Clinical trial NCT01935518: A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Historical Placebo Controlled Screening Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Peking University Third Hospital
Sponsor class: OTHER
First posted: 2013-09-05
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will examine whether fasudil is effective and safe in treating patients with amyotrophic lateral sclerosis (ALS).

Detailed description: This is an open labeled, single center clinical study with placebo of historical researches as control. 10 patients will be enrolled in the study. The basic treatment is riluzole, 50mg twice a day. For the procedure, patients will take fasudil treatment for 14 days (30mg twice a day, intravenous). 3 months later, patients will repeat the fasudil treatment. All the patients will be followed up for 6 months. The primary outcome was the decline rate of ALSFRS-R. The secondary outcomes are the survival time, endpoint time(death, tracheotomy and continuous ventilator-dependent), forced vital capacity (FVC), the short form health survey (SF-36), evaluation of cognitive function (verbal fluency and Frontal Behavioral Inventory Scale) and safety.

Eligibility criteria: Inclusion Criteria:

* Clinical diagnosis of laboratory-supported probable, probable, or definite ALS
* Age: 18-70 years
* Disease duration: 3-36 months
* Forced vital capacity: at least 60% of predicted
* ALSFRS-R: at least 30, respiratory items: at least 10
* Decline of ALSFRS-R in the last 3 months before enrollment: 1-8
* Must take riluzole, on a stable dose for at least 30 days prior to baseline visit with no serious side effects. They must continue the riluzole treatment for at least 6 months after enrollment.
* Patients of childbearing potential must be using an effective method of birth control
* Willing and able to give informed consent

Exclusion Criteria:

* Familial ALS
* Pregnant or nursing women
* Patients after tracheotomy or continuous ventilator-dependent (time with non-invasive ventilator more than 22 hours per day for 7 consecutive days.)
* After percutaneous endoscopic gastrostomy
* Alanine Transaminase (ALT) or Aspartate Transaminase (AST): at least 3 times the upper limit of normal
* Abnormal creatinine or urea nitrogen
* Severe cardiac disease, pulmonary disease, hematic disease, autoimmune disease, mental disease, dementia and substance abuse
* History of malignancy
* History of intracranial hemorrhage
* History of severe bleeding of digestive tract, lungs, nose and skin
* Allergic to fasudil
* Participating in other clinical studies or using other investigational drugs at present

Primary outcomes:
measure: The slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) score
timeFrame: Month 3, 6
Secondary outcomes:
measure: Survival time
timeFrame: 2 years
Other outcomes:
measure: Forced Vital Capacity
timeFrame: Baseline, Month 3 and 6
measure: SF-36
timeFrame: Baseline, Month 3 and 6
measure: Cognitive function
timeFrame: Baseline, Month 3 and 6
measure: Safety Labs
timeFrame: Baseline, Month 0.5, 3, 3.5, 6
measure: Adverse Events
timeFrame: Month 0.5, 3, 3.5, 6
Locations:
city: Beijing
country: China
facility: Peking University Third Hospital
Study officials:
affiliation: Peking University Third Hospital
name: Dongsheng Fan, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: