EUCTR2015-001265-11-IT进行中（未招募）1 期
Sperimentazione clinica pilota, randomizzata, in doppio cieco, controllata con simulazione per valutare l'efficacia di un'unica iniezione intravitreale di GS010 (rAAV2/2-ND4) in soggetti affetti, per un periodo di 6 mesi o inferiore, da neuropatia ottica ereditaria di Leber a causa della mutazione G11778A nel gene mitocondriale NADH deidrogenasi 4 - RESCUE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 39
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
- •Selection criteria:
- •1 Age 15 years or older
- •2 Onset of vision loss based on medically documented history or subject testimony, in at least one eye for =180 days in duration and if both eyes are affected the duration of vision loss in both eyes must be = 180 days in duration
- •3 Each eye of the subject maintaining visual ability to allow at least for counting of the examiner's fingers at any distance
- •4 Female subjects (if of childbearing potential) must agree to use effective methods of birth control up to 6 months after IVT injection and male subjects must agree to use condoms for up to 6 months after IVT injection
- •5 Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing
- •6 Signed written informed consent
- •Inclusion Criteria:
- •Subjects included in the study must satisfy all the following criteria at the Inclusion Visit (Visit 2).
- •1 Documented results of genotyping showing the presence of the G11778A mutation in the ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6) in the subject's mitochondrial DNA
- •2 Review of all selection criteria to ensure continued compliance. For the selection criterion n.2, the duration of the loss vision has to be calculated only on the basis of the date of the Visit 1.
- •3 Have a negative test for infection with human immunodeficiency virus (HIV)
- •4 Have a negative pregnancy test for women of childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)
- •Are the trial subjects under 18? yes
- •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 38
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •non selection criteria:
- •Subjects who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study
- •1 Any known allergy or hypersensitivity to GS010 or its constituents
- •2 Contraindication to IVT injection
- •3 IVT drug delivery to either eye within 30 days prior to the Screening Visit (Visit 1)
- •4 Previous vitrectomy in either eye
- •5 Narrow angle in either eye contra-indicating pupillary dilation
- •6 Presence of disorders of the ocular media, such as the cornea and lens, which may interfere with visual acuity and other ocular assessments during the study period
- •7 Vision disorders, other than LHON, involving visual disability or with the potential to cause further vision loss during the trial period.
- •8 Causes of optic neuropathy other than LHON and glaucoma.
- •9 Subjects with known mutations of other genes involved in pathological retinal or optic nerve conditions
- •10 Presence of ocular or systemic disease, other than LHON and well-controlled glaucoma, whose pathology or associated treatments might affect the retina or the optic nerve
- •11 History of amblyopia associated with a Snellen visual acuity equivalent of worse than 20/80 (equivalent to 6/24 at 6 meters, decimal acuity 0.25, LogMAR +0.6) in the affected eye
- •12 Presence of ocular conditions, which in the opinion of the Investigator will prevent good quality SD-OCT imaging from being obtained
- •13. Presence, in either eye, of uncontrolled glaucoma, defined as an IOP greater than 25 mmHg, despite maximal medical therapy with IOP lowering agents
- •14 Active ocular inflammation or history of idiopathic or autoimmune associated uveitis
- •15 Subjects participating in another clinical trial and receiving an IMP within 90 days prior to the Screening Visit (Visit 1)
- •16 Previous treatment with an ocular gene therapy product
- •17 Subjects who have undergone ocular surgery of clinical relevance (per Investigator opinion) within 90 days preceding the Screening Visit (Visit 1)
- •18 Female Subjects who are or who intend to breast feed during the trial period
- •Subjects who meet at least one of the following criteria at the Inclusion Visit (Visit 2) will not be included in the study
- •1 Any non-selection criteria which may have appeared after the screening visit
- •2 Subjects taking idebenone who have not completely discontinued the idebenone at least 7 days prior to Visit 2. If the subject has not discontinued idebenone at least 7 days prior to Visit 2, the visit may be delayed until the 7-day period is complete
- •3 Presence, at the time of study inclusion, of infectious conjunctivitis, keratitis, scleritis or endophthalmitis in either eye
- •4 Presence of systemic illness, including alcohol and drug abuse (except nicotine), or medically significant abnormal laboratory values that are deemed by the Investigator to preclude the subject's safe participation in the study
- •5 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 central nervous system
- •6 Any medical or psychological condition that, in the opinion of the Investigator, may compromise the safe participation of the subject in the study or would preclude compliance with the study protocol or ability of the subject to successfully complete the study
- •7 Subjects unable or unwilling to comply with the protocol requirements
研究者
相似试验
进行中（未招募）
1 期
A Phase III gene therapy clinical trial in LHON Subjects Affected for more 7 monthsEUCTR2015-001266-26-ITGENSIGHT BIOLOGICS40
进行中（未招募）
1 期
Ensayo clínico piloto, aleatorizado, controlado con placebo, paralelo, doble ciego, para evaluar el efecto de salbutamol intravenoso sobre marcadores de inflamación en pacientes con síndrome de distrés respiratorio agudoEUCTR2005-005951-16-ESDr. Joan Ramon Masclans. Servicio de Medicina Intensiva. Hospital Universitario Vall d'Hebron
进行中（未招募）
1 期
Ensayo clínico piloto, aleatorizado, controlado con placebo, paralelo, para evaluar la eficacia y seguridad de la tobramicina inhalada, en el paciente afecto de bronquiectasias, sin fibrosis quística e infección bronquial intermitente por Pseudomonas aeruginosa.EUCTR2005-005820-15-ESServicio de Neumologia. Hospital Universitario Vall d'Hebron35
进行中（未招募）
不适用
Ensayo clínico piloto aleatorizado, para evaluar la eficacia y tolerabilidad de quetiapina de liberación prolongada en comparación con la de amitriptilina en el tratamiento de pacientes con fibromialgiaFibromialgiaEUCTR2008-003694-41-ESniversidad de Granada
已完成
不适用
Do customised fit shoes increase physical activity in children with Down syndrome?Physical activity in Down syndromeFoot health in Down syndromePhysical Medicine / Rehabilitation - Other physical medicine / rehabilitationHuman Genetics and Inherited Disorders - Down's syndromeACTRN12616001118493Shannon Munteanu30