Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Strimvelis.
- Who it may be relevant to
- Registry conditions: Immunologic Deficiency Syndromes. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Registry for Patients Treated With Strimvelis (Previously GSK2696273) Gene Therapy: Long-Term Prospective, Non-Interventional Follow-up of Safety and Effectiveness
Overview
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 and is conducted as a post approval safety study associated with EMA marketing authorisation of Strimvelis™. In this study will be also included patients for whom the gene therapy medicinal product has been prepared starting from mobilized peripheral blood (mPB)-derived CD34+ cells (mPB-GT).
Detailed description
This is a prospective and retrospective, 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 or mPB-GT. The end of enrollment will be after the recruitment of the 50th patient and the registry will close when the 50th patient finishes the 15- year follow-up.
Interventions
- Genetic 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. In this study will be also included patients for whom the gene therapy medicinal product has been prepared starting from mobilized peripheral blood (mPB)-derived CD34+ cells, treated under hospital exemption (HE) frame, according to the Italian De
Primary outcome measures
- Frequency of adverse events of special interest [Time frame: Up to 15 years]
- Frequency of reported AEs and SAEs/ADRs [Time frame: Up to 15 years]
- Actual values of laboratory blood test results (i.e. biochemistry, haematology) at each annual visits. [Time frame: At each annual visit up to 15 years]
- Number (%) of subjects with fertility and positive pregnancy outcomes [Time frame: Up to 15 years]
- The number (%) of subjects with an abnormal retroviral insertion site (RIS) analysis. [Time frame: Up to 15 years.]
Secondary outcome measures (11)
- Overall Survival [Time frame: Up to 15 years]
- Event (Intervention) free survival [Time frame: Up to 15 years.]
- The number (%) of subjects requiring use of treatments of interest [Time frame: Up to 15 years.]
- Immune reconstitution [Time frame: Baseline and annually up to 15 years.]
- Growth [Time frame: Up to 15 years.]
- Systemic metabolite detoxification [Time frame: Baseline and annually up to 15 years.]
- Vector copy number, measured in PBMCs (peripheral blood mononuclear cells) and subpopulations. [Time frame: Up to 15 years.]
- Number and proportion of patients with severe infections, and associated length of stay [Time frame: Up to 15 years.]
- The number (%) of subjects falling into each category for pediatric development and quality of life assessments [Time frame: Up to 15 years.]
- Patient (or proxy) reported Peds-QL [Time frame: Up to 15 years.]
- Response to childhood vaccinations [Time frame: Up to 15 years.]
Eligibility criteria
Inclusion criteria
- Patients with ADA-SCID, treated with Strimvelis™ or GSK2696273, as part of its clinical development program or mPB-GT.
- Adult patients, or patients for whom their parents or legal guardians have signed the informed consent form for participation in the registry.
There are no formal exclusion criteria for participation as this registry will follow all patients who have received Strimvelis™ or GSK2696273, or mPB-GT prior to enrollment, subject to informed consent.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 1 center
- Ospedale San Raffaele — Milan
Publications
- Migliavacca M, Barzaghi F, Fossati C, Rancoita PMV, Gabaldo M, Dionisio F, Giannelli S, Salerio FA, Ferrua F, Tucci F, Calbi V, Gallo V, Recupero S, Consiglieri G, Pajno R, Sambuco M, Priolo A, Ferri C, Garella V, Monti I, Silvani P, Darin S, Casiraghi M, Corti A, Zancan S, Levi M, Cesana D, Carlucci F, Pituch-Noworolska A, AbdElaziz D, Baumann U, Finocchi A, Cancrini C, Ladogana S, Meinhardt A, M PMID 38355973
Identifiers
NCT: NCT03478670 · STRIM-003