Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

A Phase I/II, Open Label Study to Assess the Safety and Tolerability of VM202 in Subjects With Amyotrophic Lateral Sclerosis

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: VM202

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 Amyotrophic Lateral Sclerosis. Study enrollment will be staged. Enrollment will be halted after the sixth subject qualifies for treatment. A Data Safety Monitoring Board 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 Data Safety Monitoring Board.

Patients aged ≥ 21 years, but ≤ 75 years diagnosed with clinically definite, clinically probable, or clinically probable-laboratory supported Amyotrophic Lateral Sclerosis.

This study is not powered to detect differences in efficacy measures. However, descriptive statistics (N, mean, median, standard deviation, minimum and maximum values, where applicable) of clinically meaningful endpoints will be tabulated

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 21 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age ≥ 21 years, but < or = 75 years

• Subjects diagnosed with:

  • clinically definite Amyotrophic Lateral Sclerosis,
  • clinically probable Amyotrophic Lateral Sclerosis, or
  • clinically probable-laboratory supported Amyotrophic Lateral Sclerosis as specified in the revised El Escorial / Airlie House diagnostic criteria
• Onset of ALS < 2 years at Screening
• Forced Vital Capacity ≥ 60% of predicted
• Revised Amyotrophic Lateral Sclerosis Functional Rating Scale ≥ 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, antibody to Hepatitis B surface antigen (IgG and IgM), Hepatitis B surface antigen and Hepatitis C antibodies 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VM202; 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.	Biological: VM202

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Throughout the nine month follow up

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 months
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.	Day 0, Day 30, Day 60, Day 90, at 6 months and 9 months
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	Day 30, Day 60, Day 90, at 6 months and 9 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
The revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)	There are twelve questions, some asking about daily activities and how much help a patient needs with them, and some about specific symptoms.	Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 months
Muscle Circumference	Measurements will be taken bilaterally: Mid-arm: at the midpoint of a vertical line that joins the acromion process to the olecranon process; Mid forearm: at the proximal one third point of a vertical line that joins the medial epicondyle to the styloid process of the ulna; Mid-thigh: midpoint of a vertical line that joins the anterior superior iliac spine to the superior edge of the patella; Mid-leg: at the proximal one third point of a vertical line that joins the fibular head to the lateral malleolus	Day 60, Day 90, at 6 months, and 9 months
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	Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 month
muscle strength	determined by using the Medical research Council (MRC) scale	Day 30, Day 60, Day 90, at 6 months and 9 months

Collaborators and Investigators

• Sponsor: Helixmith Co., Ltd.
• Investigators: Principal Investigator: John A Kessler, MD, Northwestern University Stem Cell Institute

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): March 11, 2014
• Primary Completion (Actual): August 3, 2015
• Study Completion (Actual): March 6, 2024

Study Registration Dates

• First Submitted: January 15, 2014
• First Submitted That Met QC Criteria: January 15, 2014
• First Posted (Estimated): January 17, 2014

Study Record Updates

• Last Update Posted (Estimated): October 6, 2025
• Last Update Submitted That Met QC Criteria: September 23, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: ALS; Lou Gehrig's disease; neurodegenerative disease
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases
• Other Study ID Numbers: VMALS-001 / B

Plan for Individual participant data (IPD)

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