OBJECTIVE: To assess safety and define efficacy measures of hepatocyte growth factor (HGF) DNA plasmid, VM202, administered by intramuscular injections in patients with amyotrophic lateral sclerosis (ALS). METHODS:Eighteen participants were treated withVM202 administered in divided doses by injections alternating between the upper and lower limbs on d 0, 7, 14, and 21. Subjects were followed for nine months to evaluate possible adverse events. Functional outcome was assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R) as well as by serially measuring muscle strength, muscle circumference, and forced vital capacity. RESULTS:Seventeen of 18 participants completed the study. All participants tolerated 64 mg of VM202 well with no serious adverse events (SAE) related to the drug. Twelve participants reported 26 mild or moderate injection site reactions. Three participants experienced five SAEs unrelated to VM202. One subject died from respiratory insufficiency secondary to ALS progression. CONCLUSIONS: Multiple intramuscular injection of VM202 into the limbs appears safe in ALS subjects. Future trials with retreatment after three months will determine whether VM202 treatment alters the long-term course of ALS.
RCT Entities:
OBJECTIVE: To assess safety and define efficacy measures of hepatocyte growth factor (HGF) DNA plasmid, VM202, administered by intramuscular injections in patients with amyotrophic lateral sclerosis (ALS). METHODS: Eighteen participants were treated with VM202 administered in divided doses by injections alternating between the upper and lower limbs on d 0, 7, 14, and 21. Subjects were followed for nine months to evaluate possible adverse events. Functional outcome was assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R) as well as by serially measuring muscle strength, muscle circumference, and forced vital capacity. RESULTS: Seventeen of 18 participants completed the study. All participants tolerated 64 mg of VM202 well with no serious adverse events (SAE) related to the drug. Twelve participants reported 26 mild or moderate injection site reactions. Three participants experienced five SAEs unrelated to VM202. One subject died from respiratory insufficiency secondary to ALS progression. CONCLUSIONS: Multiple intramuscular injection of VM202 into the limbs appears safe in ALS subjects. Future trials with retreatment after three months will determine whether VM202 treatment alters the long-term course of ALS.
Authors: Ann Logan; Zsuzsanna Nagy; Nicholas M Barnes; Antonio Belli; Valentina Di Pietro; Barbara Tavazzi; Giuseppe Lazzarino; Giacomo Lazzarino; Lars Bruce; Lennart I Persson Journal: PLoS One Date: 2022-05-25 Impact factor: 3.752
Authors: John A Kessler; Aziz Shaibani; Christine N Sang; Mark Christiansen; David Kudrow; Aaron Vinik; Nari Shin Journal: Clin Transl Sci Date: 2021-02-02 Impact factor: 4.689
Authors: Antonio Vallarola; Massimo Tortarolo; Roberta De Gioia; Luisa Iamele; Hugo de Jonge; Giovanni de Nola; Enrica Bovio; Laura Pasetto; Valentina Bonetto; Mattia Freschi; Caterina Bendotti; Ermanno Gherardi Journal: Int J Mol Sci Date: 2020-11-12 Impact factor: 5.923