NCT07087054招募中3 期
A Randomized, Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Paltusotine in Adults With Carcinoid Syndrome Due to Well-Differentiated Neuroendocrine Tumors
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 141
- 试验地点
- 85
- 主要终点
- Participants will record the number of flushing per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing flushing episodes.
研究概览
简要总结
A Phase 3, randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of paltusotine treatment vs placebo as well as the long-term safety of paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The purpose of this study is to continue the evaluation of the safety, efficacy, and pharmacokinetics (PK) of paltusotine in participants with carcinoid syndrome.
详细描述
This is a global, randomized, parallel-group, placebo-controlled study to evaluate the efficacy and safety of paltusotine in adults with carcinoid syndrome. The study includes a screening period of up to 11 weeks, a double-blinded randomized control period of 16 weeks, an open label extension period of 104 weeks, and a follow-up period of 4 weeks.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ≥18 years of age, at the time of Screening.
- •Willing and able to comply with the study procedures as specified in the protocol, including at least 70% compliance with the study diary for the 2-week period.
- •Documented carcinoid syndrome requiring medical therapy. Participants must exhibit symptoms of flushing with or without frequent BMs as follows:
- •For participants who are naïve/not currently treated with somatostatin receptors ligands (SRL), they must exhibit an average of >1 flushing episode/day over a period of 14 days
- •For participants who will wash out from SRL treatment, they must be symptomatically controlled and exhibit an increase in daily average flushing episodes and an average of >1 flushing episode/day over a period of 14 days during the Washout Period.
- •Evaluable documentation of locally advanced or metastatic histopathologically confirmed well-differentiated neuroendocrine tumor(s) [NETs].
- •No significant disease progression as assessed by the Investigator within the last 6 months before randomization.
排除标准
- •Diarrhea attributed to any condition(s) other than carcinoid syndrome.
- •Uncontrolled/severe diarrhea associated with significant volume contraction, dehydration, or hypotension.
- •Requires second line treatments (eg, telotristat) for control of carcinoid syndrome symptoms in the opinion of the Investigator.
- •Treatment with specific NET therapy <4 weeks before Screening (such as everolimus or sunitinib) or hepatic embolization, radiotherapy, peptide receptor radionuclide therapy (PRRT), and/or tumor debulking <12 weeks before Screening.
- •Major surgery within 8 weeks before Screening.
- •History of another primary malignancy <3 years prior to the date of randomization, except for adequately treated basal or squamous cell carcinoma of the skin, cancer of the breast has been completely locally excised and carcinoma in situ of the cervix has also been resected, previously treated malignancy, if all treatment for that malignancy was completed at least 3 years prior to first dose of study treatment, and no current evidence of disease, concurrent malignancy determined to be clinically stable and not requiring treatment.
- •Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry.
- •Poorly controlled diabetes mellitus defined as having a hemoglobin A1c (HbA1c) ≥8.5%
- •Unable to administer short-acting (SA) octreotide (octreotide acetate injection), or prior nonresponse documented with somatostatin agonists.
- •Clinically significant concomitant disease or indicator of disease that is not a result of the primary disease under study, including but not limited to cardiovascular disease, estimated glomerular filtration rate 2×upper limit of normal [ULN], and/or total bilirubin (TB) >1.5×ULN. (Participants with previously diagnosed Gilbert's syndrome not accompanied by other hepatobiliary disorders and associated with TB
- •Alcohol or drug abuse is not permitted at any time during the study
- •Diagnosed with symptomatic cholelithiasis
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
Paltusotine 80 mg daily
Experimental
干预措施: Paltusotine (Drug)
结局指标
主要结局
Participants will record the number of flushing per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing flushing episodes.
时间窗: Measured at Week 12
Treatment group difference of change from baseline to Week 12 in flushing episodes/day averaged over the 14 days prior to Week 12.
Participants will record the number of flushing per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing flushing episodes.
时间窗: Measured at Week 12
Treatment group difference of change from baseline to Week 12 in flushing episodes/day averaged over the 14 days prior to Week 12.
次要结局
- Participants will record the number of bowel movements (BMs) per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing BMs/day.(Measured at Week 12)
- Participants will record the number of bowel movements (BMs) per day in a daily diary to assess the efficacy of paltusotine vs placebo in reducing BMs/day.(Measured at Week 12)
研究者
研究点 (85)
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