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NCT01208389 · ClinicalTrials.gov registry record · Phase 1
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
A Phase 1 study of Leber Congenital Amaurosis, sponsored by Spark Therapeutics.
- Active
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT01208389: Active Phase 1 study of Leber Congenital Amaurosis, sponsored by Spark Therapeutics.
NCT01208389 is a Phase 1 study of Leber Congenital Amaurosis that is active but no longer recruiting, run by Spark Therapeutics. The registered enrollment target is 12 participants, below the 26-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (54% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01208389, a Phase 1 study of Leber Congenital Amaurosis, is active but no longer recruiting, sponsored by Spark Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
Study Summary
The study is a follow-on to a Phase 1 dose-escalation and safety study.
Primary Outcome
The primary outcome measures are safety and tolerability. Secondary outcome measure(s) include changes in visual function as measured by subjective, psychophysical tests and by objective, physiologic tests.
Conditions Studied
Interventions
- BIOLOGICAL voretigene neparvovec-rzyl
Study Locations (1)
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2010-11 |
| Est. Completion | 2027-10 |
| Phase | Phase 1 |
What the registry record for NCT01208389 still lists
NCT01208389 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 26-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (54% lower).
The record links to 1 condition, with Leber Congenital Amaurosis appearing as the primary indexed condition, and to 1 intervention - of which voretigene neparvovec-rzyl is the first listed.
NCT01208389 reports a single indexed study location in Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT01208389 about?
NCT01208389 is a clinical study titled "Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2". The study is a follow-on to a Phase 1 dose-escalation and safety study.
What is the current status of trial NCT01208389?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2010-11. Estimated completion is 2027-10.
What conditions does trial NCT01208389 study?
This clinical trial studies the following conditions: Leber Congenital Amaurosis.
What interventions are being tested in trial NCT01208389?
The interventions under investigation include: voretigene neparvovec-rzyl (BIOLOGICAL).
Who is sponsoring clinical trial NCT01208389?
This trial is sponsored by Spark Therapeutics, which has 13 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01208389 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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