Phase 2 Study of Rasagiline for Treatment of Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 80
- 试验地点
- 10
- 主要终点
- ALS Functional Rating Scale-Revised (ALSFRS-R)
研究概览
简要总结
ALS is a disorder that weakens motor strength and lung function. Rapid loss of motor neurons in the brain and spinal cord of ALS patients causes the symptoms of increasing weakness and loss of muscle function. Motor neurons are responsible for sending signals to muscles in our bodies to trigger movement. While there are drugs to help relieve symptoms of ALS, there is no cure for ALS.
Rasagiline is a drug with possible neuroprotective characteristics. Neuroprotective means that the nervous system may be protected against weakening. It is known that rasagiline has possible neuroprotective characteristics, but the effectiveness of rasagiline for patients with ALS has not been tested. Rasagiline is approved for the treatment of Parkinson's disease.
Rasagiline for treatment of ALS is not approved by the U.S. Food and Drug Administration (FDA) and is investigational. Investigational drugs are studied to find out if they are safe and effective in the treatment of diseases or conditions.
By doing this study, researchers hope to learn if rasagiline is safe and slows disease progression in patients with ALS.
Funding Source - FDA OOPD (FDA Orphan Products Division).
详细描述
The study is a phase II, double-blind, placebo-controlled, multicenter study of rasagiline 2mg/day. Subjects will be assigned to either active agent or placebo (3:1) for twelve months. Subjects will undergo outpatient evaluations at screening, baseline, and months 1, 2, 4, 6, 8, 10 and 12 and telephone assessments at months 3, 5, 7 and 9. There will be a close-out phone call 30 days post month 12.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 21 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV).
- •21 to 80 years of age inclusive.
- •VC greater or equal to 75% of predicted at screening and baseline.
- •Onset of weakness within 2 years prior to enrollment.
- •If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.
- •Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test.
- •Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).
- •Exclusion criteria
- •Requirement for tracheotomy ventilation or non-invasive ventilation for > 23 hours per day.
- •Patients on sympathomimetic agents. This includes pseudoephedrine, phenylephrine, phenylpropanolamine, and ephedrine.
- •Patients on analgesics with serotoninergic properties such as meperidine, tramadol, methadone and propoxyphene, flexeril.
- •Patients on fluoxetine or fluvoxamine.
- •Patients taking amitriptyline > 50 mg/d, trazodone and sertraline > 100 mg/d, citalopram > 20 mg/d or paroxetine > 30 mg/d.
- •Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc).
- •Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc) over the last 30 days.
- •Has a diaphragm pacing device or plan on obtaining a diaphragm pacing device during the course of the study.
- •History of renal disease.
- •History of liver disease.
- •Current pregnancy or lactation.
- •Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.
- •History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.
- •Vital Capacity (VC) < 75% of predicted.
- •Receipt of any investigational drug within the past 30 days.
- •Women with the potential to become pregnant who are not practicing effective birth control.
- •Poorly controlled hypertensive subjects or resting systolic blood pressure (SBP) > 160 mmHg and/or diastolic (DBP) > 95 mmHg.
- •Use of BiPAP at screening.
排除标准
- 未提供
研究组 & 干预措施
Rasagiline
Rasagiline 1mg administered orally as a 2mg single dose once daily for 12 months.
干预措施: Rasagiline (Drug)
Placebo
Inactive ingredient equal to 1mg rasagiline 2mg administered as a single dose once daily for 12 months.
干预措施: Placebo (Drug)
结局指标
主要结局
ALS Functional Rating Scale-Revised (ALSFRS-R)
时间窗: ALS Functional Rating Scale-Revised (ALSFRS-R) Difference from Baseline to Month 12
Difference in ALS Functional Rating Scale - Revised (ALSFRS-R) score. The ALSFRS-R is an ordinal rating scale that assesses 12 functional activities. Each activity is scored between 0-4, with a total score ranging from 48 (normal function) to 0 (no function).
次要结局
- Change in Vital Capacity (VC)(Vital Capacity Change from Baseline to Month 12)
- Change in Quality of Life(Quality of Life Change from Baseline to Month 12)
- Difference in Survival Status Between Study Groups(Survival status at Month 12)
- Number of Participants With Adverse Events(Adverse Events from Baseline to Month 12)
- Effect of Study Drug on Apoptosis Markers(Apoptosis Marker change from Baseline to Month 12)
- Effect of Study Drug on Oxidative Stress(Oxidative Stress change from Baseline to Month 12)
研究者
Richard Barohn, MD
Gertrude and Dewey Zeigler Professor of Neurology and Chair
University of Kansas Medical Center