Actively Recruiting
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
Led by Wuhan Neurophth Biotechnology Limited Company · Updated on 2022-11-14
102
Participants Needed
1
Research Sites
349 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 75 years for Part 1; age between 12 and 75 years for Part 2
- Vision loss caused by LHON with best corrected visual acuity (BCVA) of 0.5 LogMAR or worse in either eye
- Confirmed G11778A mutation in the ND4 gene with no other primary LHON mutations, verified by a certified laboratory
- Duration of vision loss in the worse eye between more than 6 months and less than 10 years
- Pupils can be dilated adequately for eye examination and visual acuity testing
- Each eye must have visual acuity of 2.3 LogMAR or better
- Ability and willingness to sign informed consent and comply with study protocol
- Male or female participants; males must agree to contraceptive measures for at least 6 months after treatment
- Female participants must not be pregnant, not breastfeeding, and either not of childbearing potential or agree to contraception for at least 6 months after treatment
You will not qualify if you...
- Known allergy or hypersensitivity to the study drug or its components
- Contraindication to intravitreal injection in either eye
- Intravitreal drug delivery within 30 days before screening
- History of vitrectomy in either eye
- Narrow anterior chamber angle preventing pupil dilation
- Eye disorders other than LHON that can interfere with visual or ocular assessments
- Known mutations causing optic nerve or retina disease other than LHON
- Systemic or ocular diseases causing vision loss other than LHON
- Optic neuropathy from causes other than LHON
- Illness or disease that may alter visual function or affect study safety, including certain cancers and central nervous system diseases
- History of recurrent or active ocular inflammation
- Participation in another clinical trial with investigational product within 90 days prior to screening (with specific exceptions for idebenone)
- Previous ocular gene therapy in any eye
- Refusal to stop idebenone use
- Relevant ocular surgery within 90 days before screening
- Female participants who are breastfeeding or plan to breastfeed within 6 months after treatment
- History of drug or alcohol abuse or heavy smoking
- Positive for HIV, syphilis, hepatitis C antibodies, or active hepatitis B infection
- Unable or unwilling to comply with study requirements
- Failure to comply with COVID-19 local guidelines
- Any other exclusion determined by the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Beijing Tongren Hospital, Capital Medical University
Beijing, China
Actively Recruiting
Research Team
X
Xiaoning Guo, PHD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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