A Phase I/II, Open Label Study to Assess the Safety and Tolerability of VM202 in Subjects With Amyotrophic Lateral Sclerosis
Overview
- Phase
- Phase 1
- Intervention
- VM202
- Conditions
- Amyotrophic Lateral Sclerosis
- Sponsor
- Helixmith Co., Ltd.
- Enrollment
- 18
- Locations
- 1
- Primary Endpoint
- Number of Subjects With Serious and Non Serious Adverse Events
- Status
- Completed
- Last Updated
- 6 months ago
Overview
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 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
Investigators
Eligibility Criteria
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
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
Arms & Interventions
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: VM202
Outcomes
Primary Outcomes
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. Descriptive statistics will be used to characterize safety parameters.
Secondary Outcomes
- 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)
- 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)