A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome
研究概览
简要总结
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
详细描述
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.
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
- 17 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Definitive diagnosis of Wolfram syndrome
- •Insulin dependent diabetes mellitus due to Wolfram syndrome
- •At least 17 years of age
- •Participant must be willing to wear a CGM device for the duration of the study
排除标准
- •Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)
- •Any history of heart failure per New York Heart Association (NYHA)
- •History of or family history of breast and/or ovarian cancer
- •Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment
- •Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1
- •Previous treatment with gene or cellular therapy
研究组 & 干预措施
AMX0035
AMX0035 administered by mouth for 208 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant
干预措施: AMX0035 (Drug)
结局指标
主要结局
To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome
时间窗: 208 weeks
* Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments
To evaluate the effect of AMX0035 on residual beta-cell function by monitoring C-peptide levels during a 0-240 minutes mixed-meal tolerance test (MMTT)
时间窗: 24 weeks
* C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT * Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT
To assess the safety and tolerability of AMX0035 administered orally for up to 144 weeks in adult participants with Wolfram syndrome
时间窗: 148 weeks
* Incidence and severity of Adverse Events and Serious Adverse Events * Incidence of abnormalities in clinical laboratory assessments
次要结局
- To evaluate the effect of AMX0035 during a 0-240 minute MMTT(48 weeks)
- To assess the effect of AMX0035 on visual acuity(48 weeks)
- To evaluate the effect of AMX0035 on total daily insulin dose(24 weeks)
- To evaluate the effect of AMX0035 on glucose range(24 weeks)
- To evaluate the effect of AMX0035 on HbA1c levels(24 weeks and 48 weeks)