Duchenne Muscular DystrophyPhase 1/2NCT06328725
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Phase 1/2 study testing EN001 in people with Duchenne Muscular Dystrophy, run by ENCell at 2 sites in South Korea.
Trial summary
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy Read more about this study
- Who is it for?
- Ages 6 to 11 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- 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
- 88 participants
- Where
- 2 sitesSouth Korea
- Study dates
- Mar 2024to Nov 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 6 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males aged between 6 and 11 years at the time of providing written consent.
- Individuals exhibiting phenotypic signs of Duchenne Muscular Dystrophy (DMD), such as lower limb muscle weakness, a duck walk, or Gower's sign, and who are diagnosed with DMD following confirmation of a dystrophin gene mutation through genetic testing.
- Participants who meet the Time to Stand Test (TTSTAND) criteria without the use of assistive devices or help from others during screening and baseline assessments:
- Phase 1: Capable of completing the TTSTAND evaluation.
- Phase 2: TTSTAND time of 10 seconds or less.
- Participants with a 6-Minute Walk Test (6MWT) result of 75 meters or more at screening and baseline.
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- Individuals who meet the following laboratory test criteria at the time of screening and baseline:
- Hemoglobin ≥10 g/dL
- Platelet ≥50,000/μL
- Serum albumin ≥2.5 g/dL
- Gamma glutamyl transferase (γ-GT) and total bilirubin ≤ upper limit of normal (ULN)
- Serum creatinine ≤ 1.5 x ULN
- Participants who have been on a stable dose of glucocorticoids for at least 12 weeks prior to screening, with treatment maintained. Dosage adjustments for body weight changes are allowed.
- Individuals who, along with their representatives when applicable, have voluntarily agreed in writing to participate in this clinical trial.
Who cannot join
- Individuals with confirmed comorbidities at the time of screening:
- Left ventricular ejection fraction (LVEF) below 50%, as determined by echocardiography
- Percent predicted forced vital capacity (FVC%) less than 35%
- Positive for Hepatitis B surface antigen (HBsAg). However, individuals undergoing interferon or antiviral treatment can register
- Positive for Hepatitis C virus antibody (HCV Ab). Registration is possible if the HCV ribonucleic acid (RNA) test result is negative
- Positive for Human immunodeficiency virus (HIV) antibody
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- Comorbidities that are uncontrollable or require treatment that could affect the safety and efficacy evaluation of this clinical trial, based on the investigator's judgment
- Individuals with confirmed treatment history at the time of screening:
- Administration of cell therapy or gene therapy throughout life
- Administer antisense oligonucleotide (e.g., exon skipping treatment) or stop- codon readthrough treatment (e.g., aminoglycoside, ataluren) within 24 weeks before screening.
- Administration of the following medications within 12 weeks before screening: Idebenone, Resveratrol, Adenosine triphosphate
- Administration of the following medications within 12 weeks before screening. However, registration is possible if the drug is being administered at a stable dose for at least 12 weeks before screening and the dose is expected to remain unchanged during the clinical trial period. Angiotensin-converting enzyme (ACE) inhibitor Angiotensin II receptor blocker (ARB) Beta-blocker Aldosterone antagonist Ivabradine Sacubitril Growth hormone Anabolic steroids
- Major surgery within 12 weeks before screening or expected major surgery during the clinical trial period.
- Use of other investigational products (or medical devices) within 4 weeks before screening.
- Use of systemic immunosuppressants other than systemic glucocorticoids.
- Individuals requiring mechanical ventilation during the day.
- Persons with hypersensitivity to the components of the clinical investigational products.
- Individuals unwilling to use appropriate contraception from the date of written consent to the termination visit:
- Appropriate contraceptive methods are as follows, and use more than one method.
- The use of hormonal contraceptives by the partner
- Implantation of an intrauterine device or system in your partner
- Sterilization or surgical procedures for you or your partner
- Others who, in the investigator's discretion, are not willing or able to comply with the clinical trial procedures.
Trial locations and contacts
2 sites in 1 country.
South Korea2 sites
Samsung Medical Center
Seoul
Jeehun Lee
Seoul National University Hospital
Seoul
Jong-Hee Chae
About this study
Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
Read the detailed description
This clinical trial is a multi-center study conducted in two phases: Phase 1 and Phase 2. Phase 1 follows a 3+3 dose-escalation design to assess the safety, efficacy, and tolerability of EN001, an investigational product. Phase 2 evaluates the efficacy and safety of EN001 at the recommended phase 2 dose (RP2D), as determined in Phase 1, compared to a placebo.
