Last updated: 06/27/2019 16:30:11
This product has been transferred to Orchard Therapeutics Limited. GSK Clinical Study Register is no longer maintained for this study. The most up to date information is available on clinicaltrials.gov
Phase IV, GSK2696273, Registry study to follow-up patients with Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
GSK study ID
200195
Clinicaltrials.gov ID
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Trial overview
Official title: Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated with Strimvelis (or GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness
Trial description: Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder. Strimvelis (or GSK2696273) is a gene therapy intended for patients with ADA-SCID and for whom no suitable human leukocyte antigen (HLA) matched related stem cell donor is available. This therapy aims to restore ADA function in hematopoietic cell lineages, and in doing so prevents the pathology caused by purine metabolites (i.e., impaired immune function). This registry will evaluate the long term safety and effectiveness outcomes of subjects who have received Strimvelis (or GSK2696273).
Primary purpose:
Not applicable
Trial design:
Not applicable
Masking:
Not applicable
Allocation:
Not applicable
Primary outcomes:
Overall survival
Timeframe: Up to 21 years
Intervention free survival
Timeframe: Up to 15 years
Number of subjects with the use of medications/treatments of interest
Timeframe: Up to 15 years
Absolute peripheral lymphocyte for Immune reconstitution assessment
Timeframe: Up to 15 years
Absolute cluster of differentiation (CD)3+ T-cell for Immune reconstitution assessment
Timeframe: Up to 15 years
Absolute CD19+ B-cell counts for Immune reconstitution assessment
Timeframe: Up to 15 years
Phytohaemagglutinin (PHA) and anti CD-3 as a measure for T cell function
Timeframe: Up to 15 years
Growth percentile in body height
Timeframe: Up to 15 years
Growth percentile in body weight
Timeframe: Up to 15 years
Deoxyadenosine nucleotides (dAXP) levels in red blood cells for the measurement of systemic metabolite detoxification
Timeframe: Up to 15 years
Vector copy number measured in peripheral blood mononuclear cells (PBMCs)
Timeframe: Up to 15 years
Number of subjects with severe infections
Timeframe: Up to 15 years
Percentage of subjects with severe infections
Timeframe: Up to 15 years
Length of hospital stay
Timeframe: Up to 15 years
Number of subjects with non-immunological manifestations of ADA SCID
Timeframe: Up to 15 years
Pediatric development and quality of life data
Timeframe: Up to 15 years
Scores for Pediatric Quality of Life Questionnaire (Peds-QL)
Timeframe: Up to 15 years
Scores for Ages and Stages Questionnaire-3[ASQ-3]
Timeframe: Up to 15 years
Number of subjects with adverse events of interest
Timeframe: Up to 15 years
Number of subjects with any adverse events (AEs) and any serious adverse events (SAEs) as a safety measure
Timeframe: Up to 15 years
Number of subjects with abnormal clinical laboratory blood test results as a safety measure
Timeframe: Up to 15 years
Number of subjects with fertility and pregnancy related outcomes
Timeframe: Up to 21 years
Data from Retroviral Insertion Site (RIS) analysis and replication competent retrovirus (RCR)
Timeframe: Up to 21 years
Secondary outcomes:
Not applicable
Interventions:
Genetic: Strimvelis (or GSK2696273)
Enrollment:
50
Observational study model:
Cohort
Primary completion date:
2037-31-05
Time perspective:
Prospective
Clinical publications:
Not applicable
Medical condition
Immunologic Deficiency Syndromes
Product
GSK2696273
Collaborators
Not applicable
Study date(s)
March 2017 to May 2037
Type
Observational
Phase
4
Participation criteria
Sex
Female & Male
Age
Not applicable
Accepts healthy volunteers
No
- Inclusion Criteria
- Subject with ADA-SCID, treated with Strimvelis or GSK2696273 as part of its clinical development program
Inclusion and exclusion criteria
Inclusion criteria:
- Inclusion Criteria
- Subject with ADA-SCID, treated with Strimvelis or GSK2696273 as part of its clinical development program
- Adult subjects, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry
Trial location(s)
Study documents
No study documents available.
Results overview
No study documents available
Recruitment status
No longer a GSK study
Actual primary completion date
Not applicable
Actual study completion date
Not applicable
Plain language summaries
Not applicable. GSK’s transparency policy provides for Plain Language Summaries for Interventional studies.
Additional information about the trial
Not applicable
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