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Leber Hereditary Optic Neuropathy (LHON)Phase 3NCT07406854

A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

A Phase 3 study testing rAAV2-ND4 in people with Leber Hereditary Optic Neuropathy (LHON), run by Wuhan Neurophth Biotechnology Limited Company at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 12 to 75 years
Conditions the study is for
Leber Hereditary Optic Neuropathy (LHON)
What is being tested?
rAAV2-ND4
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
95 participants
Where
1 siteChina
Study dates
19 Sep 2024to 30 May 2030
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
  • 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
Show all 19Show fewer
  • 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
  • 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
  • Any other exclusions determined by the investigator

Trial locations and contacts

1 site in 1 country.

China1 site

  • Beijing Tongren Hospital, Capital Medical University

    Beijing, Beijing Municipality

Other trials in China

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation

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.

Read the detailed description

Safety run-in phase:

The safety run-in phase will enroll 6 evaluable subjects aged ≥ 12 years and ≤ 75 years , namely 4.5 x 109 vg, 0.05 mL eye/dose（bilaterally） and monitor the safety for at least 6 weeks. If there is no new safety concern evaluated by the SRC, the randomized, double-blind, sham-injection control study can be initiated.

Second Stage: randomized, double-blind, sham-injection control study:

The randomized, double-blind, sham-injection control study is to verify the efficacy and safety of NR082 in LHON caused by mitochondrial gene ND4 mutation . This part is divided into the NR082 treatment group and the control group (sham-injection group).

Being tested
rAAV2-ND4, Sham (No Treatment)
Medical subject headings
Optic Atrophy, Hereditary, Leber

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • rAAV2-ND4Genetic

    a single bilateral intravitreal (IVT) injection of NR082

  • Sham (No Treatment)Other

    sham-injection

Study groups in NCT07406854
Study groupRoleWhat they receive
Experimental: NR082 injectionExperimental4.5E9 viral genomes (vg) , 0.05 mL eye/dose , bilaterally
Receives: Genetic: rAAV2-ND4
Sham Comparator: sham-injectionSham comparatorSham intravitreal injection （bilateral）will be performed by applying pressure to the eye at the location of a typical intravitreal injection procedure using the blunt end of a syringe without a needle.
Receives: Other: Sham (No Treatment)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Efficacy of NR082 in study eye

    Proportion of ≥ 0.3 LogMAR from baseline in BCVA in the study eye in the NR082 treatment and the sham-injection at Week 52 after treatment

    Measured: 52 weeks

Secondary outcomes (3)

  1. Efficacy of NR082 in study eye and non-study eye

    Mean change from baseline in BCVA;

    Measured: 52 weeks

  2. Mean change from baseline in visual field, contrast sensitivity

    Efficacy of NR082 in study eye and non-study eye

    Measured: 52 weeks

  3. Safety and tolerability of NR082

    Incidence rates of AEs, SAEs within 52 weeks after injection of NR082

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Feb 2026
  2. Study start19 Sep 2024
  3. Primary completion30 May 2026 (estimated)
  4. Study completion30 May 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Multicenter, Randomized, Double-Masked, Sham-Controlled
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
95 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07406854
Sponsor's protocol ID
NFS-01-201
Status last verified
Feb 2026
First submitted
4 Feb 2026
Last updated
12 Feb 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing rAAV2-ND4 in people with Leber Hereditary Optic Neuropathy (LHON), run by Wuhan Neurophth Biotechnology Limited Company at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Efficacy of NR082 in study eye (time frame: 52 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07406854.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2026.

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