Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy — LHON

Leber's Hereditary Optic Neuropathy Clinical Trial

Official title:
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA

Status Active, not recruiting

Clinical Trial Summary

Hypotheses: The primary hypothesis being tested is that there will be no toxicity resulting in loss of vision to no light perception in injected eyes.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Leber's Hereditary Optic Neuropathy
Optic Atrophy, Hereditary, Leber
Optic Nerve Diseases

Clinical Trial Details

NCT number	NCT02161380
Study type	Interventional
Source	University of Miami
Contact
Status	Active, not recruiting
Phase	Phase 1
Start date	July 14, 2014
Completion date	March 31, 2024

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT01421381` - RHODOS Follow-up Single-visit Study	N/A
Completed	`NCT00747487` - Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy	Phase 2
Completed	`NCT04909398` - Pupil Dynamics and Color Vision for the Detection of Eye Diseases	N/A
Completed	`NCT02693119` - A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy	Phase 2
Withdrawn	`NCT01495715` - Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)	Phase 3
No longer available	`NCT04381091` - Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study
No longer available	`NCT02300753` - Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]	N/A