Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01208389
The ClinicalTrials.gov registry entry for NCT01208389 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 26-participant average among 6 other Leber Congenital Amaurosis trials with a reported enrollment target (54% lower). The listed sponsor is Spark Therapeutics, which has 13 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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.
Learn More About Clinical Trials
Similar trials for Leber Congenital Amaurosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
RECRUITING · Phase 1
-
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
ACTIVE NOT RECRUITING · Phase 1
-
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
ACTIVE NOT RECRUITING · Phase 1
-
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
RECRUITING · Phase 3
-
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
ACTIVE NOT RECRUITING · Phase 2
-
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
ACTIVE NOT RECRUITING · Phase 3
Read our methodology - how this data is sourced, computed, and verified.
Related
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.