Charcot-Marie-Tooth Disease, Type IAPhase 1NCT05333406
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
A Phase 1 study testing EN001 in people with Charcot-Marie-Tooth Disease, Type IA, 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 Charcot-Marie-Tooth disease (CMT) type 1A Read more about this study
- Who is it for?
- Ages 19 to 75 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease, Type IA
- 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
- 9 participants
- Where
- 1 siteSouth Korea
- Study dates
- 22 Feb 2022to 20 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 19 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females aged 19 to 75 years old
- Those diagnosed with CMT type 1A by a genetic test
- Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
- Those who have dorsiflexion muscle weakness
- Those who can comply with the requirements for clinical trials
- For women of childbearing potential, those who have a negative urine pregnancy test at screening
- Those who use a medically acceptable method of contraception until clinical trial visit 7 (short-term follow-up visit, 16 weeks): hormonal contraception, intrauterine device (IUD), intrauterine system (IUS), vasectomy, tubal ligation, or double barrier method using a cervical cap or a diaphragm with a male condom.
- Those who voluntarily agree to participate in this study and sign an IRB-approved consent form after being informed about the characteristics of this clinical trial prior to all screening tests
Who cannot join
- Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
- Patients diagnosed with type 1 or type 2 diabetes
- Those with a history of stroke or cerebral ischemic attack within 12 months of screening
- Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
- Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of screening
- Those who have ankle contractures or have surgery that may affect muscle strength assessment
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- Those who have experience with stem cell therapy or gene therapy before screening
- Those who have participated in clinical trials for chemical synthetic drugs before screening (except when 5 times the half-life has passed)
- Patients with uncontrolled hypertension (If the systolic blood pressure is 180 mmHg or higher or the diastolic blood pressure is 110 mmHg or higher)
- If there is a history of malignant tumors other than basal cell carcinoma or squamous cell carcinoma occurring in the skin within 5 years of screening
- Those who diagnosed with active pulmonary tuberculosis
- Immunosuppressed patients who are taking immunosuppressants, chemotherapy, radiation therapy, etc.
- Mental illness patients
- Those who are pregnant or lactating
- 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 Charcot-Marie-Tooth Disease (CMT) Type 1A
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A
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 Charcot-Marie-Tooth disease (CMT) type 1A.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA
- Being tested
- EN001
- Medical subject headings
- Charcot-Marie-Tooth Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EN001Drug
Also called EN001 (allogeneic umbilical cord-derived mesenchymal stem cells)
EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A Dosage for each group is as follows.
Dose group A (Low dose): 5.0x10^5 cells/kg
EN001Drug
Also called EN001 (allogeneic umbilical cord-derived mesenchymal stem cells)
EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A 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 0. Receives: Drug: EN001 |
| Dose group B (High dose) | Experimental | Participants will receive EN001 intravenously (IV) once on Day 0. 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 AEs/SAEs as assessed by CTCAE v5.0
Measured: Week 16 after treatment
Determination of Dose-limiting toxicity (DLT) levels of EN001
Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0
Measured: Up to Week 4 after dosing on Day 0
Determination of Maximum tolerated dose (MTD) levels of EN001
Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0 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 4 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: From screening up to Week 16
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 16
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 16
Number of participants with 12-lead Electrocardiography (ECG) abnormalities
Measured by result of the ECG measurements and findings
Measured: From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion)
Secondary outcomes (6)
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)
Change from baseline in disease severity CMTNS-v2 score
Disease severity measured by CMTNS-v2 score will be collected and analyzed to evaluate the exploratory efficacy of EN001.
Measured: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in gait and balance functions
Gait and balance functions measured by Ten Meter Walking Test(10MWT), Functional Disability Scale (FDS), and Overall Neuropathy Limitation Score (ONLS) leg scale will be collected and analyzed to evaluate the exploratory efficacy of EN001.
