Charcot-Marie-Tooth Disease (CMT)Phase 2SORD-CMTNCT05777226
Research of SORD-CMT Natural History and Epalrestat Treatment
A Phase 2 study testing Epalrestat in people with Charcot-Marie-Tooth Disease (CMT), run by The Third Xiangya Hospital of Central South University.
Trial summary
Charcot-Marie-Tooth disease (CMT) is a group of the most common hereditary peripheral neuropathy with high clinical and genetic heterogeneity. Biallelic pathogenic variants in SORD gene leading to loss of function of SORD protein cause axonal degeneration. Read more about this study
- Who is it for?
- Ages 14 to 50 years
- Conditions the study is for
- Charcot-Marie-Tooth Disease (CMT)
- What is being tested?
- Epalrestat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Apr 2023to Apr 2026
- Phase
- Phase 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 14 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Genetically confirmed SORD-CMT2 patients.
- Male or non-pregnant, non-lactating female patients, aged > 14 and ≤ 50 years old.
- Rule out secondary peripheral neuropathy (e.g., diabetic peripheral neuropathy)
- Voluntarily participate in clinical trials, sign informed consent, and be able to understand and abide by research procedures.
- P.S. Patients who have a strong willingness to treat and voluntarily purchase epalrestat for treatment (50mg, three times a day) are included in the treatment group.
Who cannot join
- Presence of any other cause of peripheral neuropathy.
- Subjects with other neurological disorders affecting the evaluation of study treatment.
- History of clinically significant unstable medical disease that may cause harm to subjects participating in this study within the past 1 year.
- Patients with major trauma or extremity surgery before enrollment or planned for extremity surgery before completion of follow-up.
- Patients with previous bilateral ankle stabilization surgery.
- Patients who were treated with other related drugs in the 3 months before enrollment.
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- Have any history of drug abuse; have a history of alcohol dependence in the past 3 months.
- Subjects who are positive for syphilis antibody and HIV antibody.
- Subjects whose chest X-ray and B-ultrasound show tumors.
- Patients with poor compliance and unable to complete the study follow-up.
- Patients who do not agree to participate in the study.
- Other factors are not suitable for inclusion.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Multi-center Study of Natural History of SORD-related Charcot-Marie-Tooth Disease and Epalrestat Treatment
Charcot-Marie-Tooth disease (CMT) is a group of the most common hereditary peripheral neuropathy with high clinical and genetic heterogeneity. Biallelic pathogenic variants in SORD gene leading to loss of function of SORD protein cause axonal degeneration. Current research suggests that SORD-CMT2 may be the most common subtype of AR-CMT2. The primary purpose of this study is to explore the natural history of SORD-CMT2 patients by detecting the ONLS scale score and serum sorbitol level changes at 6th, 12th, 24th, and 36th months and to evaluate the effectiveness and safety of epalrestat. Patients with strong treatment willingness and voluntary purchase of drugs are included in the epalrestat treatment group, and patients without drug treatment willingness are included in the control group. Patients in the drug treatment group take epalrestat (50 mg) orally three times daily. This study is expected to be carried out simultaneously in 5 hospitals in mainland China. About 30 SORD-CMT2 patients will be enrolled in this study, and the study period will be 36 months.
- Conditions
- Charcot-Marie-Tooth Disease (CMT)
- Being tested
- Epalrestat
- 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.
EpalrestatDrug
Also called Epalrestat Tablets（YANGTZE River Pharmaceutical Group Nanjing Pharmaceutical. Co., Ltd.）
Patients in the treatment group are intervened with Epalrestat tablets.
| Study group | Role | What they receive |
|---|---|---|
| Epalrestat treatment group | Experimental | Epalrestat;Tablet; 50mg; three times a day; 36 months Receives: Drug: Epalrestat |
| control group | No intervention | No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum sorbitol levels
Changes of serum sorbitol levels at 6, 12, 24 and 36 months
Measured: 36 months
Overall Neuropathy Limitations Scale (ONLS) scores
Changes in ONLS scale scores at 6, 12, 24, and 36 months, The total score for the arm is 5 points and 7 for the leg; the total score ranges from 0 points (no disability) to 12 points (disability)
Measured: 36 months
Secondary outcomes (1)
The 10-meter walk test (10MWRT)
Changes in the 10-meter walk test (10MWRT) at 6, 12, 24, and 36 months
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2023
- Study startApr 2023 (estimated)
- Primary completionApr 2026 (estimated)
- Study completionApr 2026 (estimated)
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 (CMT)Studies of the same condition, open and under way.
