Duchenne Muscular DystrophyPhase 1NCT05338099
Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
A Phase 1 study testing EN001 in people with Duchenne Muscular Dystrophy, run by ENCell at 1 site in South Korea.
Trial summary
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD) Read more about this study
- Who is it for?
- Ages 2 to 18 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?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteSouth Korea
- Study dates
- 18 Jan 2022to 28 Dec 2022
- Phase
- Phase 1
- 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 2 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Those aged 2 to 18 years old
- Male
- Those who are diagnosed with DMD due to a mutation in the dystrophin gene identified by a genetic test
- Phenotypic evidence of DMD
- Clinical signs or symptoms (proximal weakness, waddling gait, Gowers maneuver)
- Elevated serum creatine kinase level
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- Those who have been using systemic corticosteroids at a stable dose for 24 weeks prior to screening and are expected to maintain the constant dose throughout the study period
- Those who agree to use effective contraceptive measures until the short-term follow-up period of the clinical trial. In addition, their partner must also use a medically acceptable method of contraception (ie, oral contraceptives for women) for the same period.
- Those who are willing to agree with the ICF and whose parent or representative is willing to provide written consent for the subject's participation in the clinical trial
Who cannot join
- Those who have clinical signs or symptoms of cardiomyopathy, defined as LVEF <50% on echocardiography at screening
- If ventilatory support is required during the day or if invasive mechanical ventilation via tracheostomy is used (Non-invasive ventilation such as positive pressure ventilation is allowed at night)
- If hepatitis B core antibody and hepatitis C antibody are positive
- If there is a history of major surgery within 12 weeks or it is expected during the study period
- Those who have been exposed to gene therapy or genome editing within 24 weeks from the screening
- Those who have experience with stem cell therapy
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- Those who have been administered Translarna granules (Ataluren) within 24 weeks from the screening
- Those who are receiving treatment (other than corticosteroids) that may affect muscle strength or function within 12 weeks prior to screening
- If laboratory test values are abnormal at the time of screening
- Hemoglobin <10 g/dL
- Serum albumin <2.5 g/dL
- Platelet count <50,000/ml
- Abnormal GGT or total bilirubin (>laboratory's upper limit of normal)
- Abnormal renal function (Serum creatinine >1.5 Times laboratory's upper limit of normal)"
- Those with significant neuromuscular or genetic diseases other than DMD
- Those with significant heart, lung, liver, kidney, hematological, immunological, behavioral disease, or other clinically significant diseases including malignant tumors
- Those who have a previous or current medical condition that may adversely affect the safety of the subject, make it difficult to complete treatment, or affect the evaluation of clinical trial results at the discretion of the investigator
- Those who do not have the will or ability to comply with clinical trial procedures at the discretion of the investigator
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
Read the detailed description
It is the first in human (FIH), 3+3 design clinical trial to evaluate the safety and tolerability and determine the maximum tolerated dose (MTD) of EN001 (allogeneic umbilical cord-derived mesenchymal stem cells) in the treatment of Duchenne Muscular Dystrophy (DMD).
- 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 (Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells)
EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows.
Dose group A (Low dose): 5.0x10^5 cells/kg
EN001Drug
Also called EN001 (Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells)
EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows.
Dose group B (High dose): 2.5x10^6 cells/kg
| Study group | Role | What they receive |
|---|---|---|
| Dose group A (Low dose) | Experimental | Participants will receive EN001 intravenously (IV) once on Day 1. Before 30 minutes EN001 dosing, there will be premedication (solu-cortef 1-2 mg/kg + Lorazepam 0.1 mg/kg (max 2 mg) + Ondansetron (5 mg/m^2) + Chlorpheniramine (1 mg for 2~6 years old; 2 mg for 6~12 years old; 4 mg for over 12 years old)+ Acetaminophen) administered to assure safety of participants from issues such as immune rejection, due to the process of thawing in a frozen state of EN001. Receives: Drug: EN001 |
| Dose group B (High dose) | Experimental | Participants will receive EN001 intravenously (IV) once on Day 1. Before 30 minutes EN001 dosing, there will be premedication (solu-cortef 1-2 mg/kg + Lorazepam 0.1 mg/kg (max 2 mg) + Ondansetron (5 mg/m^2) + Chlorpheniramine (1 mg for 2~6 years old; 2 mg for 6~12 years old; 4 mg for over 12 years old)+ Acetaminophen) administered to assure safety of participants from issues such as immune rejection, due to the process of thawing in a frozen state of EN001. Receives: Drug: EN001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Measured: Week 12 after treatment
Determination of Dose-limiting toxicity (DLT) levels of EN001
Among the adverse events occurring for 2 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE 5.0
Measured: Up to Week 2 after dosing on Day 0
Determination of Maximum tolerated dose (MTD) levels of EN001
Among the adverse events occurring for 2 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE 5.0 Maximum tolerated dose defines the evaluated maximum dose level in which greater than two participants of six participants experience Dose-limiting toxicity (DLT) under the dose level. The dose level where two participants of six participants experience DLT will be the maximum tolerated dose.