Measured: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in the degree of muscle damage (%)
The degree of muscle damage measured by lower extremity magnetic resonance imaging (MRI) scan will be collected and analyzed to evaluate the exploratory efficacy of EN001.
Measured: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in nerve regeneration potential
Nerve generation potential measured by MNCV, SNCV, CMAP, and SNAP will be collected and analyzed to evaluate the exploratory efficacy of EN001.
Measured: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2022
- Study start22 Feb 2022
- Primary completion20 Dec 2022
- Study completion20 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth Disease, Type IAStudies 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- ENCell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05333406
- Sponsor's protocol ID
- ENCell_2020_02
- Status last verified
- Feb 2023
- First submitted
- 6 Jan 2022
- Last updated
- 17 Feb 2023
- 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 17 Feb 2023. 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 Charcot-Marie-Tooth Disease (CMT) Type 1A
- ClinicalTrials.gov ID
- NCT05333406
- Acronym / sponsor ID
- ENCell_2020_02
- Conditions
- Charcot-Marie-Tooth Disease, Type IA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- ENCellIndustry (pharma, biotech, devices)
- Study start
- 22 Feb 2022 (actual)
- Primary completion
- 20 Dec 2022 (actual)
- Study completion
- 20 Dec 2022 (actual)
- First posted
- 19 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 Charcot-Marie-Tooth disease (CMT) type 1A
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 Charcot-Marie-Tooth disease (CMT) type 1A.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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 0. |
| Dose group B (High dose) | Experimental | Drug: EN001Participants will receive EN001 intravenously (IV) once on Day 0. |
Who may qualify
- Ages
- 19 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Males and females aged 19 to 75 years old
- Those diagnosed with CMT type 1A by a genetic test
- Those whose CMTNS-v2 score is more than 2 and 20 or fewer points, and the severity of the disease is mild to moderate
- Those who have dorsiflexion muscle weakness
- Those who can comply with the requirements for clinical trials
Key exclusion criteria (17 listed)
- Those with other neuromuscular diseases that the investigator judges cannot participate in the clinical trial
- Patients diagnosed with type 1 or type 2 diabetes
- Those with a history of stroke or cerebral ischemic attack within 12 months of screening
- Those with a history of coronary artery diseases such as myocardial infarction or unstable angina within 12 months of screening
- Those who have undergone orthopedic surgery on the lower extremities (bone and ligament correction, artificial joint insertion, osteotomy, arthroscopic surgery) within 6 months of 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 umbilical cord-derived mesenchymal stem cells)EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A 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 umbilical cord-derived mesenchymal stem cells)EN001 intravenously (IV) in the treatment of Charcot-Marie-Tooth disease (CMT) type 1A 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 16 after treatment
Number of participants with treatment-related AEs/SAEs as assessed by CTCAE v5.0
Determination of Dose-limiting toxicity (DLT) levels of EN001
Time frame: Up to Week 4 after dosing on Day 0
Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0
Determination of Maximum tolerated dose (MTD) levels of EN001
Time frame: Up to Week 4 after dosing on Day 0
Among the adverse events occurring for 4 weeks after administration of the investigational product, Grade 3 or higher adverse events according to CTCAE v5.0 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: From screening up to Week 16
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 16
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 16
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 12-lead Electrocardiography (ECG) abnormalities
Time frame: From screening to baseline on Day 1 (Predose to end of infusion and 90 min after completion of infusion)
Measured by result of the ECG measurements and findings
6 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)
Change from baseline in disease severity CMTNS-v2 score
Time frame: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in gait and balance functions
Time frame: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in the degree of muscle damage (%)
Time frame: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
Change from baseline in nerve regeneration potential
Time frame: Screening and baseline on Day 0 (up to approximately 5 years per subject after Week 4)
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 17 Feb 2023.
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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 Charcot-Marie-Tooth Disease, Type IA, 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 8 inclusion and 17 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 17 Feb 2023. 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 16 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 17 Feb 2023.