NL-OMON53320已完成3 期
A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Acucela Inc.
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 12 至 64（—）
入选标准
- •Subjects who meet all of the following criteria at Screening and Baseline
- •(unless otherwise indicated) may be eligible for inclusion in the study:
- •1. Males or females, age * 16 years.
- •2. Clinical diagnosis of MA secondary to STGD in one or both eyes as determined
- •by the Investigator.
- •3. The subject must have 1 or more pathogenic mutation(s) of the ABCA4 (ATP
- •binding cassette subfamily A member 4) gene. If only one ABCA4 pathogenic
- •mutation is identified or if two ABCA4 pathogenic mutations that typically
- •occur on the same allele (ie, *in cis*) are identified, the subject must have a
- •typical STGD phenotype (at least one eye has flecks at the level of the retinal
- •pigmented epithelium [RPE] typically seen in STGD) and be approved by the
- •Sponsor. If 2 or more pathogenic mutations that do not typically occur on the
- •same allele are identified, a typical STGD phenotype and separate Sponsor
- •approval are not required. Segregation analysis is not required. The
- •pathogenicity of all mutations will be determined by the Sponsor working with
- •experts in ophthalmic genetics.
- •4. The study eye must meet the following criteria as determined by the central
- •reading center*s assessment of FAF imaging at Screening:
- •a. Total area of DDAF
- •i. If the lesion is unifocal: * 3.0 * 22.0 mm2 (~1.2 * 8.7 disc areas) in size.
- •ii. If the lesion is multifocal: * 1.0 * 22.0 mm2 (~0.4 * 8.7 disc areas) in
- •b. The entire lesion must be completely visualized on the macula-centered image
- •(Field 2 * Macula Image). The DDAF lesion must be able to be imaged in its
- •entirety, and all lesion borders must be * 300 microns from all image edges.
- •5. Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA letter score of * 25
- •letters (approximately * 20/320 Snellen) in the study eye at Screening.
- •6. Adequate clarity of ocular media and adequate pupillary dilation to permit
- •good quality imaging of macular atrophy in the study eye as determined by the
- •Investigator.
- •7. Able to reliably administer oral medication by self or with available
- •assistance.
- •8. Able and willing to provide written informed consent/assent
- •a. For subjects * 18 years of age: able and willing to provide written informed
- •consent before undergoing any study-related procedures.
- •b. For subjects * 16 and 18 years of age: able and willing to provide written
- •informed assent, and has a parent or legal guardian able and willing to provide
- •written informed consent for the minor before the subject undergoes any
- •study-related procedures. Where required by local regulations, both parents
- •must consent to the subject*s participation in the study, if both have legal
排除标准
- •Subjects will be excluded from participation in the study if they meet any of
- •the following criteria at Screening or Baseline (unless otherwise indicated):,
- •1. Macular atrophy associated with a condition other than STGD in either eye.,
- •2. DDAF with contiguous area of peripapillary atrophy in the study eye, as
- •determined by the reading center.
- •3. Mutation(s) in any of the following genes * elongation of very long chain
- •fatty acids-like 4 (ELOVL4), prominin 1 (PROM1), or peripherin 2
- •(PRPH2)/retinal degeneration slow (RDS) * determined by the Sponsor working
- •with experts in ophthalmic genetics to likely be disease-causing.
- •4. If tested, any mutation(s):
- •a. In a gene(s) encoding a visual cycle protein [e.g., retinal pigment
- •epithelium 65 (RPE65), lecithin:retinol acyltransferase (LRAT), retinol
- •dehydrogenase 12 (RDH12), RDH5, and retinaldehyde binding protein 1 (RLBP1)],
- •confirmed by the Sponsor working with experts in ophthalmic genetics to likely
- •be disease-causing. Testing for these mutations is not required.,
- •b. Associated with a non-STGD retinal dystrophy/degeneration, confirmed by the
- •Sponsor working with experts in ophthalmic genetics to likely be
- •disease-causing. Testing is not required.,
- •5. Presence in either eye of an active ocular disease that in the opinion of
- •the Investigator compromises or confounds visual function, including, but not
- •limited to, choroidal neovascularization, diabetic retinopathy, uveitis, other
- •macular diseases, or uncontrolled glaucoma/ocular hypertension.,
- •6. History of any intraocular or ocular surface surgery in either eye * 3
- •months prior to Screening., 7. Current or previous participation in an
- •interventional study to treat STGD using gene therapy or stem cell therapy.
- •8. Current or previous participation in a study to treat STGD using a vitamin A
- •derivative * 6 months prior to Screening.
- •9. Current or previous participation in a study to treat STGD using a
- •complement inhibitor * 6 months prior to Screening.
- •10. Known hypersensitivity to emixustat or any of the excipients in emixustat
- •tablets (ie, silicified microcrystalline cellulose, pregelatinized starch,
- •colloidal silicon dioxide, and stearic acid).,
- •11. Prohibited medications: Please refer to page 29 of the protocol.,
- •12. Any of the following laboratory abnormalities at Screening: Please refer to
- •page 30 of the protocol,
- •13. Participation in any study using an investigational drug within 30 days or
- •5 half-lives (of the investigational drug) of Screening.
- •14. Participation in any study of an interventional, investigational device
- •within 60 days of Screening.,
- •15. Anticipated participation during the study period in any study using an
- •investigational drug or an interventional, investigational device.
- •16. Presence of other medical or ophthalmic disease, physical examination
- •finding, or clinical laboratory finding that in the opinion of the Investigator
- •contraindicates the use of an investigational drug, places the subject at risk
- •by participating in the study, might interfere with the evaluation of the
- •efficacy or safety of emixustat, negatively impacts subject compliance with the
- •protocol, confounds the ability to interpret data from the study, or
- •jeopardizes the subject*s ability to complete the protocol.
- •17. Current or history of cancer (except for adequately
研究者
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