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 GSK Clinical Study Register is no longer maintained for this study.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
NCT03478670
EudraCT ID
Not applicable
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

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
    Primary completion date:
    2037-31-05
    Observational study model:
    Cohort
    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)

    Location
    Status
    Contact us
    Contact us
    Location
    GSK Investigational Site
    Milano, Lombardia, Italy, 20132
    Status
    Recruiting
    Contact us

    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
    Participate in clinical trial
    Access to clinical trial data by researchers
    Visit website