TerminatedPhase 1/2

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

Stopped: Due to the sponsor circumstances and external reasons

United States, China11 participantsStarted 2023-08-15

Plain-language summary

The objective of this clinical study is to evaluate the safety, tolerability and preliminary efficacy of NFS-02 in the treatment of LHON caused by mitochondrial ND1 gene mutation. This study will enroll subjects aged ≥ 18 years old and ≤ 75 years old to receive a single unilateral intravitreal (IVT) injection of NFS-02 to evaluate its safety, tolerability and preliminary efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND1 mutation, with laboratory test showing G3460A mutation (a CLIA-certified laboratory) and reduced visual acuity lasted for \> 6 months and \< 10 years.

Who can participate

Age range18 Years – 75 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Age at the time of signing the informed consent form: the age of the subjects must be ≥ 18 years old and ≤ 75 years old Type of Subject and Disease Characteristics
✓. The clinical manifested vision loss due to LHON, and any eye BCVA ≥ 0.5 LogMAR
✓. The genotype testing result shows the presence of G3460A mutation in the ND1 gene, and the absence of the other primary LHON associated mutations in the mitochondrial DNA (mtDNA) (ND4 \[G11778A\] or ND6 \[T14484C\]) (confirmed by a CLIA-certified international laboratory)
✓. The vision loss in the eye with worse visual acuity lasted \> 6 months and \< 10 years at screening
✓. Pupils can be adequately dilated for a thorough ocular examination and visual acuity test
✓. Each eye of the subject must maintain at least Hand Motion visual acuity (VA) (≤ 2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for refraction and VA examinations) in this study
✓. Willingness to comply with the clinical study protocol and 5 years of long-term follow-up after administration Sex
✓. Male or female

Exclusion criteria

What they're measuring

Incidence of adverse events (AEs)

Timeframe: 52 weeks

Incidence of serious adverse events (SAEs)

Timeframe: 52 weeks

Incidence of dose-limiting toxicities (DLT)

Timeframe: 52 weeks

Trial details

NCT IDNCT05820152

SponsorNeurophth Therapeutics Inc

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-06-24

View on ClinicalTrials.gov More Leber Hereditary Optic Neuropathy (LHON) trials

Plain-language summary

Who can participate

Age range18 Years – 75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Age at the time of signing the informed consent form: the age of the subjects must be ≥ 18 years old and ≤ 75 years old Type of Subject and Disease Characteristics
✓. The clinical manifested vision loss due to LHON, and any eye BCVA ≥ 0.5 LogMAR
✓. The genotype testing result shows the presence of G3460A mutation in the ND1 gene, and the absence of the other primary LHON associated mutations in the mitochondrial DNA (mtDNA) (ND4 \[G11778A\] or ND6 \[T14484C\]) (confirmed by a CLIA-certified international laboratory)
✓. The vision loss in the eye with worse visual acuity lasted \> 6 months and \< 10 years at screening
✓. Pupils can be adequately dilated for a thorough ocular examination and visual acuity test
✓. Each eye of the subject must maintain at least Hand Motion visual acuity (VA) (≤ 2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for refraction and VA examinations) in this study
✓. Willingness to comply with the clinical study protocol and 5 years of long-term follow-up after administration Sex
✓. Male or female