An Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of Immunomodulation With AT-1501 in Adults With Type 1 Diabetes Undergoing Islet Cell Transplant
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Safety- Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)
研究概览
简要总结
This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant.
详细描述
This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant. This study will also provide valuable data with respect to its potential additional uses in autoimmunity and solid organ transplant. This is a single arm open-label study and up to 6 participants will be recruited at a single center in Canada.
The objectives include:
- To assess the safety and tolerability of immunomodulation with AT-1501, in combination (AT+) with rabbit anti-thymoglobulin (ATG), etanercept and mycophenolate mofetil (MMF/EC-MPS) in adults with T1D undergoing islet cell transplant.
- To assess the efficacy of immunomodulation with AT-1501 in adults with T1D undergoing islet cell transplant.
The duration of treatment may vary from participant to participant and could be up to 20 months. Participants may receive up to 2 islet cell transplants.
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Men and women 18-65 years of age
- •A diagnosis of T1D ≥5 years with onset of disease at <40 years of age
- •Involvement in intensive diabetes management as directed by an endocrinologist or diabetologist with at least 3 clinical evaluations within the 12 months prior to Screening; using an insulin pump or multiple daily injection (MDI) insulin therapy; and, unable to achieve acceptable metabolic control because of the occurrence of severe hypoglycemia
- •At least 2 unexplained SHEs not secondary to a missed meal or dosing error, etc., in the 12 months prior to Screening
- •Glycosylated hemoglobin (HbA1c) level greater than 7% (53 mmol/mol) and less than 9.5% (80 mmol/mol) inclusive
- •Absence of stimulated C peptide (< 0.3 ng/mL) in response to a mixed meal tolerance test (MMTT) measured at 60 and 90 minutes after the start of consumption
- •Reduced awareness of hypoglycemia as defined by a Clarke Score [Clarke 1995] of 4 or more at the time of Screening, during the Screening period and within the last 6 months prior to the transplant
排除标准
- •Any previous transplant
- •HbA1c level less than 7% (53 mmol/mol)
研究组 & 干预措施
AT-1501 Single Arm
干预措施: AT-1501 (Drug)
结局指标
主要结局
Safety- Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)
时间窗: Accessed from date of transplant through 1 year post transplant for approximately 2 years
Incidence of adverse events
Efficacy- Insulin independence
时间窗: Days 75 , Day 365 post-first transplant, and final transplant and 1 year after discontinuation of AT-1501
Change in the proportion of participants that become insulin independent at Days 75 and 365 post-first, and final transplant
次要结局
- Efficacy- Graft failure(Day 365)
- Efficacy- Durability of insulin independence- long term(2 and 3 years after discontinuation of AT- 1501)
- Efficacy- HbA1c(Day 365 and free of serious hypoglycemic events from Day 28 to 365 post-first transplant)