Phase 1 is designed using the traditional 3+3 dose-escalation method to determine the maximum tolerated dose (MTD) and establish the RP2D. Dose escalation continues until the MTD is identified, which must be within the maximum planned dose (MPD) of 2.5 x 10^6 cells/kg (Cohort 2) or lower. The MTD is defined as the highest dose at which the incidence rate of dose-limiting toxicity (DLT) is less than 33%. To determine the MTD, 3-6 subjects are enrolled in each dose cohort. They receive EN001 every 6 weeks for 3 cycles, with DLTs evaluated up to the 2-week time point (Visit 7).
The Safety Review Committee (SRC) consists of the coordinating Investigator, the responsible trial monitor for subjects enrolled in cohorts requiring safety review, and the sponsor. These members participate as committee members. At the conclusion of each cohort-defined as the endpoint of the DLT assessment for the last subject in that cohort-they comprehensively review the safety data for EN001. The committee makes decisions related to dose adjustments, whether to increase or decrease the dose, and ultimately determines the RP2D.
Phase 2 clinical trials are randomized, double-blind, placebo-controlled clinical trials.
In phase 2, eligible subjects will be randomly assigned to the test group (recommended phase 2 dose (RP2D) of EN001) or the control group (placebo of EN001) in a 1:1 ratio. Efficacy and safety will be evaluated up to 48 weeks after EN001 administration compared to placebo.
In addition, test subjects participating in phase 1 and phase 2 will be followed up for safety and effectiveness for 5 years from the time of EN001 administration according to the long-term follow-up protocol.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- EN001
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EN001Drug
Also called EN001(allogenic early-passage mesenchymal stem cells derived from Wharton's jelly (WJ-MSCs) )
Phase 1
- Cohort 1: EN001 5.0x10^5 cells/kg administered intravenously (IV) 3 times at 6 week intervals.
- Cohort 2: EN001 2.5x10^6 cells/kg administered intravenously (IV) 3 times at 6 week intervals.
Phase 2
- Experimental Group: The recommended phase 2 dose (RP2D) of EN001 is administered intravenously (IV) three times at six-week intervals.
- Control Group: EN001 placebo is administered intravenously (IV) three times at six-week intervals.
| Study group | Role | What they receive |
|---|---|---|
| Phase 1 - Cohort 1 | Active comparator | EN001 5.0x10^5 cells/kg Receives: Drug: EN001 |
| Phase 1 - Cohort 2 | Active comparator | EN001 2.5x10^6 cells/kg Receives: Drug: EN001 |
| Phase 2 - Experimental Group | Placebo comparator | The recommended phase 2 dose (RP2D) of EN001 Receives: Drug: EN001 |
| Phase 2 - Control Group | Placebo comparator | EN001 placebo Receives: Drug: EN001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
<Phase 1> Adverse drug reactions related to dose limiting toxicity (DLT)
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.
Measured: Up to 14 weeks
<Phase 1> Adverse drug reactions related to discontinuation of clinical trial drug administration
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.
Measured: Up to 14 weeks
<Phase 2> Change in time to stand test (TTSTAND)
Present the changes in time to stand test (TTSTAND) at the 48-week time point compared to baseline (Visit 2). Provide the subject count, mean, standard deviation, median, minimum, and maximum for the change in each treatment group. Analyze the change as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline TTSTAND values and age as fixed effects in the analysis.
Measured: At 48 weeks compared to baseline (Visit 2)
Secondary outcomes (33)
<Phase 1> Time to stand test (TTSTAND) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTSTAND velocity (1/TTSTAND) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Time to run/walk 10 meters test (TTRW) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36,, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTRW velocity (1/TTRW) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> North Star Ambulatory Assessment (NSAA) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Time to climb 4 steps test (TTCLIMB) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTCLIMB velocity (1/TTCLIMB) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> 6-minute walk test (6MWT) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in muscle strength by region
The changes in shoulder abduction, elbow flexion/extension, knee flexion/extension, and handgrip are evaluated using hand-held myometry. (Unit: lbs)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in parameters related to pulmonary function
Through spirometry testing, % predicted forced vital capacity (FVC%), forced vital capacity (FVC, unit L), forced expiratory volume in one second (FEV1), peak expiratory flow rate (PEFR), maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP).
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, 24, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in parameters related to cardiac function
Through echocardiography, changes in left ventricular ejection fraction (LVEF), fractional shortening (FS), and left ventricular end-diastolic diameter (LVEDd) are evaluated.