Measured: Up to Week 2 after dosing on Day 0
Number of participants with Vital Signs abnormalities
Vital Signs include blood pressure (mmHg), pulse (times/minute), respiratory rate (times/minute), and body temperature (℃) and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001.
The number of participants with at least one potentially clinically significant abnormal vital sign finding were reported as treatment emergent adverse events (TEAEs).
Measured: Week 12 after screening
Number of participants with clinically significant abnormalities of Physical Examinations
Physical Examinations include general appearance, head, ears/eyes/nose/throat, cardiovascular, respiratory, abdomen, skin, lymph nodes, extremities, musculoskeletal and neurologic and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001.
Number of participants with potentially clinically significant abnormalities in physical examinations were reported as TEAEs.
Measured: From screening up to Week 12
Number of participants with abnormalities of Laboratory Parameters
Laboratory Parameters include hematology, chemistry laboratory tests, urinalysis, coagulation test and plasma viral load test and will be assessed by CTCAE 5.0 to evaluate safety and tolerability of EN001.
Number of participants with at least one potentially clinically significant abnormal finding were reported as TEAEs.
Measured: From screening up to Week 12
Number of participants with abnormalities of 12-lead Electrocardiography (ECG)
Categorical summarization ECG criteria were as follows:
- QT interval, QTcB, QTcF and QTcP: increase from baseline >30 millisecond [ms] or 60 ms; absolute value > 450 ms, >480 ms, and > 500 ms;
- heart rate (HR): change from baseline ≥20 beats per minute [bpm] and absolute value≤50 bpm or ≥120 bpm;
- PR interval: absolute value ≥220 ms and increase from baseline≥20 ms;
- QRS: ≥120 ms.
Measured: From screening to baseline on Day 0 (Predose to end of infusion and 90 min after completion of infusion)
Secondary outcomes (4)
Incidence of adverse events (AEs)
Occurrence of any adverse reactions, development of new blood clots, tumors, immune responses (like autoimmune reactions) and death, and/or serious adverse events related investigational product will be summarized by actual treatment groups respectively.
Measured: From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject)
Number of participants with abnormalities of Vital Signs, Physical Findings, and Laboratory Parameters
Abnormalities of Vital Signs, Physical Findings, and Laboratory parameters (as described above) will be collected and analyzed, and then assessed by CTCAE 5.0 to evaluate the long-term safety of EN001.
Measured: From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject)
Rate of change at the time of visit compared to baseline (percent [%]) in CK level
Creatinine kinase (CK) level will be collected and analyzed to evaluate the exploratory efficacy of EN001.
- CK level(%) = (CK level after dosing - CK level in baseline)/(CK level in baseline)*100
Measured: From screening up to the end of support (up to approximately 5 years per subject at each visit)
Change from baseline in Function tests
Function tests measured by North Star Ambulatory Assessment (NSAA), Six Minute Walk Test (6MWT), Myometry. and Lung capacity (and only K-Cross Motor Function Measure (KGMFM) will be performed under 5 years old) will be collected and analyzed to evaluate the exploratory efficacy of EN001.
- Function tests = value in visit - value in baseline
Measured: Screening and baseline on Day -1 (up to approximately 5 years per subject after Week 12)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2022
- Study start18 Jan 2022
- Primary completion28 Dec 2022
- Study completion28 Dec 2022
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- ENCell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05338099
- Sponsor's protocol ID
- ENCell_2020_01
- Status last verified
- Aug 2024
- First submitted
- 6 Jan 2022
- Last updated
- 30 Aug 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 30 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
- ClinicalTrials.gov ID
- NCT05338099
- Acronym / sponsor ID
- ENCell_2020_01
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- ENCellIndustry (pharma, biotech, devices)
- Study start
- 18 Jan 2022 (actual)
- Primary completion
- 28 Dec 2022 (actual)
- Study completion
- 28 Dec 2022 (actual)
- First posted
- 20 Apr 2022 (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.
