CompletedPhase 1/2

The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)

South Korea12 participantsStarted 2020-09-21

Plain-language summary

To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients

Age range19 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Males or females ≥ 19 years of age and ≤ 65 years of age
✓. Patients with confirmed diagnosis of CMT1A by genetic testing
✓. Patients with mild to moderate severity assessed by Charcot Marie Tooth Neuropathy Score version 2 (CMTNS v2) with a score \> 2 and ≤ 20
✓. Individuals with lower limb muscle weakness with minimum dorsiflexion or more
✓. Individuals who voluntarily consented to participate in this study and signed the IRB approved informed consent form after listening to a description on the characteristics of this clinical study prior to all screening tests
✓. Individuals who can comply with the requirements in the clinical study
✓. In case of females of child bearing potential, those who test negative in a urine or serum pregnancy test at screening
✓. Individuals who practice medically approved contraceptive methods throughout the clinical study

✕. Patients with significant respiratory, circulatory, renal, gastrointestinal, hepatic, endocrine, hematologic, psychiatric disorders or other severe diseases, or alcohol or drug addiction who may develop safety issues or cause confusion in the interpretation of the clinical study results as determined by the principal investigator
✕. Patients with other neuromuscular diseases or neuropathy-inducing factors: Patients with chronic alcohol addiction, undergoing anticancer chemotherapy, or taking neurotoxic drugs
✕. Patients diagnosed with diabetes
✕. Patients diagnosed with inflammatory bowel disease
✕. Patients with a history of stroke or cerebral ischemic attack within 12 months prior to the screening date
✕. Patients with a history of coronary artery disease, such as myocardial infarction and unstable angina pectoris, within 12 months prior to the screening date
✕. Morbidly obese patients with body mass index (BMI) ≥ 37
✕. Patients who underwent orthopedic surgery (corrective surgery for bone and ligament, artificial joint implantation, osteosynthesis, osteotomy, arthroscopic surgery) in the lower limbs within 6 months prior to the screening date

Safety of Intramuscular (IM) Injections of Engensis in Participants

Timeframe: Day 0 visit to the Day 270 visit

NCT IDNCT05361031

SponsorHelixmith Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-09-09

Results submitted2023-07-23

Who can participate

Age range19 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Males or females ≥ 19 years of age and ≤ 65 years of age
✓. Patients with confirmed diagnosis of CMT1A by genetic testing
✓. Patients with mild to moderate severity assessed by Charcot Marie Tooth Neuropathy Score version 2 (CMTNS v2) with a score \> 2 and ≤ 20
✓. Individuals with lower limb muscle weakness with minimum dorsiflexion or more
✓. Individuals who voluntarily consented to participate in this study and signed the IRB approved informed consent form after listening to a description on the characteristics of this clinical study prior to all screening tests
✓. Individuals who can comply with the requirements in the clinical study
✓. In case of females of child bearing potential, those who test negative in a urine or serum pregnancy test at screening
✓. Individuals who practice medically approved contraceptive methods throughout the clinical study

✕. Patients with significant respiratory, circulatory, renal, gastrointestinal, hepatic, endocrine, hematologic, psychiatric disorders or other severe diseases, or alcohol or drug addiction who may develop safety issues or cause confusion in the interpretation of the clinical study results as determined by the principal investigator
✕. Patients with other neuromuscular diseases or neuropathy-inducing factors: Patients with chronic alcohol addiction, undergoing anticancer chemotherapy, or taking neurotoxic drugs
✕. Patients diagnosed with diabetes
✕. Patients diagnosed with inflammatory bowel disease
✕. Patients with a history of stroke or cerebral ischemic attack within 12 months prior to the screening date
✕. Patients with a history of coronary artery disease, such as myocardial infarction and unstable angina pectoris, within 12 months prior to the screening date
✕. Morbidly obese patients with body mass index (BMI) ≥ 37
✕. Patients who underwent orthopedic surgery (corrective surgery for bone and ligament, artificial joint implantation, osteosynthesis, osteotomy, arthroscopic surgery) in the lower limbs within 6 months prior to the screening date