Research of SORD-CMT Natural History and Epalrestat Treatment

NCT ID: NCT05777226

Last Updated: 2023-04-18

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-04-30
• Study Completion Date: 2026-04-30

Brief 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. 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)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Epalrestat treatment group

Epalrestat;Tablet; 50mg; three times a day; 36 months

Group Type: EXPERIMENTAL

Epalrestat

Intervention Type: DRUG

Patients in the treatment group are intervened with Epalrestat tablets.

control group

No Intervention

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Epalrestat

Patients in the treatment group are intervened with Epalrestat tablets.

Intervention Type: DRUG

Other Intervention Names

Epalrestat Tablets（YANGTZE River Pharmaceutical Group Nanjing Pharmaceutical. Co., Ltd.）

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.
• 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.

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Third Xiangya Hospital of Central South University

OTHER

Sponsor Role: lead

Responsible Party

Ruxu Zhang

Director, Chief physician of Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Central Contacts

Zhang Ruxu, PhD

Role: CONTACT

zhangruxu@vip.163.com

+8618975172668

Other Identifiers

quick22474

Identifier Type: -

Identifier Source: org_study_id