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: At 48 weeks compared to screening (Visit 1)
<Phase 1> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2)
Present the subject count, mean, standard deviation, median, minimum, and maximum for the changes at each time point within each dosage group.
Measured: From baseline
<Phase 2> Time to stand test (TTSTAND) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, and 36 weeks compared to baseline (Visit 2)
<Phase 2> TTSTAND velocity (1/TTSTAND) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Time to run/walk 10 meters test (TTRW) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> TTRW velocity (1/TTRW) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> North Star Ambulatory Assessment (NSAA) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Time to climb 4 steps test (TTCLIMB) change
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> TTCLIMB velocity (1/TTCLIMB) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> 6-minute walk test (6MWT) change amount
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount in muscle strength by region
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount and rate of change in whole thigh muscle volume and index assessed by MRI
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 48 weeks compared to screening (Visit 1)
<Phase 2> Changes amount in parameters related to pulmonary function
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 12, 24, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount in parameters related to cardiac function
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 48 weeks compared to screening (Visit 1)
<Phase 2> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2)
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: From baseline
<Phase 2> Pediatric Outcomes Data Collection Instrument (PODCI) item score and total score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 24 and 48 weeks compared to baseline (Visit 2)
<Phase 2> Pediatric Quality of Life inventory™ (PedsQL™) item scores and total score change
Present the subject count, mean, standard deviation, median, minimum, and maximum for changes at each time point and for each dosage group. Analyze the change (or percentage change) as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline (or screening) values of each assessment variable, age, and a stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as fixed effects in the analysis. However, for variables related to TTSTAND, do not include the stratification factor (TTSTAND ≤ 8 seconds / > 8 seconds) as a fixed effect.
Measured: At 24 and 48 weeks compared to baseline (Visit 2)
<Phase 1> Adverse Event
After the application of the investigational medicinal product in the clinical trial, provide the number of subjects, incidence rate, and number of occurrences for adverse events, drug-related adverse events, serious adverse events, serious drug-related adverse events, adverse events related to discontinuation of the investigational medicinal product, drug-related adverse events related to discontinuation of the investigational medicinal product, and injection-related adverse events, categorized by dosage group. Additionally, code the occurrences by system organ class (SOC) and preferred term (PT) using the Medical Dictionary for Regulatory Activities (MedDRA), and present the number of subjects, incidence rate, and number of occurrences for each dosage group according to SOC and PT.
Measured: Up to 48weeks. 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.
<Phase 1> 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 48weeks. At Baseline and Week 4, tests are conducted before and within 4 hours after administration of the investigational drug, and PT INR and aPTT are performed only at the screening visit.
<Phase 1> 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.
For changes at each time point within each dosage group, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide frequency and percentage.
Measured: Up to 48weeks.
<Phase 2> Adverse Event
After the administration of the investigational product in the clinical trial, provide the number of subjects, incidence rate, and number of occurrences for adverse events, drug-related adverse events, serious adverse events, serious drug-related adverse events, adverse events related to discontinuation of the investigational product, drug-related adverse events related to discontinuation of the investigational product, and injection-related adverse events, categorized by treatment group. Additionally, code the occurrences by system organ class (SOC) and preferred term (PT) using the Medical Dictionary for Regulatory Activities (MedDRA), and present the number of subjects, incidence rate, and number of occurrences for each treatment group according to SOC and PT.
Measured: Up to 48weeks. 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.
<Phase 2> 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 48weeks. At Baseline and Week 4, tests are conducted before and within 4 hours after administration of the investigational drug, and PT INR and aPTT are performed only at the screening visit.
<Phase 2> 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.
For changes at each time point within each treatment group, present continuous variables with subject count, mean, standard deviation, median, minimum, and maximum. For categorical variables, provide frequency and percentage.
Measured: Up to 48weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2024
- Study startMar 2024 (estimated)
- Primary completionNov 2025 (estimated)
- Study completionNov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 Clinical Trial\> A total of 6-12 participants (3-6 participants per cohort). \<Phase 2 Clinical Trial\> A total of 76 participants (a minimum of 30 participants per group, considering a dropout rate of 20%, resulting in 38 participants per group).
