Spark Therapeutics, Inc. clinical trials
Acquired by Hoffmann-La Roche, Dec 17, 2019 · source ↗
The registry lists 14 studies whose lead sponsor is Spark Therapeutics, Inc.. 0 are recruiting or about to start. Per ClinicalTrials.gov, last updated Sep 22, 2025.
- 14studies on the registry
- 0open to participants now
- 1phase 3 study under way
- 1reach primary completion within a year
Before and after it joined Hoffmann-La Roche
- First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- 13
- First submitted after Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- 1
- Unclear, or no submission date
- 0
By the date each study was first submitted to the registry. The registry lists today's sponsor name and keeps no history, so this says when a study was filed, not who filed it.
What it is testing
The drugs Spark Therapeutics, Inc.’s studies test, most studies first; none is open now — 2 drugs with a page of their own. Each count opens the studies in search.
Studies, none open
- voretigene neparvovec1 in phase 30 open · 3
- 0 open · 1
Coming up
1 of Spark Therapeutics, Inc.’s studies under way reach primary completion within a year. The dates are the sponsor’s own estimates, which often move.
Phase 3 Other phases
- NNov ’26’26
- DDec
- JJan ’27’27
- FFeb
- MMar
- AApr
- MMay
- JJun
- JJul
- AAug
- SSep
- 1OOct
- NNov
The study
Add it to your calendar (.ics) → With an account.
All 14 at a glance
Where each study is in its life, its phase and its countries. Each count opens them in search.
Over the years
- 07
- 08
- 09
- 10
- 11
- 12
- 13
- 14
- 15
- 16
- 17
- 18
- 419
- 20
- 21
- 22
- 23
- 24
- 25
- 026
By the year each study started. 2026 so far in the lighter column.
Registry sponsor names on this page
- Spark Therapeutics, Inc.14 studies
Something wrong with this grouping? Tell us on the Hoffmann-La Roche page.
Studies
Recruiting first, then most recently updated. Showing 14 of 14. All Hoffmann-La Roche studies →
- A Patient Registry Study for Patients Treated With Voretigene Neparvovec in USCompletedNCT03597399
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2Active, not recruitingNCT01208389Phase 1/2
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Safety and Efficacy Study in Subjects With Leber Congenital AmaurosisActive, not recruitingNCT00999609Phase 3
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- A Gene Transfer Study for Hemophilia ACompletedNCT03003533Phase 1/2
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Study of a Gene Therapy Treatment for Hemophilia AWithdrawnNCT06297486Phase 3
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted after Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia AActive, not recruitingNCT03432520
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)Active, not recruitingNCT04093349Phase 1/2
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsCompletedNCT03734588Phase 1/2
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene MutationsCompletedNCT02341807Phase 1/2
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A ParticipantsCompletedNCT03876301
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe DiseaseCompletedNCT03893240Not applicable
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Safety Study in Subjects With Leber Congenital AmaurosisCompletedNCT00516477Phase 1
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- LTFU for Gene Transfer Subjects With Hemophilia BCompletedNCT00515710
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche
- Hemophilia B Gene Therapy With AAV8 VectorTerminatedNCT01620801Phase 1
Sponsor listed in the registry: Spark Therapeutics, Inc. · First submitted before Spark Therapeutics, Inc. became part of Hoffmann-La Roche