A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 32
- 试验地点
- 10
- 主要终点
- Hypoglycemia Episode Rate
研究概览
简要总结
The objective of the trial is to evaluate the efficacy and safety of dasiglucagon administered as a subcutaneous (SC) infusion in reducing hypoglycemia in children with CHI.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Months 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established and documented diagnosis of CHI based on standard of care
- •Experiencing ≥3 events of hypoglycemia per week (plasma glucose [PG] <70 mg/dL [<3.9 mmol/L]) according to the investigator's evaluation
- •Previously undergone near-total pancreatectomy or being treated with a non-surgical approach, having been evaluated as not eligible for pancreatic surgery
- •If somatostatin analogues or sirolimus are used, the therapy should be well established as judged by the investigator, especially when considering their biological half-life
排除标准
- •Previous administration of dasiglucagon
- •Known or suspected allergy to the trial drug or related products
- •Previous participation (randomization) in this trial
- •Circulatory instability requiring supportive medication or presence of pheochromocytoma
- •Requires exogenous insulin
- •Body weight of <4 kg (8.8 lbs.)
- •Documented HbA1c ≥7% subsequent to near-total pancreatectomy and within 6 months prior to screening
- •Known or suspected presence of significant central nervous system disease/injury such that in the investigator's opinion will affect trial participation
- •Use of systemic corticosteroids, e.g., hydrocortisone >20 mg/m2 body surface area or equivalent in the 5 days before screening
- •Use of anti-inflammatory biological agents, or other immune modulating agents in the 3 months prior to screening
- •Any clinically significant abnormality identified on echocardiogram that in the opinion of the investigator would affect the patient's ability to participate in the trial
- •Any recognized clotting or bleeding disorders
- •Has participated in an interventional clinical trial (investigational or marketed product) within 3 months of screening or 5 half-lives of the drug under investigation (whichever comes first), or plans to participate in another clinical trial.
研究组 & 干预措施
Standard of Care Only
4 weeks (Treatment Period 1) of standard of care only + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care
干预措施: Dasiglucagon (Drug)
Standard of Care + dasiglucagon
4 weeks (Treatment Period 1) + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care
干预措施: Dasiglucagon (Drug)
Standard of Care + dasiglucagon
4 weeks (Treatment Period 1) + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care
干预措施: Standard of Care (Other)
Standard of Care Only
4 weeks (Treatment Period 1) of standard of care only + 4 weeks (Treatment Period 2) of dasiglucagon treatment as an SC infusion starting at 10 µg/hr on top of standard of care
干预措施: Standard of Care (Other)
结局指标
主要结局
Hypoglycemia Episode Rate
时间窗: Baseline, Week 2-4 (Treatment Period 1)
Hypoglycemia episode rate, defined as average weekly number of hypoglycemic episodes (PG \<70 mg/dL \[3.9 mmol/L\]) during Weeks 2-4 as detected by self-monitored plasma glucose (SMPG). The Week 2-4 value is the average weekly number of hypoglycemic episodes across the last 3 weeks of the treatment period. The change from Baseline to Week 2-4 is also provided. The Baseline value is the average weekly number of hypoglycemic episodes during the 2-week baseline period.
次要结局
- Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia(Baseline, Week 2-4 (Treatment Period 1))
- Total Amount of Gastric Carbohydrates Administered(Baseline, Week 2-4 (Treatment Period 1))
- Percent Time in Hypoglycemia in Treatment Period 2(Baseline, Week 6-8 (Treatment Period 2))
- Rate of Gastric Carbohydrates Administrations to Treat Hypoglycemia in Treatment Period 2(Baseline, Week 6-8 (Treatment Period 2))
- Increase in Fasting Tolerance(Baseline, Week 2-4 (Treatment Period 1))
- Percent Time in Range 70-180 mg/dL(Baseline, Week 2-4 (Treatment Period 1))
- Total Amount of Gastric Carbohydrates Administered to Treat Hypoglycemia(Baseline, Week 2-4 (Treatment Period 1))
- Rate of Clinically Significant Hypoglycemia Episodes in Treatment Period 2(Baseline, Week 6-8 (Treatment Period 2))
- Clinically Significant Hypoglycemia Episode Rates(Baseline, Week 2-4 (Treatment Period 1))
- Extent of Hypoglycemia(Baseline, Week 2-4 (Treatment Period 1))
- Amount of Nightly Gastric Carbohydrates Administered(Baseline, Week 2-4 (Treatment Period 1))
- Percent Time in Hypoglycemia(Baseline, Week 2-4 (Treatment Period 1))
- Rate of Hypoglycemic Episodes(Baseline, Week 2-4 (Treatment Period 1))
- Rate of Hypoglycemic Episodes in Treatment Period 2(Baseline, Week 6-8 (Treatment Period 2))