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Research of SORD-CMT Natural History and Epalrestat Treatment (SORD-CMT)

Primary Purpose

Charcot-Marie-Tooth Disease (CMT)

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Epalrestat
Sponsored by
The Third Xiangya Hospital of Central South University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Charcot-Marie-Tooth Disease (CMT)

Eligibility Criteria

14 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    No Intervention

    Arm Label

    Epalrestat treatment group

    control group

    Arm Description

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

    No Intervention

    Outcomes

    Primary Outcome Measures

    Serum sorbitol levels
    Changes of serum sorbitol levels at 6, 12, 24 and 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)

    Secondary Outcome Measures

    The 10-meter walk test (10MWRT)
    Changes in the 10-meter walk test (10MWRT) at 6, 12, 24, and 36 months

    Full Information

    First Posted
    February 13, 2023
    Last Updated
    April 16, 2023
    Sponsor
    The Third Xiangya Hospital of Central South University
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05777226
    Brief Title
    Research of SORD-CMT Natural History and Epalrestat Treatment
    Acronym
    SORD-CMT
    Official Title
    Multi-center Study of Natural History of SORD-related Charcot-Marie-Tooth Disease and Epalrestat Treatment
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    April 2023 (Anticipated)
    Primary Completion Date
    April 2026 (Anticipated)
    Study Completion Date
    April 2026 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    The Third Xiangya Hospital of Central South University

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Charcot-Marie-Tooth Disease (CMT)

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    30 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Epalrestat treatment group
    Arm Type
    Experimental
    Arm Description
    Epalrestat;Tablet; 50mg; three times a day; 36 months
    Arm Title
    control group
    Arm Type
    No Intervention
    Arm Description
    No Intervention
    Intervention Type
    Drug
    Intervention Name(s)
    Epalrestat
    Other Intervention Name(s)
    Epalrestat Tablets(YANGTZE River Pharmaceutical Group Nanjing Pharmaceutical. Co., Ltd.)
    Intervention Description
    Patients in the treatment group are intervened with Epalrestat tablets.
    Primary Outcome Measure Information:
    Title
    Serum sorbitol levels
    Description
    Changes of serum sorbitol levels at 6, 12, 24 and 36 months
    Time Frame
    36 months
    Title
    Overall Neuropathy Limitations Scale (ONLS) scores
    Description
    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)
    Time Frame
    36 months
    Secondary Outcome Measure Information:
    Title
    The 10-meter walk test (10MWRT)
    Description
    Changes in the 10-meter walk test (10MWRT) at 6, 12, 24, and 36 months
    Time Frame
    36 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    14 Years
    Maximum Age & Unit of Time
    50 Years
    Accepts Healthy Volunteers
    No
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Zhang Ruxu, PhD
    Phone
    +8618975172668
    Email
    zhangruxu@vip.163.com

    12. IPD Sharing Statement

    Plan to Share IPD
    No

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