A Phase 2 interventional study of Epalrestat in Charcot-Marie-Tooth Disease (CMT), sponsored by The Third Xiangya Hospital of Central South University. Not yet recruiting. Open to participants aged 14 Years to 50 Years. Per ClinicalTrials.gov, last updated 2023-04-18.
Sponsored by The Third Xiangya Hospital of Central South University · Phase 2, Interventional, and Treatment
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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.
113 studies on the registry are indexed under Charcot-Marie-Tooth Disease; 37 are open to participants now.
This study's planned enrollment of 30 is below the median of 41 across 62 interventional studies indexed under Charcot-Marie-Tooth Disease.
Browse Charcot-Marie-Tooth Disease studies →The Third Xiangya Hospital of Central South University is the lead sponsor of 99 studies on the registry; 52 are open to participants now.
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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:
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The human immunodeficiency virus, which weakens the immune system.
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Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Epalrestat;Tablet; 50mg; three times a day; 36 months
Drug: Epalrestat
No Intervention
Patients in the treatment group are intervened with Epalrestat tablets.
Also known as: Epalrestat Tablets（YANGTZE River Pharmaceutical Group Nanjing Pharmaceutical. Co., Ltd.）
Serum sorbitol levels
Changes of serum sorbitol levels at 6, 12, 24 and 36 months
Time frame: 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)
Time frame: 36 months
The 10-meter walk test (10MWRT)
Changes in the 10-meter walk test (10MWRT) at 6, 12, 24, and 36 months
Time frame: 36 months
No study locations are listed for this record.
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Apr 2023; primary completion for Apr 2026, 36 months later.
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This study is not yet recruiting, as verified in Apr 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Epalrestat
The Third Xiangya Hospital of Central South University