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Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy

Study acronym: GOLD
StatusRecruiting
PhasePhase 2/3
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated November 14, 2022 (before its estimated February 29, 2024 completion). Its status may not reflect the trial's current state.

Record History

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  1. Phase: Phase 1/2 to Phase 2/3v4 Show 6 more changesShow less
    • Primary endpoints revised (3)
    • Primary completion date: Dec 30, 2023 to Feb 29, 2024
    • Study completion date: Apr 30, 2027 to Feb 29, 2028
    • Secondary endpoints: 11 to 6
    • Study description expanded, about 35 words
    • Eligibility criteria expanded, about 3 words
  2. Enrollment target: 55 to 102v3 Show 7 more changesShow less
    • Primary endpoints: 1 to 3
    • Interventions: NR082 injection (Drug) to NR082 injection (Drug), Sham Injection (Device)
    • Eligibility criteria expanded, about 83 words
    • Secondary endpoints: details revised at 1 of 11 entries
    • Study description shortened, about 42 words
    • Study arms: 1 to 2
    • Study title shortened, about 4 words
  3. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Jun 15, 2021 to Jun 18, 2021
    • Site details revised
    v2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
The objective of this clinical study is to select the optimal dose and evaluate the safety and efficacy of NR082 in treatment of LHON caused by mitochondrial ND4 gene mutation. Part 1 (Phase 1/2) is a safety dose-finding study, which will enroll subjects aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NR082 to observe its safety and efficacy. In Part 2 (Phase 3) of the clinical study, the dose recommended after the end of Part 1 is used to further verify the safety and efficacy of the study drug. Part 2 of the study is divided into the safety run-in phase and the randomized, double-blind and control study. Subjects aged ≥ 12 years and ≤ 75 years will be enrolled in the Part 2. The run-in phase will enroll 6 evaluable subjects. After monitoring for at least 6 weeks, if no new safety signals are observed, the clinical trial will enter the randomized, double-blind and control study phase upon approval by the Safety Review Committee(SRC). The clinical manifestation of all subjects is reduced visual acuity caused by LHON associated with ND4 mutation, and central laboratory test showed G11778A mutation (a CLIA-certified laboratory), while the reduced visual acuity lasted for \> 6 months and \< 10 years.
Trial Details
NCT Number NCT04912843
Lead Sponsor Wuhan Neurophth Biotechnology Limited Company
Conditions
(from sponsor)		 Leber's Hereditary Optic Neuropathy (LHON)
Related terms Optic Atrophy, Hereditary, Leber
Enrollment 102 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov