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New treatment option for Leber's Hereditary Optic Neuropathy (LHON)
Official title Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
ClinicalTrials.gov ID: NCT04912843
What this study is testing
What is NR082 injection?
NR082 injection is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for leber's hereditary optic neuropathy (lhon).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 75
You may be able to join if
- Age at signing of informed consent form
- In Part 1, the age of the people must be ≥ 18 years old and ≤ 75 years old
- In Part 2, the age of the people must be ≥ 12 years old and ≤ 75 years old, and the 6 evaluable people must be monitored for at least 6 weeks during...
- The clinical manifestation caused by LHON is vision loss, with a visual acuity of ≥ 0.5 LogMAR in BCVA in either 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...
You likely can't join if
- people 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
See the full eligibility criteria
Who can join
- Age at signing of informed consent form
- In Part 1, the age of the people must be ≥ 18 years old and ≤ 75 years old
- In Part 2, the age of the people must be ≥ 12 years old and ≤ 75 years old, and the 6 evaluable people must be monitored for at least 6 weeks during the safety run-in phase. If SRC believes that there is no safety...
- The clinical manifestation caused by LHON is vision loss, with a visual acuity of ≥ 0.5 LogMAR in BCVA in either 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...
- The duration of vision loss in the eye with worse visual acuity lasted \> 6 months and \< 10 years at screening
- Pupils can be adequately dilated for a comprehensive eye examination and visual acuity test
- Each eye of the subject must maintain the VA determined by manual visual acuity test (≤ 2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for optometry and VA examinations) in this study
- Sign the written informed consent form and willing to comply with the clinical study protocol Sex
- Male or female
- Male people:
- A male subject must agree to take contraceptive measures at least 6 months after the treatment visit, see Appendix 5 for details
- Female people:
- 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...
- 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...
What rules you out
- people 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...
- 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...
- 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: people who have completed the clinical study of idebenone as IP within 90 days prior to the screening...
- Any eye has previously received ocular gene therapy
- people 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 people 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])
- people with positive human immunodeficiency virus (HIV), syphilis and HCV antibodies are excluded; people who have clinically significant active infection requiring treatment as shown by hepatitis B test (defined as...
- Unable to tolerate or unable or unwilling to comply with all the protocol requirements
- people 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
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.