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

Официальное название

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

Обзор

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).

Подробное описание

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.

Вмешательства

• Генная терапия 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

Первичные конечные точки

• Frequency of adverse events of special interest [Срок оценки: Up to 15 years]
• Frequency of reported AEs and SAEs/ADRs [Срок оценки: Up to 15 years]
• Actual values of laboratory blood test results (i.e. biochemistry, haematology) at each annual visits. [Срок оценки: At each annual visit up to 15 years]
• Number (%) of subjects with fertility and positive pregnancy outcomes [Срок оценки: Up to 15 years]
• The number (%) of subjects with an abnormal retroviral insertion site (RIS) analysis. [Срок оценки: Up to 15 years.]

Вторичные конечные точки (11)

• Overall Survival [Срок оценки: Up to 15 years]
• Event (Intervention) free survival [Срок оценки: Up to 15 years.]
• The number (%) of subjects requiring use of treatments of interest [Срок оценки: Up to 15 years.]
• Immune reconstitution [Срок оценки: Baseline and annually up to 15 years.]
• Growth [Срок оценки: Up to 15 years.]
• Systemic metabolite detoxification [Срок оценки: Baseline and annually up to 15 years.]
• Vector copy number, measured in PBMCs (peripheral blood mononuclear cells) and subpopulations. [Срок оценки: Up to 15 years.]
• Number and proportion of patients with severe infections, and associated length of stay [Срок оценки: Up to 15 years.]
• The number (%) of subjects falling into each category for pediatric development and quality of life assessments [Срок оценки: Up to 15 years.]
• Patient (or proxy) reported Peds-QL [Срок оценки: Up to 15 years.]
• Response to childhood vaccinations [Срок оценки: Up to 15 years.]

Критерии участия

Критерии включения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Италия · 1 центр

• Ospedale San Raffaele — Milan

Публикации

• 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

Идентификаторы

NCT: NCT03478670 · STRIM-003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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