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

United Kingdom1 participantsStarted 2011-04

Plain-language 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.

Who can participate

Age range16 Years

SexMALE

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Inclusion criteria

✓. 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.
✓. Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes) and confirmed by DNA sequencing
✓. Parental/guardian voluntary consent
✓. Boys between the ages of 0 and 16

What they're measuring

Immunological reconstitution

Timeframe: 1-18 months post-infusion,then annually

Trial details

NCT IDNCT01175239

SponsorGreat Ormond Street Hospital for Children NHS Foundation Trust

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2018-12

View on ClinicalTrials.gov More X-linked Severe Combined Immunodeficiency trials