Leber’s Hereditary Optic Neuropathy
Conditions
Interventions
Test group:Idebenone tablets 150mg tid
Control group:placebo 150mg tid
Sponsors
Chinese People's Liberation Army General Hospital
Eligibility
Sex/Gender
All
Age
14 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Age at the time of signing the informed consent form: The age of 33 subjects was >=18 years old and monitored for at least 4 weeks. If the Independent Data Monitoring Committee (IDMC) believes that there is no safety issue, the age of continued enrollment can be relaxed to >=14 years old; 2. Written informed consent must be obtained from the subject or his guardian (if the subject is under the age of 18) before any research-related procedures; if the minor subject or his guardian lacks reading ability or is an adult If the subject is legally blind (>1.0 LogMAR), an impartial witness must be present throughout the informed consent process and discussion; 3. Able to comply with the schedule of visits, treatment regimens, laboratory tests, and other requirements of the study; 4. The mtDNA mutation detection belongs to any one of m.G11778A, m.T14484C, and m.G3460A; 5. Diagnosed with Leber's hereditary optic neuropathy (LHON) with involvement of the second eye for <= 1 year; 6. Able to adequately perform pupil dilation for a comprehensive ophthalmological examination and visual acuity examination; 7. Subjects (including male subjects) have taken effective contraceptive measures within 14 days before screening and agreed to have no reproductive plan and voluntarily take effective contraceptive measures from the time of signing the informed consent to 6 months after the last dose.
Exclusion criteria
Exclusion criteria: 1. Allergy history: those who are known to be allergic to idebenone and its components or coenzyme Q10 analogs; 2. Disease history: (1) There are uncontrollable clinical problems (such as severe heart, cerebrovascular, liver, kidney, digestive tract, immune, blood, endocrine, metabolic and mental and/or psychological, etc.); (2) Any ocular disease or history other than LHON that may affect vision (such as uveitis, iritis, diabetic retinopathy, etc.); (3) Those suffering from any other diseases that cause optic nerve damage or vision loss, including but not limited to intracranial tumors, optic neuritis, multiple sclerosis, ischemia, radiation, toxicity and other optic nerve diseases; (4) Suffering from systemic immune diseases; (5) Those who currently have serious infectious diseases requiring systemic treatment; (6) Those who have received eye surgery in the past or have any history of disease/surgery that affects drug absorption, distribution, metabolism, and excretion within 3 months before screening; 3. Medication or treatment history: (1) Those who have received or plan to receive gene or stem cell therapy for LHON in the past; (2) Those who have received LHON-related treatments from the time of signing the informed consent to the first medication, such as coenzyme Q10 or idebenone tablets or mecobalamin tablets, vitamin B12, traditional Chinese medicine, acupuncture and other treatments; 4. Combined with severe liver and kidney dysfunction, such as bilirubin>1.5*ULN, ALT or AST>2*ULN, creatinine>1.5*ULN, alkaline phosphatase>2.5*ULN; 5. Breastfeeding or positive serum pregnancy test; 6. Hepatitis B patients (HbsAg positive and HBV-DNA indicates virus replication), hepatitis C patients (HCV positive and HCV-RNA indicates virus replication), positive for syphilis screening (positive for specific antibodies, negative for non-specific antibodies and combined with Except for those with clinically judged inactive infection), known HIV positive history or HIV screening positive; 7. Those who smoked more than 5 cigarettes per day on average one month before enrollment, or who did not agree to smoke less than 5 cigarettes per day during the trial; 8. Alcoholics or excessive drinkers (drinking more than 14 units of alcohol per week: 1 unit = 285mL of beer, or 25mL of spirits, or 100mL of wine); 9. Other situations that the researcher deems unsuitable to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| BCVA outside the standard; | — |
Countries
China
Contacts
Public ContactShihui Wei
Chinese PLA General Hospital
Outcome results
None listed