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

Recruiting now · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Leber's Hereditary Optic Neuropathy (LHON)

In brief

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.

Key facts

Study ID

NCT04912843

Run by

Wuhan Neurophth Biotechnology Limited Company

People needed

102

Starts

2021-06-18

Expected to finish

2028-02-29

Last updated by the study team

2022-11-14

Who can join

Age: 12 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Subjects who meet any of the following criteria will be excluded from the study:
• 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 spectral-domain optical coherence tomography (FD-OCT), 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
• Presence of systemic or ocular/vision diseases, disorders or pathologies, other than LHON, known to cause or be associated with vision loss, or whose associated treatment(s) or therapy(ies) is/are known to cause or be associated with vision loss
• Presence of optic neuropathy from any cause other than LHON
• Presence of illness or disease that, in the opinion of the investigator, include symptoms and/or the associated treatments that can alter visual function, for instance cancers or pathology of the CNS, including multiple sclerosis (diagnosis of multiple sclerosis must be based on the 2010 Revisions to the McDonald Criteria) (Polman et al., 2011), and/or diseases or conditions that affect the safety of subjects participating in the study
• History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation
• Participated in another clinical study and receive IP within 90 days prior to the screening visit
• a) Exceptions: Subjects who have completed the clinical study of idebenone as IP within 90 days prior to the screening visit, and has completely discontinued idebenone at least 7 days prior to dosing are still eligible to participate in the study.
• Any eye has previously received ocular gene therapy
• Subjects who refused to stop using idebenone
• Have undergone ocular surgery of clinical relevance (per investigator's assessment) within 90 days prior to the screening visit
• Female subjects who are breastfeeding or plan to breastfeed within the first 6 months after the administration of NR082 Injection
• History of drug or alcohol abuse (including heavy smoking, i.e. > 20 cigarettes per day or > 20 pack-years [equivalent to one pack a day for 20 years or 2 packs a day for 10 years])
• Subjects with positive human immunodeficiency virus (HIV), syphilis and HCV antibodies are excluded; subjects who have clinically significant active infection requiring treatment as shown by hepatitis B test (defined as positive hepatitis B core antibody [HBcAb] or hepatitis B surface antigen [HBsAg], hepatitis B virus deoxyribonucleic acid (HBV-DNA) >1,000 copies /mL or >lower limit of quantitative detection with the local laboratory method) will be excluded
• Unable to tolerate or unable or unwilling to comply with all the protocol requirements
• Subjects from the study site fail to comply with or do not agree to comply with local and institutional guidelines for suspected 2019 novel coronavirus (COVID-19) infection/testing
• Any other exclusions determined by the investigator

Where it is running

• Beijing Tongren Hospital, Capital Medical University — Beijing, China (enrolling)

Full record on ClinicalTrials.gov

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