EUCTR2013-001405-90-FR进行中（未招募）1 期
A phase I/IIa, non randomized, escalating dose, open-label study to evaluate safety and efficacy of GS010 (rAAV2/2-ND4) in patients suffering from Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 22
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4
- •2. Age 18 years old or older at the time of study entry (informed consent signature)
- •3. Visual acuity = 1/10 of the less functional eye
- •4. Visual acuity = 3/10 of the less functional eye for the patients of cohort 4 only
- •5. Sufficient viable retinal ganglion cells (RGC) as determined by optical coherence tomography (OCT): Retinal nerve fiber layer (RNFL) thickness estimation = 80 µm
- •6. No infection with Human Immunodeficiency Virus (HIV)
- •7. Negative pregnancy test in women of childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)
- •8. Female patients (if childbearing potency) must agree to use an effective methods of birth control during 6 months after the end of treatment
- •9. Affiliated to or a beneficiary of the French health care system
- •10. Written informed consent
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 22
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •11. Any known allergy or hypersensibility to one of the product used during the trial
- •12. Contraindication to IVT surgery (anaemia Hb <8g/dl, severe cardiovascular disease, severe coagulopathy…)
- •13. Disorder of the ocular humors and of the internal retina involving visual disability
- •14. Glaucoma
- •15. Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve
- •16. Vascular retinal occlusion
- •17. Narrow angle contra-indicating pupillary dilation
- •18. Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)
- •19. Patients presenting known mutation of other genes implicated in pathological retinal conditions
- •20. Patients treated by oral corticoids within 14 days prior inclusion at study entry.
- •21. Patients who had received Idebenone during the 2 months preceding the injection.
- •22. Participation in any other clinical trial on the eye within 3 months preceding the injection and during the study period
- •23. Patients who had eye surgery within the last 3 months preceding the inclusion
- •24. Patients suffering from any systemic illness or showing medically significant abnormal laboratory values at screening
- •25. Absence of vision on the other contralateral eye
- •26. Patients affected by pathologies for which the symptoms and/or the associated treatments can alter the visual function, for instance cancers or any pathology of the central nervous system
- •27. Diabetes patients with symptoms of retinopathy or macula oedema,
- •28. Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
- •29. Patient unable or unwilling to comply with the protocol requirements
研究者
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