- 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
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–5
of 5
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
- Interventions
- AAV OPTIRPE65
- Biological
- Lead sponsor
- MeiraGTx UK II Ltd
- Industry
- Eligibility
- 3 Years to 100 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2023
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- 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
- Leber Congenital Amaurosis
- Interventions
- rAAV2-CB-hRPE65
- Biological
- Lead sponsor
- Beacon Therapeutics
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Worcester, Massachusetts • Portland, Oregon
- Conditions
- Leber Congenital Amaurosis
- Interventions
- AAV RPE65
- Biological
- Lead sponsor
- MeiraGTx UK II Ltd
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan