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Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
VM202
Sponsored by
Helixmith Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Lou Gehrig's disease, neurodegenerative disease

Eligibility Criteria

21 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Age ≥ 21 years, but < or = 75 years

  • Subjects diagnosed with:

    • clinically definite ALS,
    • clinically probable ALS, or
    • clinically probable-laboratory supported ALS as specified in the revised El Escorial / Airlie House diagnostic criteria
  • Onset of ALS < 2 years at Screening
  • Forced Vital Capacity (FVC) ≥ 60% of predicted
  • Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) ≥ 30
  • Not taking riluzole, or on a stable dose for at least thirty days prior to Screening (defined as no noted toxicities)
  • Able and willing to give informed consent
  • If female of childbearing potential, negative urine pregnancy test at Screening and using acceptable method of birth control during the study.

Exclusion Criteria:

  • Neurological symptom(s) due to vitamin B12 deficiency
  • Requires tracheotomy ventilation or noninvasive ventilation > 16 hours / day
  • Comorbidities such as Parkinson's disease, schizophrenia, renal failure, or any other severe complication that, in the Investigator's opinion, will compromise the safety of the patient or confound interpretation of the data collected in this study
  • Other neuromuscular disease
  • Inflammatory disorder of the blood vessels (inflammatory angiopathy, such as Buerger's disease)
  • Active infection
  • Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis)
  • Positive HIV or HTLV at Screening
  • Active Hepatitis B or C as determined by Hepatitis B core antibody (HBcAb), antibody to Hepatitis B surface antigen (IgG and IgM; HBsAb), Hepatitis B surface antigen (HBsAg) and Hepatitis C antibodies (Anti-HCV) at Screening
  • Subjects with known immunosuppression or currently receiving immunosuppressive drugs, chemotherapy or radiation therapy
  • Stroke or myocardial infarction within last 3 months
  • Patients with a recent history (< 5 years) of malignant neoplasm except basal cell carcinoma or squamous cell carcinoma of the skin (if excised and no evidence of recurrence);
  • Subjects requiring > 81 mg daily of acetylsalicylic acid; subjects may be enrolled if willing/able to switch to ≤ 81 mg daily of acetylsalicylic acid or to another medication
  • Subjects requiring regular COX-2 inhibitor drug(s) or non-specific COX-1/COX-2 inhibiting drugs, or high dose steroids (excepting inhaled steroids); subjects may be enrolled if willing/able to undergo medication wash-out prior to the first dosing and to refrain from taking these drugs for the duration of the study
  • Have used an investigational drug within 30 days of Screening
  • Pregnant or currently lactating
  • Major psychiatric disorder in past 6 months
  • Known drug or alcohol dependence or any other factors which will interfere with the study conduct or interpretation of the results or who in the opinion of the Investigator are not suitable to participate.

Sites / Locations

  • Northwestern University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VM202

Arm Description

Total dose of 64 mg of VM202 It will be administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart.

Outcomes

Primary Outcome Measures

Number of Subjects With Serious and Non Serious Adverse Events
Adverse events (including serious adverse events, and adverse events leading to treatment discontinuation) throughout the 9 months follow-up. Descriptive statistics will be used to characterize safety parameters.

Secondary Outcome Measures

The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
The Amyotrophic Lateral Sclerosis Function Rating Scale includes twelve questions that ask the physician to rate his/her impression of the patient's level of functional impairment in performing one of twelve common tasks (e.g., climbing stairs). Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
The Medical Research Council (MRC) Scale is a validated instrument used in assessing muscle strength. It uses the numeral grades 0-5 to characterize muscle strength as follows: 0 - No contraction;1 - Flicker or trace contraction; 2 - Active movement, with gravity eliminated; 3 - Active movement against gravity; 4 - Active movement against gravity and resistance; 5 - Normal power The MRC scale was used to assess muscle strength in the muscle groups injected with Engensis.
Change From Baseline (Day 0) in Forced Vital Capacity (%)
pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS

Full Information

First Posted
January 15, 2014
Last Updated
September 20, 2023
Sponsor
Helixmith Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT02039401
Brief Title
Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis
Official Title
A Phase I/II, Open Label Study to Assess the Safety and Tolerability of VM202 in Subjects With Amyotrophic Lateral Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
March 2014 (Actual)
Primary Completion Date
August 2015 (Actual)
Study Completion Date
December 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Helixmith Co., Ltd.

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine the safety and tolerability of intramuscular injections of VM202 at different injection sites in people with amyotrophic lateral sclerosis.
Detailed Description
A phase I/II, open label, single center study designed to assess the safety and tolerability of intramuscular injections of VM202 inpatients with ALS. Study enrollment will be staged. Enrollment will be halted after the sixth subject qualifies for treatment. A Data Safety Monitoring Board (DSMB) will conduct a safety evaluation after the first patient treated completes the Day 60 follow-up evaluation and the five other sequentially enrolled subjects complete at least the Day 30 follow-up. Enrollment will be suspended until a formal recommendation to proceed (or not proceed) is made by the DSMB. Patients aged ≥ 21 years, but ≤ 75 years diagnosed with clinically definite, clinically probable, or clinically probable-laboratory supported Amyotrophic Lateral Sclerosis (ALS). This study is not powered to detect differences in efficacy measures. However, descriptive statistics (N, mean, median, SD, minimum and maximum values, where applicable) of clinically meaningful endpoints will be tabulated

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Lou Gehrig's disease, neurodegenerative disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VM202
Arm Type
Experimental
Arm Description
Total dose of 64 mg of VM202 It will be administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart.
Intervention Type
Biological
Intervention Name(s)
VM202
Primary Outcome Measure Information:
Title
Number of Subjects With Serious and Non Serious Adverse Events
Description
Adverse events (including serious adverse events, and adverse events leading to treatment discontinuation) throughout the 9 months follow-up. Descriptive statistics will be used to characterize safety parameters.
Time Frame
Throughout the nine month follow up
Secondary Outcome Measure Information:
Title
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Description
The Amyotrophic Lateral Sclerosis Function Rating Scale includes twelve questions that ask the physician to rate his/her impression of the patient's level of functional impairment in performing one of twelve common tasks (e.g., climbing stairs). Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
Time Frame
Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 months
Title
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Description
The Medical Research Council (MRC) Scale is a validated instrument used in assessing muscle strength. It uses the numeral grades 0-5 to characterize muscle strength as follows: 0 - No contraction;1 - Flicker or trace contraction; 2 - Active movement, with gravity eliminated; 3 - Active movement against gravity; 4 - Active movement against gravity and resistance; 5 - Normal power The MRC scale was used to assess muscle strength in the muscle groups injected with Engensis.
Time Frame
Day 0, Day 30, Day 60, Day 90, at 6 months and 9 months
Title
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Description
pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS
Time Frame
Day 30, Day 60, Day 90, at 6 months and 9 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age ≥ 21 years, but < or = 75 years Subjects diagnosed with: clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised El Escorial / Airlie House diagnostic criteria Onset of ALS < 2 years at Screening Forced Vital Capacity (FVC) ≥ 60% of predicted Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) ≥ 30 Not taking riluzole, or on a stable dose for at least thirty days prior to Screening (defined as no noted toxicities) Able and willing to give informed consent If female of childbearing potential, negative urine pregnancy test at Screening and using acceptable method of birth control during the study. Exclusion Criteria: Neurological symptom(s) due to vitamin B12 deficiency Requires tracheotomy ventilation or noninvasive ventilation > 16 hours / day Comorbidities such as Parkinson's disease, schizophrenia, renal failure, or any other severe complication that, in the Investigator's opinion, will compromise the safety of the patient or confound interpretation of the data collected in this study Other neuromuscular disease Inflammatory disorder of the blood vessels (inflammatory angiopathy, such as Buerger's disease) Active infection Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis) Positive HIV or HTLV at Screening Active Hepatitis B or C as determined by Hepatitis B core antibody (HBcAb), antibody to Hepatitis B surface antigen (IgG and IgM; HBsAb), Hepatitis B surface antigen (HBsAg) and Hepatitis C antibodies (Anti-HCV) at Screening Subjects with known immunosuppression or currently receiving immunosuppressive drugs, chemotherapy or radiation therapy Stroke or myocardial infarction within last 3 months Patients with a recent history (< 5 years) of malignant neoplasm except basal cell carcinoma or squamous cell carcinoma of the skin (if excised and no evidence of recurrence); Subjects requiring > 81 mg daily of acetylsalicylic acid; subjects may be enrolled if willing/able to switch to ≤ 81 mg daily of acetylsalicylic acid or to another medication Subjects requiring regular COX-2 inhibitor drug(s) or non-specific COX-1/COX-2 inhibiting drugs, or high dose steroids (excepting inhaled steroids); subjects may be enrolled if willing/able to undergo medication wash-out prior to the first dosing and to refrain from taking these drugs for the duration of the study Have used an investigational drug within 30 days of Screening Pregnant or currently lactating Major psychiatric disorder in past 6 months Known drug or alcohol dependence or any other factors which will interfere with the study conduct or interpretation of the results or who in the opinion of the Investigator are not suitable to participate.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John A Kessler, MD
Organizational Affiliation
Northwestern University Stem Cell Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
28166654
Citation
Sufit RL, Ajroud-Driss S, Casey P, Kessler JA. Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2017 May;18(3-4):269-278. doi: 10.1080/21678421.2016.1259334. Epub 2017 Feb 6.
Results Reference
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Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

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