NCT00537602终止2 期
Single Center, Double-blind, Randomized, Placebo-controlled, 2-period/2-treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the ΔF508 Mutation
Actelion1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2007年11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
- 终止
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride
研究概览
简要总结
Cystic fibrosis is a genetic disease caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR). The purpose of the study is to investigate the effects of miglustat on CFTR function in cystic fibrosis patients.
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: only women who are surgically sterile, who are in the menopause (no menstruation for at least one year) or those of childbearing potential who are using 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.
- •For children, a reliable method of contraception must be considered, if appropriate.
- •Accepting for the duration of the study and for 3 months thereafter to use a condom and not to procreate a child (males only)
- •Cystic fibrosis patients homozygous for the ΔF508 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 who will not undergo a pregnancy test prior to enrollment in the study
- •History of significant lactose intolerance
- •History of neuropathy
- •History of cataracts or known increased risk of cataract formation
- •Presence of clinically significant diarrhea (>3 liquid stolls per days for >7 days) without definable cause within 1 month prior to screening
- •Any known factor of disease that might interfere with treatment compliance, study conduct or interruption of the results such as drug or alcohol dependence or psychiatric disease
- •FEVI <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 investigational drug or therapy (e.g., gene therapy) within 1 month prior to randomization
研究组 & 干预措施
A
Experimental
Oral miglustat capsules 200 mg t.i.d. for 1 week and a single 200 mg dose on day 8
干预措施: miglustat (Drug)
B
Placebo Comparator
Oral placebo capsules matching in appearance miglustat capsules given t.i.d. for 1 week and a single dose on day 8
干预措施: placebo (Drug)
结局指标
主要结局
Change in nasal potential difference (NPD) in response to isoproterenol in chloride-free buffer in the presence of amiloride
时间窗: Baseline (pre-dose on day 1) to end-of-treatment (day 8)
次要结局
- Change in baseline NPD response(Baseline to end-of-treatment)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
1 期
Safety and Efficacy of Inhaled OligoG CF-5/20 for the Treatment Cystic FibrosisCystic FibrosisNCT00970346AlgiPharma AS26
已完成
2 期
Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D MutationCystic FibrosisNCT01161537Vertex Pharmaceuticals Incorporated13
已完成
4 期
Efficacy of Intensive Cholecalciferol Monitoring and Supplementation on Serum vitD Levels in Pediatric Patients With CFVitamin D DeficiencyBone Density, LowInflammation ChronicCystic Fibrosis Pulmonary ExacerbationCystic FibrosisCystic Fibrosis in ChildrenNCT05276960Hospital Infantil de Mexico Federico Gomez40
已完成
2 期
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis PatientsCystic FibrosisNCT00499837Kamada, Ltd.21
已完成
2 期
Repeated Application of Gene Therapy in CF PatientsCystic FibrosisNCT01621867Imperial College London130