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 88 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ENCell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06328725
- Sponsor's protocol ID
- EN001_POWER
- Status last verified
- Mar 2024
- First submitted
- 19 Feb 2024
- Last updated
- 25 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06328725
- Acronym / sponsor ID
- EN001_POWER
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 88 participants (estimated)
- Lead sponsor
- ENCellIndustry (pharma, biotech, devices)
- Study start
- Mar 2024 (estimated)
- Primary completion
- Nov 2025 (estimated)
- Study completion
- Nov 2025 (estimated)
- First posted
- 25 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
Detailed description
This clinical trial is a multi-center study conducted in two phases: Phase 1 and Phase 2. Phase 1 follows a 3+3 dose-escalation design to assess the safety, efficacy, and tolerability of EN001, an investigational product. Phase 2 evaluates the efficacy and safety of EN001 at the recommended phase 2 dose (RP2D), as determined in Phase 1, compared to a placebo.
Phase 1 is designed using the traditional 3+3 dose-escalation method to determine the maximum tolerated dose (MTD) and establish the RP2D. Dose escalation continues until the MTD is identified, which must be within the maximum planned dose (MPD) of 2.5 x 10^6 cells/kg (Cohort 2) or lower. The MTD is defined as the highest dose at which the incidence rate of dose-limiting toxicity (DLT) is less than 33%. To determine the MTD, 3-6 subjects are enrolled in each dose cohort. They receive EN001 every 6 weeks for 3 cycles, with DLTs evaluated up to the 2-week time point (Visit 7).
The Safety Review Committee (SRC) consists of the coordinating Investigator, the responsible trial monitor for subjects enrolled in cohorts requiring safety review, and the sponsor. These members participate as committee members. At the conclusion of each cohort-defined as the endpoint of the DLT assessment for the last subject in that cohort-they comprehensively review the safety data for EN001. The committee makes decisions related to dose adjustments, whether to increase or decrease the dose, and ultimately determines the RP2D.
Phase 2 clinical trials are randomized, double-blind, placebo-controlled clinical trials.
In phase 2, eligible subjects will be randomly assigned to the test group (recommended phase 2 dose (RP2D) of EN001) or the control group (placebo of EN001) in a 1:1 ratio. Efficacy and safety will be evaluated up to 48 weeks after EN001 administration compared to placebo.
In addition, test subjects participating in phase 1 and phase 2 will be followed up for safety and effectiveness for 5 years from the time of EN001 administration according to the long-term follow-up protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
- Model description
- <Phase 1 Clinical Trial> A total of 6-12 participants (3-6 participants per cohort). <Phase 2 Clinical Trial> A total of 76 participants (a minimum of 30 participants per group, considering a dropout rate of 20%, resulting in 38 participants per group).
| Arm | Type | Receives |
|---|---|---|
| Phase 1 - Cohort 1 | Active comparator | Drug: EN001EN001 5.0x10^5 cells/kg |
| Phase 1 - Cohort 2 | Active comparator | Drug: EN001EN001 2.5x10^6 cells/kg |
| Phase 2 - Experimental Group | Placebo comparator | Drug: EN001The recommended phase 2 dose (RP2D) of EN001 |
| Phase 2 - Control Group | Placebo comparator | Drug: EN001EN001 placebo |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- Males aged between 6 and 11 years at the time of providing written consent.
- Individuals exhibiting phenotypic signs of Duchenne Muscular Dystrophy (DMD), such as lower limb muscle weakness, a duck walk, or Gower's sign, and who are diagnosed with DMD following confirmation of a dystrophin gene mutation through genetic testing.
- Participants who meet the Time to Stand Test (TTSTAND) criteria without the use of assistive devices or help from others during screening and baseline assessments:
- Participants with a 6-Minute Walk Test (6MWT) result of 75 meters or more at screening and baseline.
- Individuals who meet the following laboratory test criteria at the time of screening and baseline:
Key exclusion criteria (23 listed)
- Individuals with confirmed comorbidities at the time of screening:
- Individuals with confirmed treatment history at the time of screening:
- Individuals requiring mechanical ventilation during the day.
- Persons with hypersensitivity to the components of the clinical investigational products.
- Individuals unwilling to use appropriate contraception from the date of written consent to the termination visit:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EN001Also: EN001(allogenic early-passage mesenchymal stem cells derived from Wharton's jelly (WJ-MSCs) )Phase 1 * Cohort 1: EN001 5.0x10^5 cells/kg administered intravenously (IV) 3 times at 6 week intervals. * Cohort 2: EN001 2.5x10^6 cells/kg administered intravenously (IV) 3 times at 6 week intervals. Phase 2 * Experimental Group: The recommended phase 2 dose (RP2D) of EN001 is administered intravenously (IV) three times at six-week intervals. * Control Group: EN001 placebo is administered intravenously (IV) three times at six-week intervals. | Drug | Phase 1 - Cohort 1, Phase 1 - Cohort 2, Phase 2 - Control Group, Phase 2 - Experimental Group |
Primary outcome
<Phase 1> Adverse drug reactions related to dose limiting toxicity (DLT)
Time frame: Up to 14 weeks
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.
