Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated W… (NCT05820152) | Clinical Trial Compass
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 range
18 Years – 75 Years
Sex
ALL
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Willingness to comply with the clinical study protocol and 5 years of long-term follow-up after administration Sex
. Male or female
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 spectral-domain optical coherence tomography (SD-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