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NCT00999609 · ClinicalTrials.gov registry record · Phase 3
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
A Phase 3 study of Leber Congenital Amaurosis and Inherited Retinal Dystrophy Due to RPE65 Mutations, sponsored by Spark Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 31
- Enrollment target
- 2
- Study locations
NCT00999609 is a Phase 3 study of Leber Congenital Amaurosis and Inherited Retinal Dystrophy Due to RPE65 Mutations that is active but no longer recruiting, run by Spark Therapeutics. The registered enrollment target is 31 participants, above the 23-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (35% higher). The trial reports 2 study locations across 2 states.
The verdict
NCT00999609, a Phase 3 study of Leber Congenital Amaurosis and Inherited Retinal Dystrophy Due to RPE65 Mutations, is active but no longer recruiting, sponsored by Spark Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 31 participants
- Enrollment target
- 2
- Study locations
Study Summary
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.
Conditions Studied
Interventions
- BIOLOGICAL AAV2-hRPE65v2,voretigene neparvovec-rzyl
Study Locations (2)
Iowa
- University of Iowa - Iowa City
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 31 participants |
| Start Date | 2012-10 |
| Est. Completion | 2030-01 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00999609
The ClinicalTrials.gov registry entry for NCT00999609 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 31 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 23-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (35% higher). The listed sponsor is Spark Therapeutics, which has 13 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Leber Congenital Amaurosis appearing as the primary indexed condition, and to 1 intervention - of which AAV2-hRPE65v2,voretigene neparvovec-rzyl is the first listed.
NCT00999609 reports 2 study locations spanning 2 distinct geographic areas - top geographies include Iowa, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT00999609 about?
NCT00999609 is a clinical study titled "Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis". The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at...
What is the current status of trial NCT00999609?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 31 participants. The study started on 2012-10. Estimated completion is 2030-01.
What conditions does trial NCT00999609 study?
This clinical trial studies the following conditions: Leber Congenital Amaurosis, Inherited Retinal Dystrophy Due to RPE65 Mutations.
What interventions are being tested in trial NCT00999609?
The interventions under investigation include: AAV2-hRPE65v2,voretigene neparvovec-rzyl (BIOLOGICAL).
Who is sponsoring clinical trial NCT00999609?
This trial is sponsored by Spark Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00999609 being conducted?
This trial has 2 study locations across Iowa, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Leber Congenital Amaurosis
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Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.