Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

NCT02039401 | Completed Phase 1 Results DMC

• 1 other identifier: VMALS-001 / B
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Lou Gehrig's disease; neurodegenerative disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)

  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

  Day 0, Day 30, Day 60, Day 90, at 6 months and 9 months

• Change From Baseline (Day 0) in Forced Vital Capacity (%)

  Day 30, Day 60, Day 90, at 6 months and 9 months

Study Arms (1)

VM202

EXPERIMENTAL

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

Interventions

VM202 BIOLOGICAL

VM202

Eligibility Criteria

• Age: 21 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (1)

Northwestern University

Chicago, Illinois, 60611, United States

Location

Related Publications (1)

• 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.

  PMID: 28166654 BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Jinsub Lee, PhD.
• Organization: Helixmith, LLC

jinsub.lee@helixmith.com

+82-10-8256-0439

Study Officials

• John A Kessler, MD

  Northwestern University Stem Cell Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 15, 2014
• First Posted: January 17, 2014
• Study Start: March 11, 2014
• Primary Completion: August 3, 2015
• Study Completion: March 6, 2024
• Last Updated: October 6, 2025
• Results First Posted: September 21, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)