- Conditions
- Leber Congenital Amaurosis
- Interventions
- voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2018
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–4
of 4
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Leber Congenital Amaurosis
- Interventions
- voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
- Interventions
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2030
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Philadelphia, Pennsylvania
- Conditions
- Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
- Interventions
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 12 Months and older
- Enrollment
- 87 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more