SPARK THERAPEUTICS, INC. Clinical Trials
Studies registered on ClinicalTrials.gov (U.S. National Library of Medicine) with the company as lead sponsor or collaborator, newest start first. Values are as registered.
- 2024-03-13: Study NCT06297486; Company role Lead sponsor; Phase Phase 3; Status Withdrawn.
- 2020-10-01: Study NCT04093349; Company role Lead sponsor; Phase Phase 1, Phase 2; Status Active not recruiting.
- 2019-06-12: Study NCT03893240; Company role Lead sponsor; Phase Not applicable; Status Completed.
- 2019-01-30: Study NCT03734588; Company role Lead sponsor; Phase Phase 1, Phase 2; Status Completed.
| Start date | Study | Company role | Phase | Status | Conditions | Enrollment | Completion date |
|---|---|---|---|---|---|---|---|
| 2024-03-13 | NCT06297486 Study of a Gene Therapy Treatment for Hemophilia A | Lead sponsor | Phase 3 | Withdrawn | Hemophilia A | 0 | 2035-09-04 |
| 2020-10-01 | NCT04093349 A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) | Lead sponsor | Phase 1, Phase 2 | Active not recruiting | Pompe Disease; Pompe Disease (Late-onset); Glycogen Storage Disease Type 2; Glycogen Storage Disease Type II; LOPD; Lysosomal Storage Diseases; Acid Maltase Deficiency | 4 | 2032-04 |
| 2019-06-12 | NCT03893240 Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease | Lead sponsor | Not applicable | Completed | Pompe Disease; Pompe Disease (Late-onset); Glycogen Storage Disease Type 2; LOPD; Lysosomal Storage Diseases; Acid Maltase Deficiency | 61 | 2020-10-27 |
| 2019-01-30 | NCT03734588 Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors | Lead sponsor | Phase 1, Phase 2 | Completed Results posted | Adeno-Associated Virus (AAV); Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Factor VIII (FVIII); Factor VIII (FVIII) Deficiency; Factor VIII (FVIII) Gene; Factor VIII (FVIII) Protein; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Gene Therapy; Gene Transfer; Hematologic Diseases; Hemorrhagic Disorders; Recombinant; Vector; Inhibitors | 4 | 2023-01-19 |
| 2019-01-21 | NCT03876301 Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants | Lead sponsor | Observational | Completed | Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hematologic Diseases; Hemorrhagic Disorders; Factor VIII Deficiency | 25 | 2023-05-02 |
| 2019-01-10 | NCT03597399 A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US | Lead sponsor | Observational | Completed | Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy | 87 | 2025-06-30 |
| 2018-08-14 | NCT03432520 Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A | Lead sponsor | Observational | Active not recruiting | Hemophilia A | 29 | 2032-12 |
| 2017-01-26 | NCT03003533 A Gene Transfer Study for Hemophilia A | Lead sponsor | Phase 1, Phase 2 | Completed Results posted | Hemophilia A | 25 | 2023-12-05 |
| 2015-01-15 | NCT02341807 Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations | Lead sponsor | Phase 1, Phase 2 | Completed Results posted | Choroideremia; CHM (Choroideremia) Gene Mutations | 15 | 2022-10-12 |
| 2012-10 | NCT01620801 Hemophilia B Gene Therapy With AAV8 Vector | Lead sponsor | Phase 1 | Terminated | Hemophilia B | 4 | 2016-03 |
| 2012-10 | NCT00999609 Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis | Lead sponsor | Phase 3 | Active not recruiting Results posted | Inherited Retinal Dystrophy Due to RPE65 Mutations; Leber Congenital Amaurosis | 31 | 2030-01 |
| 2010-11 | NCT01208389 Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | Lead sponsor | Phase 1, Phase 2 | Active not recruiting | Leber Congenital Amaurosis | 12 | 2027-10 |
| 2007-09 | NCT00516477 Safety Study in Subjects With Leber Congenital Amaurosis | Lead sponsor | Phase 1 | Completed | Leber Congenital Amaurosis | 12 | 2018-03-20 |
| 2007-08 | NCT00515710 LTFU for Gene Transfer Subjects With Hemophilia B | Lead sponsor | Observational | Completed | Hemophilia B | 4 | 2017-12 |