An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 115
- 试验地点
- 28
- 主要终点
- Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
研究概览
简要总结
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
详细描述
115 PWS participants who completed clinical study C601 will be enrolled into the OLE Period. All participants in the Open Label Extension (OLE) Period will receive open-label DCCR. The actual number of participants eligible to enroll in the double-blind, placebo-controlled, randomized withdrawal (RW) period will be limited to those participants taking DCCR in the OLE Period at the time of the RW Period Visit 1 (Baseline/Randomization Visit).The treatment groups in the C602 RW Period are those participants randomized to receive DCCR and those participants randomized to receive Placebo. Participants will be randomized in a 1:1 ratio (DCCR:Placebo).
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Successful completion of clinical study C601
- •Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (subjects, as appropriate)
- •OLE Period Key
排除标准
- •Positive urine pregnancy test (in females of child-bearing potential) or females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 90 days after study participation
- •Any new disease, condition, or circumstance which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol (e.g., an anticipated change of care setting)
- •RW Period Key Inclusion Criteria:
- •Provide voluntary, written informed consent (parent[s] / legal guardian[s] of participant); provide voluntary, written assent (participants, as appropriate); this includes consent for randomization and potential treatment with placebo for up to 16 weeks
- •Currently participating in clinical study C602 and complete the OLE End of Treatment Visit procedures
- •RW Period Key Exclusion Criteria:
- •Positive urine pregnancy test (in females of child-bearing potential)
- •Females who are pregnant or breastfeeding, and/or plan to become pregnant or to breast-feed during or within 30 days after study participation
研究组 & 干预措施
OLE DCCR
75 - 525 mg DCCR
干预措施: DCCR (Drug)
RW DCCR
75 - 525 mg DCCR
干预措施: DCCR (Drug)
RW Placebo
75 - 525 mg Placebo for DCCR
干预措施: Placebo for DCCR (Drug)
结局指标
主要结局
Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported
时间窗: Baseline to end of OLE (up to 4 years)
Safety analyses will be conducted in all participants who receive at least one dose of DCCR. Adverse events will be described by type and level of severity.
Change from RW Period Baseline in HQ-CT Total Score
时间窗: RW Period Baseline to Week 16
Hyperphagia-related behaviors will be assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors. The HQ-CT consists of nine items with responses ranging from 0-4 (best to worst). Scores from 9 items will be summed for a possible total score range of 0-36.
次要结局
- Change in Body Fat Mass(Baseline to end of OLE (up to 4 years))
- Clinical Global Impression of Improvement (CGI-I)(RW Period Week 16)
- Clinical Global Impression of Severity (CGI-S)(RW Period Week 16)
- Assess the safety of DCCR by evaluating the incidence and severity of adverse events reported(through the end of the RW Period, 16 weeks)
- Change from Baseline in HQ-CT Total Score(Baseline to end of OLE (up to 4 years))