CompletedPhase 1

Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A

South Korea9 participantsStarted 2022-02-22

Plain-language summary

Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A

Who can participate

Age range19 Years – 75 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.

Inclusion criteria

✓. Males and females aged 19 to 75 years old
✓. Those diagnosed with CMT type 1A by a genetic test
✓. Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
✓. Those who have dorsiflexion muscle weakness
✓. Those who can comply with the requirements for clinical trials
✓. For women of childbearing potential, those who have a negative urine pregnancy test at screening
✓. Those who use a medically acceptable method of contraception until clinical trial visit 7 (short-term follow-up visit, 16 weeks): hormonal contraception, intrauterine device (IUD), intrauterine system (IUS), vasectomy, tubal ligation, or double barrier method using a cervical cap or a diaphragm with a male condom.
✓. Those who voluntarily agree to participate in this study and sign an IRB-approved consent form after being informed about the characteristics of this clinical trial prior to all screening tests

Exclusion criteria

✕. Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
✕. Patients diagnosed with type 1 or type 2 diabetes
✕. Those with a history of stroke or cerebral ischemic attack within 12 months of screening
✕. Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
✕. Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of screening
✕. Those who have ankle contractures or have surgery that may affect muscle strength assessment
✕. Those who have experience with stem cell therapy or gene therapy before screening
✕. Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)

What they're measuring

Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product

Timeframe: Week 16 after treatment

Determination of Dose-limiting toxicity (DLT) levels of EN001

Timeframe: Up to Week 4 after dosing on Day 0

Determination of Maximum tolerated dose (MTD) levels of EN001

Timeframe: Up to Week 4 after dosing on Day 0

Number of participants with Vital Signs abnormalities

Timeframe: From screening up to Week 16

Number of participants with clinically significant abnormalities of Physical Examinations

Timeframe: From screening up to Week 16

Number of participants with abnormalities of Laboratory Parameters

Timeframe: From screening up to Week 16

Number of participants with 12-lead Electrocardiography (ECG) abnormalities

Timeframe: From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion)

Trial details

NCT IDNCT05333406

SponsorENCell

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-12-20

View on ClinicalTrials.gov More Charcot-Marie-Tooth Disease, Type IA trials

Who can participate

Age range19 Years – 75 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.

Inclusion criteria

✓. Males and females aged 19 to 75 years old
✓. Those diagnosed with CMT type 1A by a genetic test
✓. Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
✓. Those who have dorsiflexion muscle weakness
✓. Those who can comply with the requirements for clinical trials
✓. For women of childbearing potential, those who have a negative urine pregnancy test at screening
✓. Those who use a medically acceptable method of contraception until clinical trial visit 7 (short-term follow-up visit, 16 weeks): hormonal contraception, intrauterine device (IUD), intrauterine system (IUS), vasectomy, tubal ligation, or double barrier method using a cervical cap or a diaphragm with a male condom.
✓. Those who voluntarily agree to participate in this study and sign an IRB-approved consent form after being informed about the characteristics of this clinical trial prior to all screening tests

Exclusion criteria

✕. Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
✕. Patients diagnosed with type 1 or type 2 diabetes
✕. Those with a history of stroke or cerebral ischemic attack within 12 months of screening
✕. Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
✕. Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of screening
✕. Those who have ankle contractures or have surgery that may affect muscle strength assessment
✕. Those who have experience with stem cell therapy or gene therapy before screening
✕. Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)

What they're measuring

Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product

Timeframe: Week 16 after treatment

Determination of Dose-limiting toxicity (DLT) levels of EN001

Timeframe: Up to Week 4 after dosing on Day 0

Determination of Maximum tolerated dose (MTD) levels of EN001

Timeframe: Up to Week 4 after dosing on Day 0

Number of participants with Vital Signs abnormalities

Timeframe: From screening up to Week 16

Number of participants with clinically significant abnormalities of Physical Examinations

Timeframe: From screening up to Week 16

Number of participants with abnormalities of Laboratory Parameters

Timeframe: From screening up to Week 16

Number of participants with 12-lead Electrocardiography (ECG) abnormalities

Timeframe: From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion)