Organization
Spark Therapeutics
11 clinical trials
Clinical trial
Gene-transfer, Open-label, Dose-escalation Study of SPK-8011 [Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene] in Individuals With Hemophilia AStatus: Completed, Estimated PCD: 2023-12-05
Clinical trial
A Long-Term Follow-Up Study in Subjects Who Received an Adenovirus-Associated Viral Vector Serotype 2 Containing the Human RPE65 Gene (AAV2-hRPE65v2, Voretigene Neparvovec-rzyl) Administered Via Subretinal InjectionStatus: Active (not recruiting), Estimated PCD: 2030-03-01
Clinical trial
A Multi-Center Evaluation of the Long-Term Safety and Efficacy of Spark-sponsored Gene Therapies in Males With Hemophilia AStatus: Active (not recruiting), Estimated PCD: 2032-12-01
Clinical trial
A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the RetinaStatus: Completed, Estimated PCD: 2022-10-12
Clinical trial
A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in USStatus: Active (not recruiting), Estimated PCD: 2025-06-01
Clinical trial
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsStatus: Completed, Estimated PCD: 2020-10-14
Clinical trial
A Phase 3, Single-arm, Open-label, Multicenter Study of the Safety and Efficacy of Dirloctocogene Samoparvovec (SPK 8011, Adeno-associated Viral Vector With B-domain Deleted Human Factor VIII Gene) in Adults With Severe or Moderately Severe Hemophilia AStatus: Recruiting, Estimated PCD: 2027-05-01
Clinical trial
A Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis (LCA) Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 to the Retinal Pigment Epithelium (RPE) [AAV2-hRPE65v2-301]Status: Active (not recruiting), Estimated PCD: 2015-07-01
Clinical trial
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 StudyStatus: Active (not recruiting), Estimated PCD: 2030-03-01
Clinical trial
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe DiseaseStatus: Active (not recruiting), Estimated PCD: 2032-04-01
Clinical trial
A Multi-Center, Observational Study in Males With Hemophilia AStatus: Completed, Estimated PCD: 2023-05-02