A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

A Historical Placebo Controlled Screening Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01935518
• Other study ID #: PUTH-2013121
• Status: Recruiting
• Phase: Phase 2
• First received: September 1, 2013
• Last updated: September 5, 2013
• Start date: September 2013
• Est. completion date: May 2015

Study information

• Verified date: September 2013
• Source: Peking University Third Hospital
• Contact: Dongsheng Fan, MD, PhD
• Phone: 0086-15611908107
• Email: dsfan[@]sina.com
• Is FDA regulated: No
• Health authority: China: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: May 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 70 Years

Eligibility

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

Study Design

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Fasudil
All the patients will take the fasudil treatment for 14 days (30mg twice a day, intravenous). 3 months later they will repeat the treatment mentioned before.

Locations

Country	Name	City	State
China	Peking University Third Hospital	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University Third Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Forced Vital Capacity		Baseline, Month 3 and 6	No
Other	SF-36		Baseline, Month 3 and 6	No
Other	Cognitive function	verbal fluency and Frontal Behavioral Inventory Scale (FBI)	Baseline, Month 3 and 6	No
Other	Safety Labs	blood test including blood RT, liver function, renal function, serum electrolyte, myocardial enzyme, glucose and coagulation function	Baseline, Month 0.5, 3, 3.5, 6	Yes
Other	Adverse Events		Month 0.5, 3, 3.5, 6	Yes
Primary	The slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) score		Month 3, 6	No
Secondary	Survival time		2 years	No