NCT03440814已完成3 期
A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 127
- 试验地点
- 29
- 主要终点
- Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)
研究概览
简要总结
The purpose of this is study is to evaluate the effects of DCCR (diazoxide choline controlled release tablets) in children and adults with Prader-Willi syndrome.
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
- Triple (Participant, Care Provider, Investigator)
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
- •Provide voluntary, written informed consent (parent(s) / legal guardian(s) of patient); provide voluntary, written assent (patients, as appropriate)
- •Genetically-confirmed Prader-Willi syndrome and hyperphagic
- •In a stable care setting for at least 6 months prior to Visit 1
- •Caregiver must have been caring for the patient for at least 6 months prior to Visit 1
排除标准
- •Have participated in an interventional clinical study (i.e., investigational drug or device, approved drugs or device evaluated for unapproved use) within prior 3 months
- •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 30 days after study participation
- •Any other known disease and/or condition, which would prevent, in the opinion of the Investigator, the patient from completing all study visits and assessments required by the protocol
研究组 & 干预措施
DCCR
Experimental
75 - 450 mg DCCR
干预措施: DCCR (Drug)
Placebo
Placebo Comparator
75 - 450 mg placebo for DCCR
干预措施: Placebo for DCCR (Drug)
结局指标
主要结局
Hyperphagia Questionnaire (HQ-CT) Change From Baseline at Visit 7 (Week 13)
时间窗: Baseline to Visit 7 (Week 13)
Hyperphagia-related behaviors were assessed by the validated hyperphagia questionnaire for clinical trials (HQ-CT), an instrument designed to measure symptoms of food related preoccupations and behaviors that was completed by the caregiver. The HQ-CT consists of nine items with responses ranging from 0-4 units each (possible total score range: 0-36). The HQ-CT was assessed at Screening, Baseline (Visit 2), and approximately every 4 weeks post-dose at Week 4, Week 8, and Week 13. A decrease in score from baseline represented improvement.
次要结局
- Caregiver Global Impression of Change (GI-C) at Visit 7 (Week 13)(at Visit 7 (Week 13))
- Change in Fat Mass (kg) From Baseline at Visit 7 (Week 13)(Baseline to Visit 7 (Week 13))
- Clinical Global Impression of Improvement (CGI-I) at Visit 7 (Week 13)(at Visit 7 (Week 13))
研究者
研究点 (29)
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相关资讯
FDA Extends Review of Soleno Therapeutics' DCCR for Prader-Willi Syndrome- The FDA extended the review period for Soleno Therapeutics' DCCR (diazoxide choline) for Prader-Willi syndrome, setting a new PDUFA date of March 27, 2025.
- The extension was prompted by the FDA classifying recent information from Soleno as a major amendment to the New Drug Application (NDA).
- The FDA has not raised any concerns regarding the safety, efficacy, or manufacturing of DCCR during the review process.
- DCCR has been granted Priority Review, Breakthrough Therapy, Fast Track, and Orphan Drug designations, highlighting its potential to address unmet needs in PWS.last yearFDA Foregoes Advisory Committee Meeting for Soleno's DCCR in Prader-Willi Syndrome- The FDA will continue its review of Soleno Therapeutics' DCCR for Prader-Willi syndrome (PWS) without an advisory committee meeting.
- DCCR, a diazoxide choline extended-release tablet, aims to be the first approved therapy for hyperphagia in PWS patients.
- Phase 3 trial data suggested DCCR may alleviate hyperphagia and aggressive behaviors, though the primary endpoint was not met.
- A final decision on DCCR approval is expected by December 27, with priority review and orphan drug status already granted.last yearFDA Grants Priority Review to Soleno's DCCR for Prader-Willi Syndrome- The FDA has accepted Soleno Therapeutics' NDA for DCCR, granting Priority Review for treating hyperphagia in Prader-Willi Syndrome (PWS) patients aged four and older.
- A PDUFA target action date is set for December 27, 2024, with the FDA planning an advisory committee meeting to discuss DCCR's application.
- DCCR, an extended-release formulation of diazoxide choline, has shown promise in addressing hyperphagia and other PWS-related symptoms in clinical trials.
- The FDA's decision is supported by data from Phase 3 trials and an extension study, highlighting DCCR's potential to improve the lives of PWS patients and their families.2 years ago