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Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

NCT02341807 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2024-01-25

Study results available
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Summary

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

Conditions

  • Choroideremia
  • CHM (Choroideremia) Gene Mutations

Interventions

BIOLOGICAL

AAV2-hCHM

Comparison of different dosages of AAV2-hCHM

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-15
Primary Completion
2022-10-12
Completion
2022-10-12

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 NCT02341807 on ClinicalTrials.gov