EH301 for the Treatment of ALS

• ClinicalTrials.gov Identifier: NCT03489200
• Recruitment Status: Completed
• First Posted: April 5, 2018
• Last Update Posted: May 18, 2018
• Sponsor: Elysium Health
• Collaborators: Fundación Universidad Católica de Valencia San Vicente Mártir; University of Valencia
• Information provided by (Responsible Party): Elysium Health

Tracking Information

• First Submitted Date: March 27, 2018
• First Posted Date: April 5, 2018
• Last Update Posted Date: May 18, 2018
• Actual Study Start Date: January 2017
• Actual Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 16, 2018)

ALSFRS-r [ Time Frame: 6 months ]

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.

Original Primary Outcome Measures (submitted: April 3, 2018)

• ALSFRS-R [ Time Frame: 6 months ]
  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.
• Anthropometry [ Time Frame: 6 months ]
  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.
• FVC [ Time Frame: 6 months ]
  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.
• MRC Index [ Time Frame: 6 months ]
  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.
• Electromyogram [ Time Frame: 6 months ]
  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.

Current Secondary Outcome Measures (submitted: May 16, 2018)

• MRC [ Time Frame: 6 months ]
  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.
• FVC [ Time Frame: 6 months ]
  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.
• Electromyogram [ Time Frame: 6 months ]
  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.
• Anthropometry [ Time Frame: 6 months ]
  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.

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: EH301 for the Treatment of ALS
• Official Title: EH301 for the Treatment of Amyotrophic Lateral Sclerosis: a Placebo-Controlled Study
• Brief Summary: 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis

Intervention

• Other: EH301
  1-(beta-D-Ribofuranosyl)nicotinamide chloride and 3,5-Dimethoxy-4'-hydroxy-trans-stilbene
• Other: Placebo
  No intervention- placebo

Study Arms

• Experimental: EH301
  Intervention: Other: EH301
• Placebo Comparator: Placebo
  Intervention: Other: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 3, 2018): 32
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: June 2017
• Actual Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Spain

Administrative Information

• NCT Number: NCT03489200
• Other Study ID Numbers: H1479983999044
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Elysium Health
• Original Responsible Party: Same as current
• Current Study Sponsor: Elysium Health
• Original Study Sponsor: Same as current

Collaborators

• Fundación Universidad Católica de Valencia San Vicente Mártir
• University of Valencia

• Investigators: Not Provided
• PRS Account: Elysium Health
• Verification Date: May 2018