NL-OMON53574招募中3 期
Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment ofStargardt Disease in Adolescent Subjects - LBS-008-CT03
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 10
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
- •1.Male or female subjects 12 to 20 years old, inclusive.
- •2.Subject must have clinically diagnosed STGD1 with at least 1 mutation
- •identified in the ABCA4 gene.
- •3.Ability to adequately examine the fundus of the study eye at enrollment.
- •a.The study eye will be the eye that meets all inclusion and exclusion
- •b.If both eyes meet all inclusion and exclusion criteria, the eye with the
- •smaller lesion will be selected to be the study eye.
- •c.If both eyes meet all inclusion and exclusion criteria and have lesions of
- •equal size, then the eye with the better BCVA will be selected to be the study
- •d.If both eyes meet all inclusion and exclusion criteria, have lesions of equal
- •size, and have equal BCVA, the default will be the right eye.
- •4.Subject must have a defined aggregate atrophic lesion size within 3 disc
- •areas (7.62 mm2) as imaged by FAF (DDAF) in the study eye and confirmed by the
- •central reading center. The lesion, or at least 1 focal lesion if multiple
- •lesions exist (multifocal atrophic lesions), must be in the macular region and
- •greater than 0.02 disc areas (0.05 mm2).
- •5. Subjects must present with BCVA of 20/200 or better for the study eye based
- •on ETDRS letter score. No minimum VA is required in the fellow eye.
- •6. Subject and their parent(s) or legal guardian are willing to provide their
- •consent on an Institutional Review Board (IRB)/Independent Ethics Committee
- •(IEC)/Human Research Ethics Committee (HREC)-approved informed consent form
- •(ICF) prior to participating in any study-related procedures.
- •7. Subject agrees to comply with all protocol requirements.
排除标准
- •1. Any ocular disease other than STGD1 at screening that, in the opinion of the
- •investigator, would complicate assessment of a treatment effect.
- •2. History of ocular surgery in the study eye in the last 3 months.
- •3. Investigational drug use of any kind in the last 3 months or within 5
- •half-lives of the investigational drug, whichever is shorter.
- •4. Any prior gene therapy.
- •5. Vitamin A deficiency as defined based upon plasma values less than 20 µg/dL
- •(=0.7 µmol/L).
- •6. Use of medications such as isotretinoin (13-cis-retinoic acid) or other
- •retinol modulators or derivatives that may impact the effect of the study drug
- •in the last 2 weeks or within the washout period of the medication, whichever
- •is longer, before beginning study treatment administration.
- •7. Unwilling to discontinue vitamin A or beta-carotene supplement use.
- •8. Use of any known drugs or supplements that are moderate or strong
- •inhibitors/inducers of cytochrome P450 (CYP) enzymes (e.g., rifampin,
- •barbiturates, phenothiazines, cimetidine, carbamazepine, St. John*s wort)
- •within 30 days of study drug administration or consumption of foods that are
- •moderate or strong inhibitors/inducers of CYP enzymes (e.g., grapefruit,
- •pomegranate, star fruit, bitter orange [Seville orange]) within 48 hours of
- •study drug administration, and that, in the investigator*s judgement, may
- •impact subject safety or the validity of the study results.
- •9. Presence of any life-threatening disease(s), including current treatment for
- •malignancies.
- •10. Alanine transaminase/aspartate aminotransferase >2.5 × the upper limit of
- •normal at screening.
- •11. Renal insufficiency, as defined by an estimated glomerular filtration rate
- •(Bedside Schwartz) <30 mL/min/1.73 m2 at screening.
- •12. Pregnant or nursing (breastfeeding) females; females of childbearing
- •potential who are unwilling or unable to use an acceptable method of
- •contraception (or abstinence). Females of childbearing age must have a negative
- •pregnancy test prior to randomization.
- •13. Male subjects who do not agree that female spouses/partners will use
- •adequate contraception (e.g., condoms) or be of nonchildbearing potential (ie,
- •surgically sterile).
- •14. Unwilling or unable to provide signed informed consent/assent.
- •15. In the opinion of the investigator, the subject is not suitable for entry
- •into the study.
研究者
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