Charcot Marie Tooth Disease, Type 1Phase 1NCT05947578
The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease.
A Phase 1 study testing CLZ-2002 in people with Charcot Marie Tooth Disease, Type 1, run by Cellatoz Therapeutics, Inc at 1 site in South Korea.
Trial summary
A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Charcot Marie Tooth Disease, Type 1
- What is being tested?
- CLZ-2002
- 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
- 19 Jun 2023to 9 Aug 2024
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects aged 18 years or older
- Subjects with a proven diagnosis of Charcot-Marie-Tooth disease type 1 at the time of screening visit
- Subjects who have muscle weakness in at least foot dorsiflexion (clinical assessment) at the time of screening visit
- Subjects with a CMT neuropathy score (CMTNS-v2) of 2 or more and 30 or fewer points at the time of screening visit
- Subjects who can understand and are willing to sign a written informed consent document are willing to comply with all study procedures and schedule visits.
Who cannot join
- Subjects who have any other neuromuscular diseases
- Subjects who have undergone upper and lower limb bone surgery within six months before screening visit
- Subjects who have concerns about muscle strength measurements due to the previous surgery
- Subjects who have severe active infection including severe/purulent cellulitis at the injection sites at screening visit
- Subjects who have a history of hospitalization due to hypersensitivity to antihistamines or allergy or hypersensitivity to certain substances such as food or drugs
- Subjects who have a history of unstable cardiovascular disease defined by the presence of myocardial infarction (STEMI or NSTEMI) within 6 months before the screening or the presence of unstable angina pectoris (in the case of increased frequency of symptoms, increased severity, or signs of prolonged symptoms at moderate activity or rest)
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- Subjects who have a history of a transient ischemic attack (TIA) or stroke within 6 months before screening visit
- Subjects who have a positive HIV antibody test, hepatitis B antigen, or hepatitis C antibody test result
- Subjects who have a history of malignant tumors within 5 years before screening visit
- Subjects who have received systemic steroids (inhaled steroids are allowed), immunotherapy, or cytotoxic therapy within 14 days before screening, or who are expected to receive such treatment during the study period
- Subjects who have participated in other clinical trials within 30 days before screening visit
- Pregnant and lactating women or women of childbearing potential and men who plan a pregnancy or are unwilling to use adequate birth control methods\& until 30 days after the end of drug administration
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects With Charcot-Marie-Tooth Type 1(CMT 1)
A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1)
Read the detailed description
This study is the First In Human (FIH) clinical trial for evaluating the safety and tolerability of IM injections of CLZ-2002 in patients with Charcot-Marie-Tooth disease (CMT) Type 1.
CLZ-2002 is the allogeneic mesenchymal stem cell-derived Neuronal Regeneration Promoting Cells. These cells are Schwann cell-like cells differentiated from tonsillar mesenchymal stem cells. CLZ-2002 helps the remyelination of the damaged peripheral nerves by restoring the myelin sheaths. It also induces the nerve regeneration and myelination pathways in the sciatic nerve and restores abnormal muscle tissues in Charcot-Marie-Tooth disease type 1 (CMT1).
- Conditions
- Charcot Marie Tooth Disease, Type 1
- Being tested
- CLZ-2002
- Keywords
- Charcot-Marie-Tooth Disease, CMT1, Cell therapy, Tonsillar mesenchymal stem cells (T-MSC), Neuronal Regeneration Promoting Cells, Schwann cell-like cells
- 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.
CLZ-2002Drug
Also called Allogeneic tonsillar mesenchymal stem cell-derived schwann cell like cells
Low dose group (6×10^6 cells), Mid dose group (12×10^6 cells), High dose group (24×10^6 cells)
| Study group | Role | What they receive |
|---|---|---|
| CLZ-2002 | Experimental | CLZ-2002 injection is intramuscular in lower limbs on Day 1. Receives: Drug: CLZ-2002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of intramuscular (IM) injections of CLZ-2002 in Participants
Frequency and percentage of treatment-emergent adverse events (TEAEs) and serious adverse events after injection through laboratory tests, physical examinations, vital signs, and electrocardiogram measurements conducted during the clinical trial.
