Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-03-28

Study Completion Date

2037-05-31

Brief Summary

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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 evaluates the long term safety and effectiveness outcomes of subjects who have received Strimvelis (or GSK2696273).

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Detailed Description

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This is a prospective, non-interventional follow-up registry of patients with ADA-SCID treated with Strimvelis™. The registry does not have a comparator group and the product will have been given on a single occasion prior to entering this registry. Safety and effectiveness will be assessed for a target number of 50 patients who will have received Strimvelis™ (or GSK2696273) comprising patients treated prior to marketing authorisation (i.e. clinical studies and compassionate use programs) and those treated after marketing authorisation (including within compassionate use and early access programs). The registry will close to enrolment when 50 patients have been enrolled but will not close completely until the 50th patient finishes their 15 year follow-up.

Conditions

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Immunologic Deficiency Syndromes

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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ADA-SCID subjects treated with Strimvelis

Subjects with ADA-SCID who have received Strimvelis (previously GSK2696273) gene therapy, comprising patients treated prior to marketing authorisation (i.e. clinical studies and compassionate use programs) and those treated after marketing authorisation (including within compassionate use and early access programs).

Strimvelis

Intervention Type GENETIC

Strimvelis is a CD34+ cell enriched dispersion of human autologous bone marrow derived hematopoietic stem/progenitor cells transduced with a retroviral vector containing the human ADA gene. It will be administered as an intravenous infusion once only.

This is an observational registry that includes all patients who have previously received Strimvelis™ or GSK2696273.

Interventions

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Strimvelis

Strimvelis is a CD34+ cell enriched dispersion of human autologous bone marrow derived hematopoietic stem/progenitor cells transduced with a retroviral vector containing the human ADA gene. It will be administered as an intravenous infusion once only.

This is an observational registry that includes all patients who have previously received Strimvelis™ or GSK2696273.

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

1. Patient with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program.
2. Adult patients, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry.

Eligible Sex

ALL

Accepts Healthy Volunteers

No