Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis
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.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/II, Open Label Study to Assess the Safety and Tolerability of VM202 in Subjects With Amyotrophic Lateral Sclerosis|
- Number of subjects with serious and non serious adverse events [ Time Frame: Throughout the nine month follow up ] [ Designated as safety issue: Yes ]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.
- 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 ] [ Designated as safety issue: No ]
- Copies of VM202 in whole blood [ Time Frame: Day 0, 7, 14, 21 (pre and post last injection), and Day 30, 60 and 90 ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]determined by using the Medical research Council (MRC) scale
|Study Start Date:||February 2014|
|Estimated Study Completion Date:||September 2015|
|Estimated Primary Completion Date:||September 2015 (Final data collection date for primary outcome measure)|
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02039401
|United States, Illinois|
|Chicago, Illinois, United States, 60611|
|Principal Investigator:||John A Kessler, MD||Northwestern University Stem Cell Institute|