Measured: Day 1 (visit 2) to Week 24 (visit 6)
Secondary outcomes (7)
Changes from baseline in the disease severity of CMTNS-v2 score at Weeks 4, 12, and 24.
The range of CMTNS-v2 (Charcot Marie Tooth Neuropathy Score-v2) ranges from 0-36. The disease severity is classified as mild (≤10), moderate (11 to 20), and severe (>21).
Measured: Weeks 4, 12 and 24
Changes from baseline in neurological examination at Weeks 1, 4, 12 and 24
The principal investigator will assess the neurological examination which is investigated the evidence of muscle weakness in the arms, legs, hands, and feet; as well as signs of muscle wasting, reduced reflexes, and any sensory loss.
Measured: Weeks 1, 4, 12 and 24
Changes from baseline in the overall neuropathy limitation scale (ONLS) at Weeks 4, 12 and 24
The Overall Neuropathy Limiting Scale (ONLS) assesses scale scores in the lower extremities as the Total Neuropathy Limiting Scale. A 1-point stage represents a clinically meaningful change in terms of disability.
Measured: Weeks 4, 12 and 24
Changes from baseline in functional disability using the functional disability scale (FDS) at Weeks 4, 12, and 24
The Functional Disability Scale (FDS) primarily evaluates impairment in motor function and is assessed by the neurologist (principal investigator) during the visit.
The 9 stages of FDS are as follows:
0; normal, 1; normal but with cramps and fatigability, 2; inability to run, 3; walking difficulty but still possible unaided, 4; If able to walk with a cane (walk with cane), 5; walk with crutches, 6; walk with a walker, 7; wheelchair bound, 8; bedridden.
Measured: Weeks 4, 12, and 24
Changes from baseline in the 10-meter walk test at Weeks 4, 12, and 24
This test measures the time required for a subject to walk 10 m through the 10-Meter Walking Test (10MWT)
Measured: Weeks 4, 12, and 24
Change from baseline in the fatty infiltration level of lower limb muscles at Week 24.
The degree of fat infilteration seen on MRI was classified into five grades from 0-4 and compared as follow: No fat infiltration at all (Grade 0; no fat), some traces of fat infiltration (Grade1; some fatty streak), more muscle than fat (Grade2; fat <muscle), when muscle and fat are similar (Grade3; fat = muscle), and when fat is more than muscle (Grade 4; fat>muscle).
Measured: Week 24
Changes from baseline in the velocity of Motor and Sensory nerve conduction at Weeks 4, 12, and 24
Motor and sensory nerve conduction studies will be performed on the median nerve, ulnar nerve, peroneal nerve, tibial nerve, and sural nerve using skin surface stimulation electrodes and recording electrodes. using the skin surface stimulation and recording method.
The studies will be performed with the amplitudes of the compound muscle action potential (CMAP) and the sensory nerve action potential (SNAP) from the positive maximum value to the negative maximum value.
Measured: Weeks 4, 12, and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2023
- Study start19 Jun 2023
- Primary completion9 Aug 2024
- Study completion9 Aug 2024
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 1Studies 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
- Not applicable
- Intervention model
- Sequential
- Model description
- Dose group A (Low dose) Dose group B (Mid dose) Dose group C (High dose)
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellatoz Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05947578
- Sponsor's protocol ID
- CLZ-CMT-101
- Status last verified
- Jun 2023
- First submitted
- 16 Jun 2023
- Last updated
- 17 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects With Charcot-Marie-Tooth Type 1(CMT 1)
- ClinicalTrials.gov ID
- NCT05947578
- Acronym / sponsor ID
- CLZ-CMT-101
- Conditions
- Charcot Marie Tooth Disease, Type 1
- Keywords
- Charcot-Marie-Tooth Disease, CMT1, Cell therapy, Tonsillar mesenchymal stem cells (T-MSC), Neuronal Regeneration Promoting Cells, Schwann cell-like cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Cellatoz Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- 19 Jun 2023 (actual)
- Primary completion
- 9 Aug 2024 (actual)
- Study completion
- 9 Aug 2024 (actual)
- First posted
- 17 Jul 2023 (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 Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1)
Detailed description
This study is the First In Human (FIH) clinical trial for evaluating the safety and tolerability of IM injections of CLZ-2002 in patients with Charcot-Marie-Tooth disease (CMT) Type 1.
