NCT00742092已完成2 期
Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
Actelion1 个研究点 分布在 1 个国家目标入组 11 人开始时间: 2008年8月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- The sum of responses in nasal potential difference (NPD) after perfusion with isoproterenol and chloride-free buffer (TCS: Total Chloride Secretion), in the presence of amiloride.
研究概览
简要总结
Single Center, Double-Blind, Randomized, Placebo-Controlled, Two-Period/Two-Treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 12 years and older
- •Male or female Non-pregnant women who are to remain non-pregnant for 3 months after the end of the study. Women of childbearing potential must use a reliable method of contraception. Reliable methods of contraception for female patients include the following:
- •barrier type devices (e.g., female condom, diaphragm and contraceptive sponge) used ONLY in combination with a spermicide
- •intrauterine devices
- •oral contraceptive agent
- •Depo-Provera™ (medroxyprogesterone acetate)
- •levonorgestrel implants Abstention, the rhythm method or contraception by the partner alone are NOT reliable methods of contraception. A woman is considered to have child-bearing potential unless she meets at least one of the following criteria:
- •6 weeks post-surgical bilateral salpingo-oophorectomy or hysterectomy
- •Premature ovarian failure confirmed by a specialist gynecologist
- •Age > 50 years and not treated with any kind of HRT for at least 2 years prior to screening, and with amenorrhea for at least 24 consecutive months prior to screening and a serum FSH level of > 40 IU/L at screening.
- •Age > 55 years and treated with HRT prior to screening with an appropriate medical documentation of spontaneous amenorrhea for at least 24 months. For female patients in the pediatric age range, a reliable method of contraception must be considered, if appropriate.
- •Male patients accepting for the duration of the study and for 3 months thereafter to use a condom and not to procreate a child (not in case of azoospermia)
- •Cystic fibrosis patients homozygous for the F508del mutation as confirmed by genetic test
- •Signed informed consent prior to any study-mandated procedure
排除标准
- •Any condition prohibiting the correct measurement of the NPD such as upper respiratory tract infection
- •Acute upper respiratory tract or pulmonary exacerbation requiring antibiotic intervention within 2 weeks of screening
- •Severe renal impairment (creatinine clearance < 30 mL/min as per Cockroft and Gault)
- •Female patients of childbearing potential who will not undergo a pregnancy test prior to enrollment into the study
- •History of significant lactose intolerance
- •History of neuropathy
- •Presence of clinically significant diarrhea (> 3 liquid stools per day for > 7 days) without definable cause within 1 month prior to screening
- •Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease
- •FEV1 < 25% of predicted normal
- •Oxygen saturation at rest < 88%
- •Active or passive smoking as measured using the Smokelyzer®
- •Hypersensitivity to miglustat or any excipients
- •Planned treatment or treatment with another investigationaldrug or therapy (e.g., gene therapy) within 1 month prior to randomization
- •Breast-feeding, pregnant women or women who plan to become pregnant during the course of the study.
研究组 & 干预措施
1
Experimental
miglustat
干预措施: miglustat (Drug)
2
Placebo Comparator
placebo
干预措施: placebo (Drug)
结局指标
主要结局
The sum of responses in nasal potential difference (NPD) after perfusion with isoproterenol and chloride-free buffer (TCS: Total Chloride Secretion), in the presence of amiloride.
时间窗: Change from baseline (pre-dose on Day 1) to end-of-treatment (Day 8)
次要结局
- Change in basline nasal potential difference (NPD) response(From baseline (pre-dose on Day 1) to end-of-treatment)
研究者
研究点 (1)
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