- More trials run by The Third Xiangya Hospital of Central South UniversityThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ruxu Zhang, The Third Xiangya Hospital of Central South University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05777226
- Sponsor's protocol ID
- quick22474
- Acronym
- SORD-CMT
- Status last verified
- Apr 2023
- First submitted
- 13 Feb 2023
- Last updated
- 18 Apr 2023
- 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 18 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center Study of Natural History of SORD-related Charcot-Marie-Tooth Disease and Epalrestat Treatment
- ClinicalTrials.gov ID
- NCT05777226
- Acronym / sponsor ID
- SORD-CMT / quick22474
- Conditions
- Charcot-Marie-Tooth Disease (CMT)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- The Third Xiangya Hospital of Central South UniversityUniversity, hospital or other
- Study start
- Apr 2023 (estimated)
- Primary completion
- Apr 2026 (estimated)
- Study completion
- Apr 2026 (estimated)
- First posted
- 21 Mar 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.
Charcot-Marie-Tooth disease (CMT) is a group of the most common hereditary peripheral neuropathy with high clinical and genetic heterogeneity. Biallelic pathogenic variants in SORD gene leading to loss of function of SORD protein cause axonal degeneration. Current research suggests that SORD-CMT2 may be the most common subtype of AR-CMT2. The primary purpose of this study is to explore the natural history of SORD-CMT2 patients by detecting the ONLS scale score and serum sorbitol level changes at 6th, 12th, 24th, and 36th months and to evaluate the effectiveness and safety of epalrestat. Patients with strong treatment willingness and voluntary purchase of drugs are included in the epalrestat treatment group, and patients without drug treatment willingness are included in the control group. Patients in the drug treatment group take epalrestat (50 mg) orally three times daily. This study is expected to be carried out simultaneously in 5 hospitals in mainland China. About 30 SORD-CMT2 patients will be enrolled in this study, and the study period will be 36 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Epalrestat treatment group | Experimental | Drug: EpalrestatEpalrestat;Tablet; 50mg; three times a day; 36 months |
| control group | No intervention | –No Intervention |
Who may qualify
- Ages
- 14 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Genetically confirmed SORD-CMT2 patients.
- Male or non-pregnant, non-lactating female patients, aged > 14 and ≤ 50 years old.
- Rule out secondary peripheral neuropathy (e.g., diabetic peripheral neuropathy)
- Voluntarily participate in clinical trials, sign informed consent, and be able to understand and abide by research procedures.
- P.S. Patients who have a strong willingness to treat and voluntarily purchase epalrestat for treatment (50mg, three times a day) are included in the treatment group.
Key exclusion criteria (12 listed)
- Presence of any other cause of peripheral neuropathy.
- Subjects with other neurological disorders affecting the evaluation of study treatment.
- History of clinically significant unstable medical disease that may cause harm to subjects participating in this study within the past 1 year.
- Patients with major trauma or extremity surgery before enrollment or planned for extremity surgery before completion of follow-up.
- Patients with previous bilateral ankle stabilization surgery.
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 |
|---|---|---|
| EpalrestatAlso: Epalrestat Tablets（YANGTZE River Pharmaceutical Group Nanjing Pharmaceutical. Co., Ltd.）Patients in the treatment group are intervened with Epalrestat tablets. | Drug | Epalrestat treatment group |
Primary outcome
Serum sorbitol levels
Time frame: 36 months
Changes of serum sorbitol levels at 6, 12, 24 and 36 months
Overall Neuropathy Limitations Scale (ONLS) scores
Time frame: 36 months
Changes in ONLS scale scores at 6, 12, 24, and 36 months, The total score for the arm is 5 points and 7 for the leg; the total score ranges from 0 points (no disability) to 12 points (disability)
1 secondary outcome
The 10-meter walk test (10MWRT)
Time frame: 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2023.
- 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Epalrestat in people with Charcot-Marie-Tooth Disease (CMT), run by The Third Xiangya Hospital of Central South University.
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: status not confirmed, as of the sponsor's last update on 18 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
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 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 Zhang Ruxu, PhD (+8618975172668, zhangruxu@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serum sorbitol levels (time frame: 36 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Apr 2023.