EUCTR2019-004981-16-NL进行中（未招募）1 期
Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene (LCA10-IVS26”) - EDIT-101 Single Ascending Dose Study in Participants with LCA10-IVS26
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
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
- •Adult participants enrolling in Cohorts 1, 2, or 3 must be at least 18
- •years of age at the time of informed consent. Pediatric participants
- •enrolling in Cohorts 4 or 5 must be 3 to 17 years of age, inclusive, at the
- •time of informed consent.
- •CEP290-related retinal degeneration caused by a homozygous or
- •compound heterozygous mutation involving
- •c.2991+1655A>G in IVS26 of the CEP290 gene confirmed by DNA
- •sequencing (ie, 1 or 2 intron 26
- •c.2991+1655A>G mutations) and 100% match for both gRNA and PAM
- •Male or Female.
- •A sexually mature male participant must agree to use contraception as
- •detailed in Section 10.7 of this protocol
- •from the time of informed consent through at least 12 months after
- •study intervention, and to refrain from
- •donating sperm during this period.
- •A female participant is eligible to participate if she is not pregnant (has a
- •negative urine pregnancy result prior to study intervention), not
- •breastfeeding, and at least one of the following conditions applies: Not a
- •WOCBP as defined in Protocol Section 10.7, OR A WOCBP, or who
- •reaches childbearing potential during the study, who agrees to follow
- •the contraceptive guidance in Protocol Section 10.7 from the time of
- •informed consent through at least 12 months after study intervention.
- •The participant (or guardian, in the case of a minor) must provide
- •written informed consent prior to any study related procedures. Minors
- •must provide assent in accordance with country and local regulations, as
- •applicable.
- •Both eyes must be at least LP. The study eye will be the worse seeing
- •eye and must meet the following BCVA
- •Cohort 1: BWD, WFP, or LP
- •Cohorts 2 - 5: LP to 0.4 logMAR (20/50 Snellen equivalent). ). Note: The sentinel participant in each of these cohorts will have severe vision loss with a logMAR BCVA of =1.6 to 3.9 (20/800 or worse to LP) in the study eye. Subsequent participants in each cohort will have LP to 0.4 logMAR (20/50 Snellen equivalent) best-corrected visual acuity in the study eye.
- •If both eyes have the same BCVA but the worse seeing eye (as
- •determined by the participant and the examiner) does not meet the
- •above criteria, the better seeing eye may be designated as the study eye
- •as long as the participant agrees and the better seeing eye meets all
- •eligibility criteria.
- •Photoreceptor ONL identifiable in fovea by spectral domain OCT in the
- •Able, as assessed by the investigator, and willing to complete study
- •assessments and follow study instructions
- •for the duration of the study.
- •If currently enrolled in Study EDIT-NHS01 (the Natural History Study of
- •CEP290-Related Retinal
- •Degeneration), the participant must agree to complete the early
- •termination visit for that study, and be
- •withdrawn from that study, before enrolling in this study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 8
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Other known disease-causing mutations documented in the participant's
- •medical history or identified through
- •the retinal dystrophy gene panel evaluation performed at screening
- •(including but not limited to bi-allelic
- •mutations in other genes known to cause LCA) that, in the opinion of the
- •investigator, would interfere with the
- •potential therapeutic effect of the investigational product or the quality
- •of the assessments.
- •Achieves a passing score for the Visual Function Navigation course at the
- •maximum level of difficulty (ie, passes the most challenging Visual Function Navigation course under the
- •dimmest lighting conditions) with
- •either eye independently or both eyes together.
- •In either eye, cataract surgery in the last 3 months before the screening
- •In either eye, any active ocular/intraocular infection or inflammation
- •(such as blepharitis, infectious
- •conjunctivitis, keratitis, scleritis, endophthalmitis, idiopathic or
- •autoimmune-associated uveitis, or herpetic
- •lesions), assessed at screening.
- •In either eye, history of steroid-responsive intraocular pressure
- •increases such that the affected eye had a
- •pressure > 25 mm Hg following corticosteroid exposure despite topical
- •intraocular-pressure-lowering
- •pharmacologic therapy.
- •In either eye, Argus retinal implant.
- •In the study eye, absence of clear ocular media and adequate pupil
- •dilation, assessed at screening, to permit good quality OCT images.
- •In the study eye, presence of vitreous hemorrhage.
- •In the study eye, any history of rhegmatogenous retinal detachment.
- •In the study eye, spherical equivalent of the refractive error
- •demonstrating more than -8 diopters of myopia and more than +6
- •diopters of hyperopia (prior to cataract or refractive surgery), assessed
- •at screening.
- •Uncontrolled diabetes mellitus (hemoglobin A1c =10%) in the last 3
- •months before the screening visit or at
- •Active gastric ulcer at screening.
- •Use of systemic immunosuppressive medications for any chronic disease
- •in the last 3 months before the
- •screening visit, or during the screening period.
- •Any vaccination/immunization in the last 28 days before screening, or
- •during the screening period.
- •An inability or unwillingness to take the course of oral prednisone that is
- •required in this study.
- •Current enrollment in an investigational interventional drug or device
- •study (ocular or non-ocular) or
- •participation in such a study within 6 months before the screening visit.
- •(This does not include observational
- •studies. Prior or current participation in Study EDIT-NHS01 is not an
- •exclusion.)
- •Received prior gene therapy or oligonucleotide treatment of any kind.
- •The participant has a condition or is in a situation which, in the
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