; No; Quantification of transgene copy numbers is determined on sorted cell populations by real-time PCR methodology. Detailed integration analysis may be used to investigate specific clonal expansions.; Normalisation of nutritional status, growth, and development; until 5 years post-infusion of gene-modified cells; No; Normalisation of nutritional status, growth, and development will be assessed at each follow-up visit by the investigator through clinical examinations.
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Participant Information Sheet
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Sorry, not currently available
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Website
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Sorry, not currently available
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Recruitment Status
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Recruiting
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Nation
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England
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Location
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London
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