A Randomized, Double-blind, Crossover, Placebo-controlled Phase IIa Clinical Study Evaluating the Efficacy and Safety of Recombinant Acylated Glucagon Like Peptide-2 Analog (FT1) for Injection in Patients With Short Bowel Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Treatment-related Adverse Events
研究概览
简要总结
The goal of this clinical trial is to learn if FT1 is safe and works to treat short bowel syndrome (SBS) in adults. It will also learn about the PK/PD profile of FT1.
Researchers will compare FT1 to a placebo (a look-alike substance that contains no drug) to see if FT1 is safe and effective in patients with SBS.
Participants will
- Receive multiple injections of FT1 or placebo according to weight.
- Visit the clinic for assessment.
详细描述
In this placebo-controlled, double-blind, randomized, crossover phase 2 trial, 8 adult SBS patients are treated with once-weekly FT1 or placebo (1:1) for 5 weeks, followed by a washout period of at least 6 weeks, and then the alternate treatment for a further 5 weeks.
Efficacy is evaluated through a 72 hour metabolic balance study conducted at baseline and at the end of each treatment cycle.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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
- •Age ≥ 18 years, male or female.
- •SBS secondary to surgical resection of the small intestine, screened for at least 12 months after pre intestinal resection surgery;
- •Stable weight before screening; For patients requiring parenteral support (PS), PS volume remained stable (with changes in volume or energy content<25%) within 14 days prior to randomization;
- •Willing to undergo colonoscopy and remove polyps assessed by researchers to be at risk of cancer;
- •During the trial period, there were no plans to perform any major abdominal surgeries (such as intestinal resection exceeding 10% or surgeries that alter intestinal anatomy, such as stoma surgery);
- •During the baseline metabolic balance study, the average daily fecal wet rearrangement amount was ≥ 800g;
排除标准
- •Having undergone major abdominal surgery (such as intestinal resection exceeding 10%) within the past 6 months prior to screening;
- •History of clinically significant intestinal adhesions and/or chronic abdominal pain;
- •History of persistent radiation enteritis, celiac disease, refractory diarrhea, etc;
- •Patients with malignant tumors within the past 5 years (excluding fully treated cervical carcinoma in situ, basal cell or squamous cell carcinoma of the skin, local prostate cancer after radical surgery, and ductal carcinoma in situ of the breast after radical surgery);
- •History of gallstones in the past 3 years, except for those who have undergone cholecystectomy for gallstones; Acute cholecystitis or biliary obstruction related diseases that have not been treated within the previous month or during the screening period;
- •IBD patients with active inflammatory bowel disease (IBD), or requiring increased or altered immunosuppressive therapy in the past 3 months, or receiving biologic therapy in the past 6 months;
- •Occurrence of central venous catheter-related bloodstream infections within 2 months prior to and during the screening period;
- •Patients diagnosed with decompensated heart failure (NYHA grade III or above) and/or unstable angina and/or myocardial infarction from 6 months prior to screening until the first administration of the study drug;
- •Screening for individuals with rectal bleeding within the first 3 months;
- •Individuals with absorption instability caused by cystic fibrosis, untreated megacolon disease, or known DNA abnormalities (such as familial adenomatous polyposis, Fanconi syndrome);
- •Serious active, uncontrolled, untreated, acute onset systemic diseases (such as cardiovascular, respiratory, renal, infectious, endocrine, liver or central nervous system, etc.);
- •Pregnant or breastfeeding women.
- •The investigator believes the subject is unsuitable for participating in this clinical study.
结局指标
主要结局
Treatment-related Adverse Events
时间窗: From the first administration to study completion, appropriately 5 months.
To evaluate the adverse events as characterized by type, frequency, severity as graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 6.0, timing, seriousness, and relationship to study therapy after administration.
Changes in fecal wet weight from baseline to the end of treatment
时间窗: At the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles)
The difference in changes in fecal wet weight in the 72-hour metabolic balance study after treatment compared to baseline
次要结局
- Changes in urine volume from baseline to the end of treatment(At the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles))
- The Area Under the Curve from dosing to the time of the last measured concentration (AUC0-t)(Up to 8 days, from Day 29 (the last dose administration) to Day 36 (7 days after the last dose) in each treatment cycle (each cycle is 5 weeks))
- Maximum plasma concentration (Cmax)(Up to 8 days, from Day 29 (the last dose administration) to Day 36 (7 days after the last dose) in each treatment cycle (each cycle is 5 weeks))
- Changes in L-citrulline levels in plasma(From Day 1 of the first cycle to the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles))