NCT02921620撤回3 期
A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients
Protalix0 个研究点开始时间: 2017年7月1日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 主要终点
- IBSSS Part 1
研究概览
简要总结
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years. Patients must have diarrhea defined as ≥ 3 stools a day with an average consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient electronic diary and moderate to severe gastrointestinal symptoms as defined by the Irritable Bowel Symptom Severity Score (IBSSS) Part 1 average > 175 derived from at least two IBSSS assessments during screening period. Patients will receive intravenous infusions of PRX-102 1 mg/kg or placebo every two weeks for 6 months.
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
- 14 Years 至 45 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males, age 14-45 years, naïve to enzyme replacement therapy (ERT) or off ERT or off chaperone treatment for at least 6 months and negative for anti-PRX-102 antibodies
- •A documented diagnosis of Fabry disease: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN)
- •eGFR by CKD-EPI > 30 ml/min/1.73 m2
- •Moderate to severe gastrointestinal symptoms as defined by:
- •Average score of > 175 from at least two Irritable Bowel Symptom Severity Score (IBSSS) Part 1 assessments before randomization.
- •Average stool consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient diary during 2 weeks prior to randomization out of the 4 week of screening period and
- •≥ 3 stools a day with a consistency of ≥ 5 on the BSFS during the week before randomization.
- •Completed electronic BSFS diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
排除标准
- •Patients will be evaluated to rule out other gastrointestinal comorbidity than Fabry disease as responsible for the gastrointestinal symptoms by:
- •i. Medical History for non Fabry gastrointestinal comorbidity ii. Occult blood in stool iii. Stool culture for bacteria and parasites iv. Calprotectin in stool v. Sigmoidoscopy
- •Use of any kind of laxatives
- •Initiation of anti-diarrheal medications during the screening period
- •History of renal dialysis or transplantation
- •Use of, or change in dose of, angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) for less than 4 weeks prior to screening
- •Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- •Congestive heart failure NYHA Class IV
- •Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- •Known history of hypersensitivity to Gadolinium contrast agent
- •Known allergies to ERT
- •Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
结局指标
主要结局
IBSSS Part 1
时间窗: Every 2 weeks for 6 months
Irritable Bowel Syndrome Severity Score
次要结局
- Plasma Lyso-Gb3(Every 4 weeks for 6 months)
- Plasma Gb3(Every 4 weeks for 6 months)
- Urine Lyso-GB3(Every 6 weeks for 6 months)
- Stool frequency(After every bowel movement for 6 months)
- Body Weight(Every 2 weeks for 6 months)
- Frequency of pain medication use(Every 2 weeks for 6 months)
研究者
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