Trial reportOrphanet journal of rare diseases2026
An open-label single-arm phase 1/2a study to evaluate the safety and exploratory efficacy of a VM202 in patients with Charcot-Marie-Tooth disease 1A.
Trial report in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05361031 (Single Center, Open Label, Repeat Intramuscular Administration, 270 Days, Phase I/2a Clinical Trial to Evaluate Safety, Tolerability of Investigational Product), which is not on this map. Cited by 2 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Single Center, Open Label, Repeat Intramuscular Administration, 270 Days, Phase I/2a Clinical Trial to Evaluate Safety, Tolerability of Investigational Product (Engensis: VM202) With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
Who cites it
2 citing papers in PubMed.
- Peripheral Myelin Protein-22 and Its Prominence in Charcot-Marie-Tooth Disease.Chemical reviews · 2026Review
- Contemporary Strategies of Gene and Cell Therapy in the Treatment of Peripheral Nervous System Injuries and Disorders.International journal of molecular sciences · 2026Review
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThis is the gene therapy trial in patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Intramuscular injections of VM202, a plasmid DNA encoding human hepatocyte growth factor, was investigated for safe and exploratory effective in patients with CMT1A.
methodsThis study was an open-label single-arm phase 1/2a trial. Thirteen patients with CMT1A were screened, and 12 consented and enrolled between September 2020 and November 2020. Patients received intramuscular injections of 14 mg of VM202 at baseline, and on days 14, 90, and 104 in both legs. Safety evaluations and clinical assessments using the CMT neuropathy score version 2 (CMTNSv2), CMT examination score (CMTES), Rasch-modified CMTNSv2 (CMTNSv2-R), Rasch-modified CMTES (CMTES-R), functional disability scale (FDS), overall neuropathy limitation score, and 10-meter walk test were performed throughout a 270-day follow-up period. A Wilcoxon signed-rank test was used for statistical comparisons of continuous variables.
resultsThe primary objective of this study was to assess the safety and tolerability of intramuscular injections of VM202 in patients with CMT1A. All participants tolerated VM202 well, without any serious adverse events related to the study drug. The secondary objective was to evaluate exploratory efficacy. CMTNSv2, CMTES, CMTNSv2-R, and CMTES-R decreased between baseline and day 270 with mean decreases of 2.17, 2.50, 2.08, and 2.33 points, respectively (p < 0.01). Also, FDS decreased with a mean percent decrease of 0.58 (p < 0.05). However, ONLS leg scale and 10-meter walk test were not statistically significant changes.
conclusionsIntramuscular injections of a VM202 appear to be safe and tolerated in CMT1A patients with an exploratory efficacy warrant confirmation in a phase 2b randomized controlled trial. TRIAL REGISTRATION INFORMATION: Name of the trial registry (VM202 for CMT1A), Registration number (NCT05361031), URL of the registry ( http://www.clinicaltrials.gov ) and date of registration (2022–03-31, retrospectively registered).
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