Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis

• ClinicalTrials.gov Identifier: NCT02039401
• Recruitment Status: Completed
• First Posted: January 17, 2014
• Last Update Posted: October 24, 2019
• Sponsor: Helixmith Co., Ltd.
• Information provided by (Responsible Party): Helixmith Co., Ltd.

Tracking Information

• First Submitted Date: January 15, 2014
• First Posted Date: January 17, 2014
• Last Update Posted Date: October 24, 2019
• Study Start Date: February 2014
• Actual Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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: Same as current

Current Secondary Outcome Measures (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: 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: 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
• 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: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis
• Intervention: Biological: VM202

Study Arms

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

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 15, 2014): 18
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: December 2017
• Actual Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 21 Years to 75 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02039401
• Other Study ID Numbers: VMALS-001 / B
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Helixmith Co., Ltd.
• Original Responsible Party: Same as current
• Current Study Sponsor: Helixmith Co., Ltd.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: John A Kessler, MD; Northwestern University Stem Cell Institute

• PRS Account: Helixmith Co., Ltd.
• Verification Date: October 2019