EH301 for the Treatment of ALS

EH301 for the Treatment of Amyotrophic Lateral Sclerosis: a Placebo-Controlled Study

The objective of this trial is to evaluate the efficacy and tolerability of EH301 in patients with amyotrophic lateral sclerosis. Patients with ALS are randomized to receive either EH301 or placebo daily and undergo active evaluation for 6 months.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Other: EH301; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Spain
  • Valencia, Spain
    • Universidad Católica de Valencia San Vicente Mártir

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• 18 years or older
• Diagnosis of probable or definite (sporadic or familial) ALS by El Escorial criteria
• Onset of symptomatology for more than 6 months
• If female: not lactating; negative pregnancy test; agree to use an effective method of birth control throughout study

Exclusion Criteria:

• Tracheostomy, invasive ventilation, or non-invasive positive pressure ventilation
• Gastrostomy
• Evidence of major psychiatric disorder or clinically evident dementia
• Diagnosis of a neurodegenerative disease in addition to ALS
• Current medication apart from riluzole that in the opinion of the investigator would make the patient unsuitable for study participation
• Recent history (within the previous 6 months) or current evidence of alcohol or drug abuse
• Concurrent unstable disease involving any system (e.g. carcinoma other than basal cell carcinoma), any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of coronary artery disease, or any other condition that in the opinion of the investigator would make the patient unsuitable for study participation
• Baseline QTc (Bazett) > 450 msec for males and > 470 msec for females
• Known hepatitis B/C or HIV positive serology
• Renal impairment defined as blood creatinine > 2x ULN
• Hepatic impairment and/or liver enzymes (ALT or AST) > 3x ULN
• Hemostasis disorders or current treatment with oral anticoagulants
• Participated in any other investigational drug or therapy study with a non-approved medication, within the previous 3 months
• No medical insurance

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Other: Placebo; No intervention- placebo
Experimental: EH301	Other: EH301; 1-(beta-D-Ribofuranosyl)nicotinamide chloride and 3,5-Dimethoxy-4'-hydroxy-trans-stilbene

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ALSFRS-r	ALS Functional Rating Scale - revised score is a summation of ratings across across fine and gross motor, bulbar and respiratory functional domains. The scale ranges from 0-48, with higher total score representing better outcomes. The twelve subscales are rated on a five-point scale from 0 (can't do) to 4 (normal ability) and combined to compute the total score. This outcome measures change observed from baseline to 6 months.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MRC	Medical Research Council Scale measures the strength of muscles in ALS patients. This 11-step grading scale is a summation of scores for 8 different muscles, each with a subscale of 0 (no muscle contraction) to 5 (normal power). To calculate the total MRC Index a progressively increasing number from 0 (0 in the scale) to 10 (5 in the scale) is given to each step and each muscle. The total MRC index per patient corresponds to the sum of the numbers given to all 8 muscles. This outcome measures change observed in MRC Index from baseline to 6 months.	6 months
FVC	Forced Vital Capacity is a predictor of survival and disease progression in ALS patients and is measured using a touch spirometer and expressed as the % of standard value depending on sex, weight, and age parameters. This outcome measures change observed from baseline to 6 months.	6 months
Electromyogram	A surface electromyogram (EMG) measuring device is used to measure muscle activity in the same muscles as the MRC Index. Muscle activity is expressed as the EMG amplitude (uV). This outcome measures change in EMG amplitude observed from baseline to 6 months.	6 months
Anthropometry	Fat and skeletal muscle weights (kg) are calculated following standard anthropometric procedures. Fat and skeletal weight outcomes refer to change observed comparing baseline vs 6 months.	6 months

Collaborators and Investigators

• Sponsor: Elysium Health
• Collaborators: University of Valencia; Fundación Universidad Católica de Valencia San Vicente Mártir

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2017
• Primary Completion (Actual): June 1, 2017
• Study Completion (Actual): June 1, 2017

Study Registration Dates

• First Submitted: March 27, 2018
• First Submitted That Met QC Criteria: April 3, 2018
• First Posted (Actual): April 5, 2018

Study Record Updates

• Last Update Posted (Actual): May 18, 2018
• Last Update Submitted That Met QC Criteria: May 16, 2018
• Last Verified: May 1, 2018

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
• Other Study ID Numbers: H1479983999044

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No