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EH301 for the Treatment of ALS

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Not Applicable
Locations
Spain
Study Type
Interventional
Intervention
EH301
Placebo
Sponsored by
Elysium Health
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Universidad Católica de Valencia San Vicente Màrtir

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

EH301

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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.
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.

Full Information

First Posted
March 27, 2018
Last Updated
May 16, 2018
Sponsor
Elysium Health
Collaborators
Fundación Universidad Católica de Valencia San Vicente Mártir, University of Valencia
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1. Study Identification

Unique Protocol Identification Number
NCT03489200
Brief Title
EH301 for the Treatment of ALS
Official Title
EH301 for the Treatment of Amyotrophic Lateral Sclerosis: a Placebo-Controlled Study
Study Type
Interventional

2. Study Status

Record Verification Date
May 2018
Overall Recruitment Status
Completed
Study Start Date
January 2017 (Actual)
Primary Completion Date
June 2017 (Actual)
Study Completion Date
June 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Elysium Health
Collaborators
Fundación Universidad Católica de Valencia San Vicente Mártir, University of Valencia

4. Oversight

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

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderOutcomes Assessor
Allocation
Randomized
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
EH301
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Other
Intervention Name(s)
EH301
Intervention Description
1-(beta-D-Ribofuranosyl)nicotinamide chloride and 3,5-Dimethoxy-4'-hydroxy-trans-stilbene
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
No intervention- placebo
Primary Outcome Measure Information:
Title
ALSFRS-r
Description
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.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
MRC
Description
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.
Time Frame
6 months
Title
FVC
Description
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.
Time Frame
6 months
Title
Electromyogram
Description
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.
Time Frame
6 months
Title
Anthropometry
Description
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.
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Universidad Católica de Valencia San Vicente Màrtir
City
Valencia
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No

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EH301 for the Treatment of ALS

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