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

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

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

Study Overview

• Status: Unknown
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Fasudil

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.

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Dongsheng Fan, MD, PhD; Phone Number: 0086-15611908107; Email: dsfan@sina.com

Study Locations

• China
  • Beijing, China, 100191
    • Recruiting
    • Peking University Third Hospital
    • Contact: Xiaolu Liu, MD; Phone Number: 0086-1082265032; Email: liuxiaolupku@gmail.com
    • Principal Investigator: Dongsheng Fan, MD, PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 68 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The slope of decline of the ALS Functional Rating Scale-Revised (ALSFRS-R) score	Month 3, 6

Secondary Outcome Measures

Outcome Measure	Time Frame
Survival time	2 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Forced Vital Capacity		Baseline, Month 3 and 6
SF-36		Baseline, Month 3 and 6
Cognitive function	verbal fluency and Frontal Behavioral Inventory Scale (FBI)	Baseline, Month 3 and 6
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
Adverse Events		Month 0.5, 3, 3.5, 6

Collaborators and Investigators

• Sponsor: Peking University Third Hospital
• Investigators: Principal Investigator: Dongsheng Fan, MD, PhD, Peking University Third Hospital

Study record dates

Study Major Dates

• Study Start: September 1, 2013
• Primary Completion (Anticipated): March 1, 2015
• Study Completion (Anticipated): May 1, 2015

Study Registration Dates

• First Submitted: September 1, 2013
• First Submitted That Met QC Criteria: September 1, 2013
• First Posted (Estimate): September 5, 2013

Study Record Updates

• Last Update Posted (Estimate): September 6, 2013
• Last Update Submitted That Met QC Criteria: September 5, 2013
• Last Verified: September 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Enzyme Inhibitors; Membrane Transport Modulators; Protein Kinase Inhibitors; Calcium-Regulating Hormones and Agents; Calcium Channel Blockers; Fasudil
• Other Study ID Numbers: PUTH-2013121