Spark Therapeutics is the lead sponsor of 4 current clinical trials in our USA and UK registers. None of its ClinicalTrials.gov trials is recruiting at the moment. The most common phase is Phase 1/2. The most-studied condition is Leber Congenital Amaurosis.
| Study | Phase | Status | Start | Registers |
|---|---|---|---|---|
|
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT04093349 · Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2
|
Phase 1/2 | Active, closed | 2020-10 | UK, USA |
|
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609 · ClinicalTrials.gov ↗ · Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
|
Phase 3 | Active, closed | 2012-10 | USA |
|
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
NCT01208389 · ClinicalTrials.gov ↗ · Leber Congenital Amaurosis
|
Phase 1/2 | Active, closed | 2010-11 | USA |
|
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A
NCT03432520 · ClinicalTrials.gov ↗ · Hemophilia A
|
Observational | Active, closed | 2018-08 | USA |
Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.
| Register | Trials | Recruiting |
|---|---|---|
| Clinical trials in USA | 4 | 0 |
| Clinical trials in UK | 1 | 0 |
A trial with sites in more than one country appears in each of those registers.
| Sponsor | Trials |
|---|---|
| Laboratoires Thea | 1 |
| HuidaGene Therapeutics | 1 |
Sponsors with the most current trials registered for this condition.
Spark Therapeutics is the lead sponsor of 4 current trials on ClinicalTrials.gov in our USA and UK registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.
Not at the moment. None of its 4 ClinicalTrials.gov trials is recruiting; they are active but closed to enrolment, not yet open, or enrolling by invitation. Only the study team can confirm whether a patient is eligible.
Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.
Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.
This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.