<Phase 1> Adverse drug reactions related to discontinuation of clinical trial drug administration
Time frame: Up to 14 weeks
Present the frequency and percentage of dose-limiting toxicity (DLT) occurrence across dose cohorts, along with detailed information on the types of DLTs.
<Phase 2> Change in time to stand test (TTSTAND)
Time frame: At 48 weeks compared to baseline (Visit 2)
Present the changes in time to stand test (TTSTAND) at the 48-week time point compared to baseline (Visit 2). Provide the subject count, mean, standard deviation, median, minimum, and maximum for the change in each treatment group. Analyze the change as the dependent variable using a repeated measures mixed model (MMRM) with treatment group, visit (6, 12, 18, 24, 36, 48 weeks), and the interaction between treatment group and visit as factors. Include baseline TTSTAND values and age as fixed effects in the analysis.
33 secondary outcomes
<Phase 1> Time to stand test (TTSTAND) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTSTAND velocity (1/TTSTAND) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Time to run/walk 10 meters test (TTRW) change amount
Time frame: At 6, 12, 18, 24, 36,, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTRW velocity (1/TTRW) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> North Star Ambulatory Assessment (NSAA) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Time to climb 4 steps test (TTCLIMB) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> TTCLIMB velocity (1/TTCLIMB) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> 6-minute walk test (6MWT) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in muscle strength by region
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in parameters related to pulmonary function
Time frame: At 12, 24, and 48 weeks compared to baseline (Visit 2)
<Phase 1> Changes amount in parameters related to cardiac function
Time frame: At 48 weeks compared to screening (Visit 1)
<Phase 1> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2)
Time frame: From baseline
<Phase 2> Time to stand test (TTSTAND) change amount
Time frame: At 6, 12, 18, 24, and 36 weeks compared to baseline (Visit 2)
<Phase 2> TTSTAND velocity (1/TTSTAND) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Time to run/walk 10 meters test (TTRW) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> TTRW velocity (1/TTRW) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> North Star Ambulatory Assessment (NSAA) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Time to climb 4 steps test (TTCLIMB) change
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> TTCLIMB velocity (1/TTCLIMB) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> 6-minute walk test (6MWT) change amount
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount in muscle strength by region
Time frame: At 6, 12, 18, 24, 36, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount and rate of change in whole thigh muscle volume and index assessed by MRI
Time frame: At 48 weeks compared to screening (Visit 1)
<Phase 2> Changes amount in parameters related to pulmonary function
Time frame: At 12, 24, and 48 weeks compared to baseline (Visit 2)
<Phase 2> Changes amount in parameters related to cardiac function
Time frame: At 48 weeks compared to screening (Visit 1)
<Phase 2> Change rate of creatine kinase (CK) at each visit after administration of investigational product compared to baseline (Visit 2)
Time frame: From baseline
<Phase 2> Pediatric Outcomes Data Collection Instrument (PODCI) item score and total score change
Time frame: At 24 and 48 weeks compared to baseline (Visit 2)
<Phase 2> Pediatric Quality of Life inventory™ (PedsQL™) item scores and total score change
Time frame: At 24 and 48 weeks compared to baseline (Visit 2)
<Phase 1> Adverse Event
Time frame: Up to 48weeks. 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.
<Phase 1> Laboratory examination
Time frame: Up to 48weeks. At Baseline and Week 4, tests are conducted before and within 4 hours after administration of the investigational drug, and PT INR and aPTT are performed only at the screening visit.
<Phase 1> Vital sign
Time frame: Up to 48weeks.
<Phase 2> Adverse Event
Time frame: Up to 48weeks. 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.
<Phase 2> Laboratory examination
Time frame: Up to 48weeks. At Baseline and Week 4, tests are conducted before and within 4 hours after administration of the investigational drug, and PT INR and aPTT are performed only at the screening visit.
<Phase 2> Vital sign
Time frame: Up to 48weeks.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing EN001 in people with Duchenne Muscular Dystrophy, run by ENCell at 2 sites 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 14 inclusion and 23 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: status not confirmed, as of the sponsor's last update on 25 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Results are not available in the source record.
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?
<Phase 1> Adverse drug reactions related to dose limiting toxicity (DLT) (time frame: Up to 14 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Mar 2024.