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SarcopeniaPhase 1/2NCT07808905

Evaluate the Efficacy and Safety of EN001 in Patients With Sarcopenia

A Phase 1/2 study testing EN001 in people with Sarcopenia, run by ENCell at 1 site in South Korea.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial 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 Read more about this study

Who is it for?
Ages 60 years and older
Conditions the study is for
Sarcopenia
What is being tested?
EN001
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
132 participants
Where
1 siteSouth Korea
Study dates
1 Mar 2027to 31 Oct 2029
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 60 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 1) 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)
  • 2) 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)
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  • (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)
  • 3) 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
  • 4) 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
  • 5) 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
  • 6) 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,
  • AST or ALT ≤3 × ULN,
  • International Normalized Ratio (INR) ≤1.3
  • 7) Men who have agreed to use the appropriate contraceptive method(s), as specified in the protocol, from the date of signing the informed consent form through the end-of-study visit
  • Appropriate contraception is defined as follows and is achieved by applying one or more methods of contraception :
  • Partner's use of hormonal contraceptives
  • Partner's placement of an intrauterine device(IUD) or intrauterine system(IUS)
  • Sterilization procedure or surgery in the participant or partner
  • Double contraceptive method: use of a male condom in combination with other contraceptive methods [hormonal contraceptives, IUD/IUS placement, or sterilization procedure/surgery)
  • Abstinence: Absolute abstinence. If, in the examiner's judgment, the subject's age, occupation, lifestyle, or sexual orientation warrants contraception, strict abstinence from sexual intercourse is also acceptable. However, periodic abstinence (e.g. Karenda method, ovulation method, symptomatic temperature method), abstinence, and external vaginal ejaculation are not recognized as appropriate contraceptive methods.
  • 8) Individuals who(or whose legally authorized representative) have voluntarily agreed to participate in this clinical trial.

Who cannot join

  • 1)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.)
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  • 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)
  • 2) 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:
  • Growth hormones (e.g., insulin-like growth factor-1 [IGF-1] analogues, somatropin)
  • Sex hormones (e.g., testosterone, estrogen, dehydroepiandrosterone [DHEA])
  • Ghrelin or megestrol acetate
  • Myostatin or myostatin inhibitors (e.g., stamulumab, domagrozumab, landogrozumab)
  • Bimagrumab
  • β-receptor blockers, ACE inhibitors, TNF-α antagonists, or GLP-1 agonists (Subjects receiving any of these medications at a stable dose for ≥12 weeks prior to screening and who plan to continue the treatment throughout the study may be enrolled.)
  • Troponin Activator
  • Prior treatment with any cell therapy or gene therapy at any time in their lifetime
  • Those who have received other investigational medicinal products or medical devices within 4 weeks prior to screening
  • 3) Subjects with a Korean version of Montreal Cognitive Assessment (MoCA-K) score of less than 23 at screening
  • 4) Persons with hypersensitivity to the components of clinical investigational products
  • 5) Those who are unable to read or write
  • 6) Those who are deemed inappropriate to participate in clinical trials according to the judgment of the investigator*
  • Examples of inappropriate reasons include, but are not limited to, the following:
  • History of drug or alcohol dependence or abuse
  • Medical history (e.g., autoimmune diseases such as myositis, dystonia, etc.) or surgical history that may affect efficacy evaluations, other than those specified in the inclusion/exclusion criteria
  • Subjects who are currently participating in, or plan to participate in, high-intensity bodybuilding, muscle-building, or muscle-strengthening programs (However, routine physical activity is not restricted.)

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

ENCell contact

+82-2-6205-8054

encell@encellinc.com

South Korea1 site

  • Samsung Medical Center

    Seoul

    Jong-Geol Do

Other trials in South Korea

About this study

Official title: 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

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

Read the detailed description

This clinical trial is a multicenter study consisting of two stages (Phase 1 and Phase 2). Phase 1 is designed with a 3+3 dose escalation design to evaluate the safety, including tolerability, of EN001 and explore efficacy. Phase 2 will evaluate the efficacy and safety of EN001 at the recommended Phase 2 dose (RP2D), as determined in Phase 1, compared with placebo.

--Phase 1 The study was designed using the traditional 3+3 dose escalation method to confirm the maximum tolerated dose (MTD) and determine the recommended phase 2 dose (RP2D).

Dose increase is carried out until the maximum tolerated dose (MTD) is confirmed at the high dose (Cohort 3), which is the maximum planned dose (MPD), or at a lower dose. The maximum tolerated dose (MTD) is defined as the highest dose at which the incidence of dose limiting toxicity (DLT) is lower than 33%. To determine the maximum tolerated dose (MTD), 3-6 test subjects from each dose cohort are enrolled and EN001 is administered three times at 4-week intervals, and dose-limiting toxicity (DLT) is evaluated until 4 weeks (visit 5).

Safety data for EN001 confirmed by the end of each Cohort (i.e., the end of the dose-limiting toxicity (DLT) evaluation of the last dosed subject in the Cohort) are comprehensively reviewed to determine all matters related to dose, such as increase or decrease in dose, and finally the recommended phase 2 dose (RP2D) is determined.

-- Phase 2 The Phase 2 is a randomized, double-blind, placebo-controlled clinical trial. Eligible subjects will be randomly assigned in a 1:1:1 ratio to Study Group 1 (EN001 Low dose, which is the recommended Phase 2 dose [RP2D]), Study Group 2 (EN001 High dose), or the placebo control group. The efficacy and safety of EN001 will be evaluated in comparison with placebo.

