Phase 1/2, open label, dose-finding followed by 2-year extension study to evaluate safety and tolerability of Tinlarebant in adolescent subjects with Stargardt disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- RBP4 PTY LTD
- 入组人数
- 12
研究概览
简要总结
The following conclusions can be drawn based on the study results: - Tinlarebant is well tolerated with a favorable safety profile; the dose of 5 mg was found to be well tolerated among adolescent subjects with STGD1. - Results confirm the mechanism of action of tinlarebant in potently reducing plasma RBP4 and retinol levels. - The PK/PD profile of tinlarebant showed a clear correlation between the increase of tinlarebant in blood and decreased plasma RBP4. Levels of RBP4 remained suppressed during daily treatment over the 24 month study period and returned to approximately 90% of the baseline value following 28 days of drug cessation. - All study subjects showed only foveal involved QDAF lesions at baseline which are predictive of subsequent transition to atrophic (DDAF) lesions and loss of visual acuity. However, FAF imaging data showed no transition to DDAF lesions in 41.7% of subjects who received 5 mg tinlarebant and in the subject who received 2 mg tinlarebant, and no clinically significant loss of visual acuity, which suggests efficacy of tinlarebant treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 12 Years 至 18 Years（—）
- 性别
- All
入选标准
- •1. Male or female subjects aged 12 to 18 years old.
- •2. Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.
- •3. Ability to adequately examine fundus of study eye at enrolment.
- •4. Subject must have a defined aggregate atrophic lesion size.
- •5. Subjects must have a BCVA of 20/400 or better for the study eye based on ETDRS letter score.
- •6. Subject and their parent(s) or legal representative are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved Informed Consent Form (ICF) prior to participating in any study-related procedures.
- •7. The subject agrees to comply with all protocol requirements.
排除标准
- •1. Any ocular diseases other than Stargardt disease that will complicate assessments.
- •2. Ocular surgery in the study eye in the previous 3 months
- •3. Participated in any clinical study in the previous 3 months.
- •4. Use of retinol modulators or derivatives which may impact study drug effect
- •5. Recent use of drugs, supplements or eating foods that are moderate or strong inhibitors/inducers of cytochrome P450 enzymes and could be considered to impact subject safety or study results
- •6. Vitamin A deficiency.
- •7. Life-threatening or currently in treatment for malignancies
- •8. Abnormal ALT, AST and Creatinine test values.
- •9. Females must have a negative pregnancy test before dosing.
- •10. Both males and females, including their partners need to agree to have acceptable method of contraception.