A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Tolerability of PBI-4050 and of Its Effects on the Inflammatory, Fibrosis, Diabetes and Obesity Biomarkers in Subjects With Alström Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Description and number of abnormal laboratory values and adverse events that are related to treatment.
研究概览
简要总结
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks, provided the subject signs informed consent.
详细描述
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome. Approximately 18 subjects will be enrolled. The duration of study participation is approximately 35 weeks for each subject and comprises of 9 on site visits and telephone contacts in between visits.
Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks (Extension Period [EP]), provided the subject signs informed consent. The extension period includes a further 3 on site visits and telephone contacts in between visits. The total duration of the study participation is extended to approximately 71 or 83 weeks. The Data Safety Monitoring Board (DSMB) will determine if the safety data continue to support treatment for an additional 36 or 48 weeks.
At the completion of the EP End of Treatment, subjects will be allowed to enrol in the Alström Rollover Study PBI-4050-CT-9-10 and continue ongoing study medication without any break in treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Subject has recent or on-going infection requiring systemic treatment with an anti-infective agent within 30 days before screening.
- •Subject has had at least two documented episodes of severe hypoglycaemia within 12 months before screening
- •Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- •Subject has alanine transaminase (ALT) or aspartate transaminase (AST) level ≥ 5 × upper limit of normal (ULN) at screening.
- •Subject is currently using weight loss medications at screening. Subjects may be re-screened after stopping the weight loss medication for a period of at least 5 half-lives.
- •Subject has used any moderate/potent inducer or inhibitor of CYP2C9 isozyme or strong inducer or inhibitor of cytochrome P450 (CYP) 3A isozyme within 30 days prior to the first study drug administration.
- •Subject has a history of chronic alcohol or other substance abuse as determined at screening.
- •Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at screening.
- •Subject has any condition that, in the investigator's opinion, is likely to interfere with study conduct and compliance
- •Subject has a history of an allergic reaction to PBI-4050 or any of its excipients.
研究组 & 干预措施
PBI-4050
Four 200 mg capsules (total 800 mg) administered orally, once daily.
干预措施: PBI-4050 (Drug)
结局指标
主要结局
Description and number of abnormal laboratory values and adverse events that are related to treatment.
时间窗: Primary on 24 weeks; Final on all data (including Extension Period)
次要结局
- Change from baseline in metabolic syndrome parameters over time.(24 weeks and end of Extension Period)
- Change from baseline in biomarkers in blood and urine over time(24 weeks and end of Extension Phase)
- Change from baseline in cardiac function parameter: NT-proBNP(24 weeks and end of Extension Phase)
- Change from baseline of antidiabetic treatment(24 weeks and end of Extension Phase)