CLZ-2002 is the allogeneic mesenchymal stem cell-derived Neuronal Regeneration Promoting Cells. These cells are Schwann cell-like cells differentiated from tonsillar mesenchymal stem cells. CLZ-2002 helps the remyelination of the damaged peripheral nerves by restoring the myelin sheaths. It also induces the nerve regeneration and myelination pathways in the sciatic nerve and restores abnormal muscle tissues in Charcot-Marie-Tooth disease type 1 (CMT1).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Dose group A (Low dose) Dose group B (Mid dose) Dose group C (High dose)
| Arm | Type | Receives |
|---|---|---|
| CLZ-2002 | Experimental | Drug: CLZ-2002CLZ-2002 injection is intramuscular in lower limbs on Day 1. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Subjects aged 18 years or older
- Subjects with a proven diagnosis of Charcot-Marie-Tooth disease type 1 at the time of screening visit
- Subjects who have muscle weakness in at least foot dorsiflexion (clinical assessment) at the time of screening visit
- Subjects with a CMT neuropathy score (CMTNS-v2) of 2 or more and 30 or fewer points at the time of screening visit
- Subjects who can understand and are willing to sign a written informed consent document are willing to comply with all study procedures and schedule visits.
Key exclusion criteria (12 listed)
- Subjects who have any other neuromuscular diseases
- Subjects who have undergone upper and lower limb bone surgery within six months before screening visit
- Subjects who have concerns about muscle strength measurements due to the previous surgery
- Subjects who have severe active infection including severe/purulent cellulitis at the injection sites at screening visit
- Subjects who have a history of hospitalization due to hypersensitivity to antihistamines or allergy or hypersensitivity to certain substances such as food or drugs
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 |
|---|---|---|
| CLZ-2002Also: Allogeneic tonsillar mesenchymal stem cell-derived schwann cell like cellsLow dose group (6×10^6 cells), Mid dose group (12×10^6 cells), High dose group (24×10^6 cells) | Drug | CLZ-2002 |
Primary outcome
Safety and tolerability of intramuscular (IM) injections of CLZ-2002 in Participants
Time frame: Day 1 (visit 2) to Week 24 (visit 6)
Frequency and percentage of treatment-emergent adverse events (TEAEs) and serious adverse events after injection through laboratory tests, physical examinations, vital signs, and electrocardiogram measurements conducted during the clinical trial.
7 secondary outcomes
Changes from baseline in the disease severity of CMTNS-v2 score at Weeks 4, 12, and 24.
Time frame: Weeks 4, 12 and 24
Changes from baseline in neurological examination at Weeks 1, 4, 12 and 24
Time frame: Weeks 1, 4, 12 and 24
Changes from baseline in the overall neuropathy limitation scale (ONLS) at Weeks 4, 12 and 24
Time frame: Weeks 4, 12 and 24
Changes from baseline in functional disability using the functional disability scale (FDS) at Weeks 4, 12, and 24
Time frame: Weeks 4, 12, and 24
Changes from baseline in the 10-meter walk test at Weeks 4, 12, and 24
Time frame: Weeks 4, 12, and 24
Change from baseline in the fatty infiltration level of lower limb muscles at Week 24.
Time frame: Week 24
Changes from baseline in the velocity of Motor and Sensory nerve conduction at Weeks 4, 12, and 24
Time frame: Weeks 4, 12, and 24
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 2025.
- Individual participant dataSharing planned: No
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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 CLZ-2002 in people with Charcot Marie Tooth Disease, Type 1, run by Cellatoz Therapeutics, Inc 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 5 inclusion and 12 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 2025. 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?
Safety and tolerability of intramuscular (IM) injections of CLZ-2002 in Participants (time frame: Day 1 (visit 2) to Week 24 (visit 6)). 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 2025.