EUCTR2021-003253-36-DE进行中（未招募）1 期
Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 90
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
- 性别
- All
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 criteria.
- •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 eye.
- •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 (Fleckenstein et al, 2011; Sunness et al, 2007). 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 90
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 15
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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 (eg, 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 (eg, 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 m2at 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 (Appendix 13.5).
- •13. Male subjects who do not agree that female spouses/partners will use adequate contraception (eg, condoms) or be of nonchildbearing potential (ie, surgically sterile) (Appendix 13.5).
- •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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