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.
A phase I/II, open label, single center study designed to assess the safety and tolerability of intramuscular injections of VM202 inpatients with ALS. Study enrollment will be staged. Enrollment will be halted after the sixth subject qualifies for treatment. A Data Safety Monitoring Board (DSMB) 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 DSMB. Patients aged ≥ 21 years, but ≤ 75 years diagnosed with clinically definite, clinically probable, or clinically probable-laboratory supported Amyotrophic Lateral Sclerosis (ALS). This study is not powered to detect differences in efficacy measures. However, descriptive statistics (N, mean, median, SD, minimum and maximum values, where applicable) of clinically meaningful endpoints will be tabulated
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.