Clinical Trials Directory

Trials / Sponsors / Spark Therapeutics, Inc.

Spark Therapeutics, Inc.

Industry · 15 registered clinical trials.

StatusTrialPhaseStarted
WithdrawnStudy of a Gene Therapy Treatment for Hemophilia A
Hemophilia A
Phase 32024-03-13
Active Not RecruitingA Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2
Phase 1 / Phase 22020-10-01
CompletedNeutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pomp
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2
N/A2019-06-12
CompletedDose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals
Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Phase 1 / Phase 22019-01-30
CompletedLead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
2019-01-21
CompletedA Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
2019-01-10
Active Not RecruitingLong-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
Hemophilia A
2018-08-14
CompletedA Gene Transfer Study for Hemophilia A
Hemophilia A
Phase 1 / Phase 22017-01-26
Active Not RecruitingLong-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
Inherited Retinal Dystrophy Due to RPE65 Mutations
2015-06-01
CompletedSafety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Choroideremia, CHM (Choroideremia) Gene Mutations
Phase 1 / Phase 22015-01-15
Active Not RecruitingSafety and Efficacy Study in Subjects With Leber Congenital Amaurosis
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Phase 32012-10-01
TerminatedHemophilia B Gene Therapy With AAV8 Vector
Hemophilia B
Phase 12012-10-01
Active Not RecruitingPhase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
Leber Congenital Amaurosis
Phase 1 / Phase 22010-11-01
CompletedSafety Study in Subjects With Leber Congenital Amaurosis
Leber Congenital Amaurosis
Phase 12007-09-01
CompletedLTFU for Gene Transfer Subjects With Hemophilia B
Hemophilia B
2007-08-01