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
Detailed description
It is the first in human (FIH), 3+3 design clinical trial to evaluate the safety and tolerability and determine the maximum tolerated dose (MTD) of EN001 (allogeneic umbilical cord-derived mesenchymal stem cells) in the treatment of Duchenne Muscular Dystrophy (DMD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose group A (Low dose) | Experimental | Drug: EN001Participants will receive EN001 intravenously (IV) once on Day 1. Before 30 minutes EN001 dosing, there will be premedication (solu-cortef 1-2 mg/kg + Lorazepam 0.1 mg/kg (max 2 mg) + Ondansetron (5 mg/m^2) + Chlorpheniramine (1 mg for 2~6 years old; 2 mg for 6~12 years old; 4 mg for over 12 years old)+ Acetaminophen) administered to assure safety of participants from issues such as immune rejection, due to the process of thawing in a frozen state of EN001. |
| Dose group B (High dose) | Experimental | Drug: EN001Participants will receive EN001 intravenously (IV) once on Day 1. Before 30 minutes EN001 dosing, there will be premedication (solu-cortef 1-2 mg/kg + Lorazepam 0.1 mg/kg (max 2 mg) + Ondansetron (5 mg/m^2) + Chlorpheniramine (1 mg for 2~6 years old; 2 mg for 6~12 years old; 4 mg for over 12 years old)+ Acetaminophen) administered to assure safety of participants from issues such as immune rejection, due to the process of thawing in a frozen state of EN001. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Those aged 2 to 18 years old
- Male
- Those who are diagnosed with DMD due to a mutation in the dystrophin gene identified by a genetic test
- Phenotypic evidence of DMD
- Those who have been using systemic corticosteroids at a stable dose for 24 weeks prior to screening and are expected to maintain the constant dose throughout the study period
Key exclusion criteria (18 listed)
- Those who have clinical signs or symptoms of cardiomyopathy, defined as LVEF <50% on echocardiography at screening
- If ventilatory support is required during the day or if invasive mechanical ventilation via tracheostomy is used (Non-invasive ventilation such as positive pressure ventilation is allowed at night)
- If hepatitis B core antibody and hepatitis C antibody are positive
- If there is a history of major surgery within 12 weeks or it is expected during the study period
- Those who have been exposed to gene therapy or genome editing within 24 weeks from the screening
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 (Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells)EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows. Dose group A (Low dose): 5.0x10^5 cells/kg | Drug | Dose group A (Low dose) |
| EN001Also: EN001 (Allogeneic early-passage Wharton's jelly-derived mesenchymal stem cells)EN001 intravenously (IV) in the treatment of Duchenne Muscular Dystrophy (DMD) Dosage for each group is as follows. Dose group B (High dose): 2.5x10^6 cells/kg | Drug | Dose group B (High dose) |
Primary outcome
Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product
Time frame: Week 12 after treatment
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Determination of Dose-limiting toxicity (DLT) levels of EN001
Time frame: Up to Week 2 after dosing on Day 0
Among the adverse events occurring for 2 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE 5.0
Determination of Maximum tolerated dose (MTD) levels of EN001
Time frame: Up to Week 2 after dosing on Day 0
Among the adverse events occurring for 2 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE 5.0 Maximum tolerated dose defines the evaluated maximum dose level in which greater than two participants of six participants experience Dose-limiting toxicity (DLT) under the dose level. The dose level where two participants of six participants experience DLT will be the maximum tolerated dose.
Number of participants with Vital Signs abnormalities
Time frame: Week 12 after screening
Vital Signs include blood pressure (mmHg), pulse (times/minute), respiratory rate (times/minute), and body temperature (℃) and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001. The number of participants with at least one potentially clinically significant abnormal vital sign finding were reported as treatment emergent adverse events (TEAEs).
Number of participants with clinically significant abnormalities of Physical Examinations
Time frame: From screening up to Week 12
Physical Examinations include general appearance, head, ears/eyes/nose/throat, cardiovascular, respiratory, abdomen, skin, lymph nodes, extremities, musculoskeletal and neurologic and will be assessed by CTCAE v 5.0 to evaluate safety and tolerability of EN001. Number of participants with potentially clinically significant abnormalities in physical examinations were reported as TEAEs.
Number of participants with abnormalities of Laboratory Parameters
Time frame: From screening up to Week 12
Laboratory Parameters include hematology, chemistry laboratory tests, urinalysis, coagulation test and plasma viral load test and will be assessed by CTCAE 5.0 to evaluate safety and tolerability of EN001. Number of participants with at least one potentially clinically significant abnormal finding were reported as TEAEs.
Number of participants with abnormalities of 12-lead Electrocardiography (ECG)
Time frame: From screening to baseline on Day 0 (Predose to end of infusion and 90 min after completion of infusion)
Categorical summarization ECG criteria were as follows: 1. QT interval, QTcB, QTcF and QTcP: increase from baseline >30 millisecond [ms] or 60 ms; absolute value > 450 ms, >480 ms, and > 500 ms; 2. heart rate (HR): change from baseline ≥20 beats per minute [bpm] and absolute value≤50 bpm or ≥120 bpm; 3. PR interval: absolute value ≥220 ms and increase from baseline≥20 ms; 4. QRS: ≥120 ms.
4 secondary outcomes
Incidence of adverse events (AEs)
Time frame: From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject)
Number of participants with abnormalities of Vital Signs, Physical Findings, and Laboratory Parameters
Time frame: From screening to the end of treatment/withdrawal visit (up to approximately 5 years per subject)
Rate of change at the time of visit compared to baseline (percent [%]) in CK level
Time frame: From screening up to the end of support (up to approximately 5 years per subject at each visit)
Change from baseline in Function tests
Time frame: Screening and baseline on Day -1 (up to approximately 5 years per subject after Week 12)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EN001 in people with Duchenne Muscular Dystrophy, 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 9 inclusion and 18 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: finished, as of the sponsor's last update on 30 Aug 2024. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants of any Adverse Events (AEs)/Serious Adverse Events (SAEs) related investigational product (time frame: Week 12 after treatment). The study lists 7 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 30 Aug 2024.