Conditions
Sarcopenia
Being tested
EN001, EN001 Placebo
Keywords
EN001, Sarcopenia, ENCell, MSC
Medical subject headings
Sarcopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EN001Drug

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

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

  • EN001Drug

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

    -Phase2-Cohort 2 : EN001 Medium dose administered intravenously (IV) 3times at 4 week intervals.

  • EN001Drug

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

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

  • EN001Drug

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

    -Phase2-Cohort 1 : EN001 Low dose(Recommended Phase 2 Dose[RP2D] of EN001) administered intravenously (IV) 3times at 4 week intervals.

  • EN001Drug

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

    -Phase2-Cohort2 : EN001 High dose(Recommended Phase 2 Dose[RP2D] of EN001) administered intravenously (IV) 3times at 4 week intervals.

  • EN001 PlaceboDrug

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

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

Study groups in NCT07808905
Study groupRoleWhat they receive
Phase1 - Cohort 1Active comparatorEN001 Low dose
Receives: Drug: EN001
Phase1 - Cohort 2Active comparatorEN001 Medium dose
Receives: Drug: EN001
Phase1 - Cohort 3Active comparatorEN001 High dose
Receives: Drug: EN001
Phase2 - Cohort 1Active comparatorEN001 Low dose
Receives: Drug: EN001
Phase2 - Cohort 2Active comparatorEN001 High dose
Receives: Drug: EN001
Phase2 - PlaceboPlacebo comparatorEN001 Placebo
Receives: Drug: EN001 Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. [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.

    Measured: Up to 12 weeks

  2. [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.

    Measured: at Week 24

Secondary outcomes (13)

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

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

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

  2. [Phase1, Phase2] Change in handgrip strength

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

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

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

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

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

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

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

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

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

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

    Measured: At 12 and 24 weeks compared to baseline(Visit2)

  6. [Phase1, Phase2] SarQoL domain and total score change

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

    Measured: At 12 and 24 weeks compared to baseline(Visit2)

  7. [Phase1, Phase2] Change in Skeletal Muscle Mass Index(SMI)

    Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.

    Measured: At 24 weeks compared to baseline(Visit2)

  8. [Phase1, Phase 2] Adverse Event

    By group that Number of subjects, incidence rate (%), confidence interval (two-sided 95%), presents the number of occurrences. Additional, The pre-treatment adverse event that occurred before administration of clinical trial drugs is presented in detail.

    Measured: Up to 24weeks. However, adverse drug reactions that persist at the end of the clinical trial will be followed up until the possible adverse reactions are resolved or it is determined that further follow-up is not meaningful.

  9. [Phase1, Phase2] Vital sign

    Number of participants with clinically significant abnomalities in vital signs after EN001 administration. Vital Signs include blood pressure (mmHg), pulse (times/minute), respiratory rate (times/minute), and body temperature (℃) and will be assessed. By cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.

    Measured: Up to 24 weeks

  10. [Phase1, Phase2] Laboratory examination

    Number of participants with clinically significant abnormalities in Laboratory parameters after EN001 administration. Hematological tests, Blood chemical tests, Blood coagulation test, Urine test, Serum virus test. It is conducted through blood and urine collection, and the PI checks whether the test results are normal, abnormal, and clinically significant.

    Measured: Up to 24 week. Serum virus testing is performed only during screening. At Baseline, Week4 and Week8, hematological tests, blood chemical tests, D-dimer are performed before and within 4 hours after administration of the IP.

  11. [Phase1, Phase2] electrocardiography

    Number of participants with clinically significant abnormalities in electrocardiography after EN001 administration. Based on the PR Interval, QRS Duration, QTc Interval and QTcF Interval, PI checks whether the test results are normal, abnormal, and clinically significant. by cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.

    Measured: At Baseline, Week 4, Week 8, Week 12, Week 24

  12. [Phase1, Phase2] X-ray

    by cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.

    Measured: Before administration of investigational product at Baseline, within 4 hours after completion of administration, and at Weeks 4, 8, 12, and 24

  13. [Phase1, Phase2] Physical Examinations

    Number of participants with clinically significant abnormalities in Physical Examinations after EN001 administration. Based on general appearance, head,ears/eyes/nose/throat, cardiovascular, respiratory, abdomen, skin, lymph nodes, extremities, musculoskeletal and neurologic, PI checks whether the test results are normal, abnormal, and clinically significant. By cohort and visit, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide shift table.

    Measured: Up to 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Sep 2026
  2. Study start1 Mar 2027 (estimated)
  3. Primary completion31 Oct 2029 (estimated)
  4. Study completion31 Oct 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Phase 1 Design : 3+3 Dose-escalation, Open label, Single-center / Phase 2 Design : Randomized, Double-blind, Multi-center
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
132 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07808905
Sponsor's protocol ID
EN001_SCT12
Status last verified
Sep 2026
First submitted
2 Sep 2026
Last updated
11 Sep 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing EN001 in people with Sarcopenia, run by ENCell at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact ENCell (+82-2-6205-8054, encell@encellinc.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

[Phase1] Dose limiting toxicity (DLT) and adverse drug reactions related to discontinuation of investigational product administration (time frame: Up to 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07808905.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2026.

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