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Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02039401
Recruitment Status : Completed
First Posted : January 17, 2014
Last Update Posted : October 24, 2019
Information provided by (Responsible Party):
Helixmith Co., Ltd.

Tracking Information
First Submitted Date  ICMJE January 15, 2014
First Posted Date  ICMJE January 17, 2014
Last Update Posted Date October 24, 2019
Study Start Date  ICMJE February 2014
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 15, 2014)
Number of subjects with serious and non serious adverse events [ Time Frame: Throughout the nine month follow up ]
Adverse events (including serious adverse events, and adverse events leading to treatment discontinuation) throughout the 9 months follow-up will be described according to severity and to their relationship with the study drug and injection procedure. Descriptive statistics will be used to characterize safety parameters.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 15, 2014)
  • Hepatocyte Growth Factor (HGF) serum levels [ Time Frame: immediately pre-treatment on Day 0, immediately pre-treatment on Day 7, immediately pre-treatment on Day 14, immediately pre-treatment on Day 21, on Day 30, Day 60 and Day 90 ]
  • Copies of VM202 in whole blood [ Time Frame: Day 0, 7, 14, 21 (pre and post last injection), and Day 30, 60 and 90 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: January 15, 2014)
  • The revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) [ Time Frame: Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 months ]
    There are twelve questions, some asking about daily activities and how much help a patient needs with them, and some about specific symptoms.
  • Muscle Circumference [ Time Frame: Day 60, Day 90, at 6 months, and 9 months ]
    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
  • Forced vital capacity [ Time Frame: Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 month ]
    pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS
  • muscle strength [ Time Frame: Day 30, Day 60, Day 90, at 6 months and 9 months ]
    determined by using the Medical research Council (MRC) scale
Original Other Pre-specified Outcome Measures Same as current
Descriptive Information
Brief Title  ICMJE Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis
Official Title  ICMJE A Phase I/II, Open Label Study to Assess the Safety and Tolerability of VM202 in Subjects With Amyotrophic Lateral Sclerosis
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 Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE Biological: VM202
Study Arms  ICMJE 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.
Intervention: Biological: VM202
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 15, 2014)
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2017
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 21 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02039401
Other Study ID Numbers  ICMJE VMALS-001 / B
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Helixmith Co., Ltd.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Helixmith Co., Ltd.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: John A Kessler, MD Northwestern University Stem Cell Institute
PRS Account Helixmith Co., Ltd.
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP