Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS

  • STATUS
    Recruiting
  • End date
    Jul 25, 2022
  • participants needed
    120
  • sponsor
    University Medical Center Goettingen
Updated on 26 January 2021
Investigator
Paul Lingor, MD
Primary Contact
Universit tsklinikum Carl Gustav Carus Dresden (7.4 mi away) Contact
+13 other location

Summary

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder and therapeutic options are limited. The rho kinase (ROCK) inhibitor Fasudil was shown to be neuroprotective, induced axonal regeneration and improved survival and behavioral outcome in models of ALS and other neurodegenerative diseases. The aim of this phase IIa, multi-center and double-blind study is to analyze the safety, tolerability and efficacy of fasudil in two different doses compared to placebo in approximately 16 trial sites in Germany, France and Switzerland. Intravenous application of fasudil will be performed in 80 patients and placebo in 40 patients two times daily for 20 treatment days. The hypothesis is that fasudil is safe and well-tolerated and its application will significantly improve the clinical outcome in patients with ALS.

Details
Condition Myelopathy, Amyotrophic Lateral Sclerosis, Amyotrophic Lateral Sclerosis (ALS), Spinal Cord Disorders, lou gehrig's disease
Treatment Placebo, fasudil
Clinical Study IdentifierNCT03792490
SponsorUniversity Medical Center Goettingen
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria
Disease duration more than 6 months and less than 24 months (inclusive). Disease onset defined as date of first muscle weakness, excluding fasciculations and cramps
Vital capacity more than 65% of normal (slow vital capacity; best of three measurements)
Age: 18 years
Patients have to be treated with Riluzole (2 x 50mg/d), must be stable for at least four weeks before randomization
Patients who have started on Edaravone therapy shall continue Edaravone treatment. Edaravone treatment must not be discontinued for reasons of trial participation
Women of childbearing age must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. Acceptable methods of birth control with a low failure rate i.e. less than 1% per year) when used consistently and correct are such as implants, injectables, combined oral contraceptives, hormonal intrauterine devices (IUDs), sexual abstinence or vasectomized partner
Capable of thoroughly understanding all information given and giving full informed consent according to good clinical practice (GCP)
Patients have to have a valid health insurance, when recruited in a center in France

Exclusion Criteria

Previous participation in another clinical study involving trial medication within the preceding 12 weeks or five terminal half times of the longest to be eliminated trial medications (whichever is longer) or previous participation in this trial
Tracheostomy or continuous assisted ventilation of any type during the preceding three months before randomization or a significant pulmonary disorder not attributed to ALS, which may complicate the evaluation of respiratory function, intermittent non-invasive ventilation is permitted
Patients with a history of intracranial bleeding, known intracerebral aneurysms or Moyamoya disease, or positive family history for the above. If only family history positive, magnetic resonance (MR)- or x-ray-based cranial imaging not older than 24 months must confirm absence of bleeding, aneurysms or Moyamoya
Gastrostomy
Any medical condition known to have an association with motor neuron dysfunction or involving neuromuscular weakness or another neurodegenerative disease, e.g. Parkinson's disease (PD) or Alzheimer's disease (AD), which might confound or obscure the diagnosis of ALS
Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment
Patients with known arterial hypotension (resting blood pressure <90/60 mmHg) or previous hypotensive episodes or requiring treatment for increasing of blood pressure, such as fludrocortisone, midodrine, etilefrine, cafedrine or theodrenaline
Patients with an uncontrollable or unstable arterial hypertensive disease (resting blood pressure >180 mmHg systolic and/or >120 mmHg diastolic under current antihypertensive medication)
Known pulmonary hypertension and any medication prescribed for treatment of pulmonary hypertension
Confirmed hepatic insufficiency or abnormal liver function (stable aspartate transaminase (ASAT) and/or alanine aminotransferase (ALAT) greater than 3 times the upper limit of the normal range) and determined to be non-transient through repeat testing
Renal insufficiency with a glomerular filtration rate (GFR) <60 ml/min/1,73m (calculated by Modification of Diet in Renal Disease (MDRD) equation) and determined to be non-transient through repeat testing
Major psychiatric disorder, significant cognitive impairment or clinically evident dementia precluding evaluation of symptoms
Hypersensitivity to any component of the study drug
Liable to be not cooperative or comply with the trial requirements (as assessed by the investigator), or unable to be reached in the case of emergency
Pregnant or breast-feeding females or females with childbearing potential, if no adequate contraceptive measures are used
Prisoners or subjects who are involuntary incarcerated
Patients subject to legal protection measures
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