NCT04094948撤回2 期
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Stably Treated With Enzyme Replacement Therapy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Changes in 6 minute walk test (MWT) distance
研究概览
简要总结
The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD
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
- •Diagnosis of Pompe disease by blood Acid alpha-glucosidase (GAA) assay and GAA gene sequencing,
- •Age: 18+ years at enrollment,
- •Receiving enzyme replacement therapy (ERT) at a stable dose for >104 weeks,
- •FVC >15% of expected (supine).
- •Subjects are capable of giving written consent.
- •Able to walk at least 100 meters on the 6 minute walk test (6MWT) (with assistive devices permitted).
排除标准
- •Continuous invasive ventilation (via tracheostomy or endotracheal tube)
- •6MWT distance >90% of expected performance (% expected)
- •FVC >90% of expected (upright).
- •Clinically relevant illness within two weeks of enrollment including fever > 38.2o C, vomiting more than once in 24 hours, seizure, or other symptom deemed contraindicative to new therapy.
- •Chronic heart disease (Myocardial infarction, arrythmia, cardiomyopathy)
- •Tachycardia
- •History of seizure disorder
- •Hyperthyroidism
- •Pheochromocytoma
- •History of diabetes
- •History of hypersensitivity to β2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent),
- •Patients on a non-standard schedule for ERT; for example, weekly infusions as opposed to infusions every two weeks.
- •Treatment for asthma in the previous 12 months.
- •Renal insufficiency (elevated serum creatinine).
- •Having started respiratory muscle strength training in the last 6 months prior to study day 1 or having discontinued respiratory muscle strength training in the 6-month period preceding study day 1, or having started respiratory strength training greater than 6 months prior to study day 1 and unwilling to continue for the first year of study participation.
- •Received an investigational drug or participated in another interventional study within 90 days of Study Day
- •Anti-rhGAA IgG with sustained titer >1:25.600 for >6 months at time of enrollment.
- •The use of the following concommitant meds is prohibited during the study:
- •diuretics (water pill);
- •digoxin (digitalis, Lanoxin);
- •beta-blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal);
- •tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor);
- •Monoamine oxidase (MAO) inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or
- •other bronchodilators such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).
研究组 & 干预措施
clenbuterol
Experimental
The initial dose of clenbuterol will be 40 mcg per oral each morning for one week, followed by 40 mcg twice per day (BID) for the next 5 weeks until Week 6. If the 40 mcg BID per oral is well tolerated, the dose will be increased to 80 mcg each morning/40 mcg each evening for one week, followed by 80 mcg BID for the next 5 weeks until the Week 12 visit. If 80 mcg BID is tolerated at Week 12, the subject will continue on that dose until Week 52.
干预措施: Clenbuterol (Drug)
placebo
Placebo Comparator
Initially, one capsule each morning for one week, followed by one capsule BID for the next 5 weeks until Week 6. If tolerated, the dose will be increased to two capsules each morning and 1 capsule each evening for one week, followed by two capsules BID for the next 5 weeks until the Week 12 visit. If two capsules BID is tolerated at Week 12, the subject will continue on that dose until Week 52.
干预措施: Placebos (Drug)
结局指标
主要结局
Changes in 6 minute walk test (MWT) distance
时间窗: Baseline (week 0) through 52 weeks
次要结局
- Changes in pulmonary function tests: forced expiratory volume in 1 second (FEV1)(Baseline (week 0) through 52 weeks)
- Changes in pulmonary function tests: forced vital capacity (FVC)(Baseline (week 0) through 52 weeks)
- Changes in pulmonary function tests: maximum expiratory pressure (MEP)(Baseline (week 0) through 52 weeks)
- Changes in pulmonary function tests: Maximum inspiratory pressure (MIP)(Baseline (week 0) through 52 weeks)
- Changes in graded functional test: Gait, Stairs, Gower, Chair (GSCS)(Baseline (week 0) through 52 weeks)
- Changes in graded functional test: Quick Motor Function Test (QMFT)(Baseline (week 0) through 52 weeks)
- Changes in the concentration of alanine transaminase (ALT) in serum(Baseline (week 0) through 52 weeks)
- Changes in the concentration of creatine kinase (CK) in serum(Baseline (week 0) through 52 weeks)
- Changes in the concentration of urinary glucose tetramer (Glc4)(Baseline (week 0) through 52 weeks)
研究者
Dwight Koeberl, M.D., Ph.D.
Professor of Pediatrics
Duke University
研究点 (1)
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