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Safety Study in Subjects With Leber Congenital Amaurosis

NCT00516477 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-11-02

No results posted yet for this study

Summary

The purpose of this study is to determine whether gene transfer will be safe and effective in the treatment of Leber Congenital Amaurosis (LCA).

Conditions

  • Leber Congenital Amaurosis

Interventions

BIOLOGICAL

voretigene neparvovec-rzyl

Subjects will be dosed unilaterally (one eye) beginning with the lowest dose. Subjects will be injected with AAV2-hRPE65v2 by means of a subretinal injection. Dose escalation to the next cohort will be dependent on assessment of the safety data by the DSMB out to at least 4 weeks following the injection. Because there is a delay between time of delivery of AAV2 and the peak transgene expression there will be a delay of six weeks between all subjects.

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Clinical Director · Spark Therapeutics, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-09-30
Primary Completion
2018-03-20
Completion
2018-03-20
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00516477 on ClinicalTrials.gov