A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Her… (NCT07406854) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation
China95 participantsStarted 2024-09-19
Plain-language summary
The objective of this clinical study is to evaluate the safety and efficacy of NR082 in the treatment of LHON caused by mitochondrial ND4 gene mutation. This study will enroll subjects aged ≥ 12 years old and ≤ 75 years old to receive a single bilateral intravitreal (IVT) injection of NR082 to evaluate safety and efficacy. The clinical manifestations of all subjects are to be reduced visual acuity caused by LHON associated with ND4 mutation, with laboratory test showing G11778A mutation and reduced visual acuity lasted for \>6 months and \<10 years.
Who can participate
Age range12 Years – 75 Years
SexALL
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Inclusion Criteria:
* Age at the time of signing the informed consent form: the age of the subjects must be ≥ 12 years old and ≤ 75 years old
* The clinical manifestation of all subjects is reduced visual acuity caused by LHON associated with ND4 mutation, while the reduced visual acuity lasted for \> 6 months and \< 10 years
* The clinical manifestation caused by LHON is vision loss, with a visual acuity of ≥ 0.5 LogMAR and ≤1.68 in BCVA in both eye The genotype test result is that there is G11778A mutation in ND4 gene, and there are no other primary LHON-associated mutations in the mitochondrial DNA (mtDNA) (ND1\[G3460A\] or ND6\[T14484C\]) (confirmed by a CLIA-certified international laboratory) Pupils can be adequately dilated for a comprehensive eye examination and visual acuity test
Exclusion Criteria:
* Any known allergy and/or hypersensitivity to the study drug or its constituents Contraindication to IVT injection in any eye
* IVT drug delivery to any eye within 30 days prior to the screening visit History of vitrectomy in either eye
* Narrow anterior chamber angle in any eye contra-indicating pupillary dilation
* Presence of disorders or diseases of the eye or adnexa, excluding LHON, which may interfere with visual or ocular assessments, including optical coherence tomography during the study
* Presence of known/documented mutations, other than the LHON-related mutation, which are known to cause pathology of the optic nerve, retina or afferent visual system
* Pres…
What they're measuring
1
Efficacy of NR082 in study eye
Timeframe: 52 weeks
Trial details
NCT IDNCT07406854
SponsorWuhan Neurophth Biotechnology Limited Company