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

RecruitingPhase 2, Phase 3Ages 12 Years - 75 YearsAllWuhan Neurophth Biotechnology Limited Company
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In plain words

What the trial is, and whether it could suit you

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 โ‰ฅ 18 years old and โ‰ค 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 โ‰ฅ 12 years and โ‰ค 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 t

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria Age 1. Age at signing of informed consent form 1. In Part 1, the age of the subjects must be โ‰ฅ 18 years old and โ‰ค 75 years old 2. In Part 2, the age of the subjects must be โ‰ฅ 12 years old and โ‰ค 75 years old, and the 6 evaluable subjects must be monitored for at least 6 weeks during the safety run-in phase. If SRC believes that there is no safety issue, the randomized double-blind control study will be initiated Subject Type and Disease Characteristics 2. The clinical manifestation caused by LHON is vision loss, with a visual acuity of โ‰ฅ 0.5 LogMAR in BCVA in either eye 3. 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) 4. The duration of vision loss in the eye with worse visual acuity lasted \> 6 months and \< 10 years at screening 5. Pupils can be adequately dilated for a comprehensive eye examination and visual acuity test 6. Each eye of the subject must maintain the VA determined by manual visual acuity test (โ‰ค 2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for optometry and VA examinations) in this study 7. Sign the written informed consent form and willing to comply with the clinical study protocol Sex 8. Male or female 1. Male subjects: โ€ข A male subject must agree to take contraceptive measures at least 6 months after the treatment visit, see Appendix 5 for details 2. Female subjects: * A female subject is eligible to participate if she is not pregnant (see Appendix 5), not breastfeeding, and at least one of the following conditions applies: i) Not a woman of childbearing potential (WOCBP) as defined in Appendix 5 or ii) A WOCBP who agrees to follow the contraception guidance in Appendix 5 for at least 6 months after the treatment visit Informed Consent 9. Written informed consent form must be obtained from the subject or his/her parent/legal guardian (if the subject is under 18 years of age) (Part 2) before any study-related procedures are performed (see Section 10.2) If the subject is legally identified as blind (\>1.0 LogMARor decimal acuity meter reading \< 0.1), an impartial witness must be present throughout the informed consent process and discussion process. Exclusion Criteria Subjects who meet any of the following criteria will be excluded from the study: 1. Any known allergy and/or hypersensitivity to the study drug or its constituents 2. Contraindication to IVT injection in any eye 3. IVT drug delivery to any eye within 30 days prior to the screening visit 4. History of vitrectomy in either eye 5. Narrow anterior chamber angle in any eye contra-indicating pupillary dilation 6. 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 7. 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 8. 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 9. Presence of optic neuropathy from any cause other than LHON 10. 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 11. History of recurrent uveitis (idiopathic or immune-related) or active ocular inflammation 12. 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. 13. Any eye has previously received ocular gene therapy 14. Subjects who refused to stop using idebenone 15. Have undergone ocular surgery of clinical relevance (per investigator's assessment) within 90 days prior to the screening visit 16. Female subjects who are breastfeeding or plan to breastfeed within the first 6 months after the administration of NR082 Injection 17. History of drug or alcohol abuse (including heavy smoking, i.e. \> 20 cigarettes per day or \> 20 pack-years \[equivalent to one pack a day for 20 years or 2 packs a day for 10 years\]) 18. 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 19. Unable to tolerate or unable or unwilling to comply with all the protocol requirements 20. 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 21. Any other exclusions determined by the investigator
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What it tests

  • NR082 injection
  • Sham Injection
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Where

1 site
Beijing Tongren Hospital, Capital Medical University
Beijing, China
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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