Evaluate the Efficacy and Safety of EN001 in Patients With Sarcopenia

NCT07808905 | Not Yet Recruiting Phase 1

• 1 other identifier: EN001_SCT12
• Study Type: interventional
• Target: 132
• Locations: 1 country
• Sites: 1

Brief Summary

A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Clinical Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Sarcopenia

Conditions

Sarcopenia

Keywords

EN001; Sarcopenia; ENCell; MSC

Outcome Measures

Primary Outcomes (2)

• [Phase1] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration

  Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs. Adverse events related to discontinuation of clinical investigational drug administration, discontinuation of clinical investigational drug administration Regarding related adverse drug reactions, the number of subjects in each cohort, incidence rate (%), and Two-sided 95% confidence intervals and number of occurrences are presented.

  Up to 12 weeks

• [Phase2] Change from baseline in the SPPB(Short Physical Performance Battery) at Week 24

  For all efficacy outcome measures, descriptive statistics (number of subjects, mean, standard deviation, median, minimum, and maximum) will be presented by treatment group and assessment time point, along with two-sided 95% confidence intervals.

  at Week 24

Secondary Outcomes (13)

• [Phase1, Phase2] SPPB(Short Physical Performance Battery) score change

  At 4,8,12, 18, and 24 weeks compared to baseline(Visit2)

• [Phase1, Phase2] Change in handgrip strength

  At 4,8,12, 18, and 24 weeks compared to baseline(Visit2)

• [Phase1, Phase2] Change in time to complete the 5-Times Chair Stand Test

  At 4,8,12, 18, and 24 weeks compared to baseline(Visit2)

• [Phase1, Phase2] Change in 6-Minute Walk Test (6MWT) distance

  At 4,8,12, 18, and 24 weeks compared to baseline(Visit2)

• [Phase1, Phase2] Change in Appendicular Skeletal Muscle Mass (ASM) measured by DXA

  At 12 and 24 weeks compared to baseline(Visit2)

Study Arms (6)

Phase1 - Cohort 1

ACTIVE COMPARATOR

EN001 Low dose

Drug: EN001

Phase1 - Cohort 2

ACTIVE COMPARATOR

EN001 Medium dose

Drug: EN001

Phase1 - Cohort 3

ACTIVE COMPARATOR

EN001 High dose

Drug: EN001

Phase2 - Cohort 1

ACTIVE COMPARATOR

EN001 Low dose

Drug: EN001

Phase2 - Cohort 2

ACTIVE COMPARATOR

EN001 High dose

Drug: EN001

Phase2 - Placebo

PLACEBO COMPARATOR

EN001 Placebo

Drug: EN001 Placebo

Interventions

EN001 Placebo DRUG

-Phase2-Placebo : EN001 Placebo administered intravenously (IV) 3times at 4 week intervals.

Also known as: EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs) )

Phase2 - Placebo

EN001 DRUG

\- Phase1-Cohort 1: EN001 Low dose administered intravenously (IV) 3 times at 4 week intervals.

Also known as: EN001(Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells(WJ-MSCs)

Phase1 - Cohort 1

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \) Male or Female aged 60 years or older at the time of providing written consent. : Female subjects must meet one of the following criteria for postmenopausal women\* :
• Postmenopausal women:
• Natural amenorrhea for 12 months
• At least 6 weeks after surgical menopause (bilateral oophorectomy)
• \) Individuals diagnosed with sarcopenia at the time of screening, based on the diagnostic criteria in Section 15.1 of the Asian Working Group for Sarcopenia(AWGS) 2019 guidlines, meeting (1) or (2) together with (3):
• (1) Handgrip Strength: \< 28kg(men), \<18kg(women)
• (2) 6-Metre Walk test\<1.0m/s, or 5-Times Chair Stand Test ≥12seconds, or Short Physical Performance Battery(SPPB)≤9points
• (3) Appendicular Skeletal Muscle Mass(ASM) by dual-energy X-ray absorptiometry(DXA):7.0kg/m\^2(men), \<5.4kg/m\^2(women)
• \) Individuals with a nutritional status score of ≥8points on the Screening component of the Long Mini Nutritional Assessment (Long MNA) at the time of screening
• \) Individuals with a body weight of ≥35 kg and a Body Mass Index (BMI) of ≥15 kg/m² and \<30 kg/m² at the time of screening
• \) Individuals who have stably adhered to the diet and exercise regimen provided as part of this clinical trial during a run-in period of at least 4 weeks, and who are willing to comply with the regimen throughout the trial period
• \) Individuals meeting the following laboratory test criteria at the time of screening and baseline:
• Hemoglobin ≥10 g/dL,
• White Blood Cell ≥2,500/μL,
• Platelet ≥100,000/μL,

You may not qualify if:

• )Those with the following comorbidities confirmed at the time of screening and baseline
• Patients with chronic kidney disease(CKD-EPI;Estimated Glomerular Filtration Rate(eGFR) \<30 mL/min)
• Individuals with uncontrolled thyroid disease(hyperthyroidism or hypothyroidism)
• Patients with uncontrolled diabetes(HbA1c \>8.5%)
• Individuals with a neuromuscular or neurological disease (e.g., Parkinson's disease, amyotrophic lateral sclerosis \[ALS\], stroke affecting lower extremity function, muscular dystrophy, epilepsy, multiple sclerosis, etc.)
• Individuals with severe cardiac disease classified as New York Heart Association (NYHA) Functional Classification III or IV (cardiomyopathy, unstable angina, myocardial infarction, valvular heart disease, arrhythmia, aortic disease, etc.)
• Patients with uncontrolled hypertension((Systolic Blood Pressure (SBP) \>180 mmHg or Diastolic Blood Pressure (DBP) \>110 mmHg)
• Any one of the serum virus tests (HBsAg, anti-HBc, anti-HCV, HIV Ag/Ab) is positive
• (If anti-HBc positive) However, registration is possible if the HBV DNA test result is negative.
• (If anti-HCV positive) However, registration is possible if the HCV RNA test result is negative.
• Any malignancy within 5 years before screening (excluding basal cell carcinoma or squamous cell carcinoma of the skin, localized prostate cancer, or carcinoma in situ of the cervix may be enrolled even if the 5-year period has not elapsed, provided the investigator determines that the condition has been successfully treated and is in complete remission)
• Any medical condition or history of surgery within 24 weeks prior to screening that may affect physical activity or function, including walking or exercise (e.g., fracture, severe knee or hip osteoarthritis, peripheral vascular disease)
• \) Subjects with a confirmed history of the following treatments:
• Systemic corticosteroids equivalent to prednisone \>10 mg/day at screening
• Any of the following medications that may affect muscle at screening:

Sponsors & Collaborators

• ENCell: lead

Study Sites (1)

Samsung Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Sarcopenia

Condition Hierarchy (Ancestors)

Muscular Atrophy; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Atrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Central Study Contacts

ENCell

CONTACT

+82-2-6205-8054; encell@encellinc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The Phase 1 study is conducted as an open-label trial, whereas the Phase 2 study is designed as a randomized, double-blind trial.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase 1 Design : 3+3 Dose-escalation, Open label, Single-center / Phase 2 Design : Randomized, Double-blind, Multi-center

Study Record Dates

• First Submitted: September 2, 2026
• First Posted: September 9, 2026
• Study Start (Estimated): March 1, 2027
• Primary Completion (Estimated): October 31, 2029
• Study Completion (Estimated): October 31, 2029
• Last Updated: September 11, 2026

Record last verified: 2026-09

Locations

KR (1)