CompletedPhase 1

Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A

South Korea6 participantsStarted 2024-05-30

Plain-language summary

An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A (CMT1A)

Who can participate

Age range19 Years

SexALL

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Inclusion criteria

✓. Individuals who have voluntarily agreed to participate in this clinical trial.
✓. Men and women aged 19 years or older at the time of providing written consent.
✓. Individuals who meet all of the following genetic and clinical diagnostic criteria:
✓. Genetic diagnosis: CMT1A type
✓. Clinical diagnosis:
✓. Women and men of childbearing potential who have agreed to use the appropriate contraceptive method(s) outlined in the protocol during the clinical trial period.

Exclusion criteria

✕. Those with the following comorbidities confirmed at the time of screening
✕. Subjects with neuromuscular diseases other than CMT1A or neuropathy- inducing factors (uremia) that may affect the safety and efficacy evaluation of this clinical trial, according to the judgment of the investigator.
✕. Individuals diagnosed with type 1 or type 2 diabetes
✕. Individuals diagnosed with active pulmonary tuberculosis
✕. Patients with uncontrolled hypertension (systolic blood pressure over 180 mmHg or diastolic blood pressure over 110 mmHg)
✕. Subjects with other clinically significant diseases, including significant heart, lung, liver, kidney, hematological, immunological or behavioral diseases or malignant tumors, according to the investigator's judgment
✕. Individuals who display the specified test abnormalities in laboratory tests at the time of screening:
✕. Those who have ankle contracture or have undergone surgery that may affect muscle strength measurement tests

What they're measuring

Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration

Timeframe: Up to 8 weeks

Trial details

NCT IDNCT06328712

SponsorENCell

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-11-01

View on ClinicalTrials.gov More Charcot-Marie-Tooth Disease Type 1A trials