Gandouling in the Treatment of Wilson's Disease

The First Affiliated Hospital of Anhui University of Traditional Chinese Medicine

To evaluate the efficacy and safety of gandouling tablet in the treatment of clinical symptoms of Wilson's diasease.

Study Overview

• Status: Recruiting
• Conditions: Wilson's Disease
• Intervention / Treatment: Drug: Gandouling

Detailed Description

To evaluate the efficacy and safety of gandouling tablet in the treatment of clinical symptoms of Wilson's diasease.Test various indicatorsbefore test, 4 weeks, 12 weeks and 24 weeks.

• Study Type: Interventional
• Enrollment (Estimated): 240
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Jun Li, doctor; Phone Number: 18788899919; Email: 18788899919@163.com

Study Locations

• China
  • Anhui
    • Hefei, Anhui, China, 230037
      • Recruiting
      • Jun Li
      • Contact: Jun Li, doctor; Phone Number: 18788899919; Email: 18788899919@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 11 years to 81 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Comply with the diagnosis of Wilson's disease "Guidelines for Diagnosis and Treatment of Wilson's disease 2021"
• The diagnosis of TCM syndromes complies with the syndrome of phlegm and blood stasis; Other copper-removing drugs
• Patients who have been treated with complexing agents for copper-removing treatment can enter the study after a 2-week washout period
• Age ≥15 years
• Informed consent of patients or legal representatives, And sign the informed consent form.

Exclusion Criteria:

• Severe cerebral WD patients: obvious torsion spasm, dysphagia, or bed rest and other severe neurological impairment will interfere with the safety of the subjects (UWDRS Part I neurological function score ≥156 points)
• Severe liver Type WD patients: Decompensated liver cirrhosis or liver cancer, manifested as portal hypertension, ascites, splenomegaly (WBC<3.0*109/L, PLT<50*1012/L), esophageal varices, gastrointestinal bleeding, Moderate to severe anemia or hepatic encephalopathy; severe liver fibrosis shown by imaging or any laboratory abnormality (UWDRS liver function score ≥ 17 points)
• Moderate to severe depression, recent suicidal thoughts or behavior, Severe psychiatric symptoms (UWDRS Part III Psychiatric Symptom Score ≥ 54 points)
• History of epileptic seizures within 6 months
• Complicated with serious diseases such as brain tumors, brain trauma, blood diseases, Cardiogenic diseases, HIV, etc.
• Nephritis, nephrotic syndrome, or kidney disease stage 3 or more
• Pregnant, planned pregnancy or breastfeeding women
• Cognitive dysfunction MMSE≤26 points
• Those who are currently participating in other clinical trials
• Cannot comply with the follow-up plan

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment group; Gandouling	Drug: Gandouling; The experimental group was given gandouling tablet + zinc gluconate simulant; The course of treatment was 24 weeks.; Other Names: zinc gluconate
Placebo Comparator: Control group; Zinc gluconate	Drug: Gandouling; The experimental group was given gandouling tablet + zinc gluconate simulant; The course of treatment was 24 weeks.; Other Names: zinc gluconate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Unified Wilson's Disease Rating Scale(UWDRS)	Unified Wilson's Disease Rating Scale(UWDRS),the minimum is 0, and the maximum value is 320. Higher scores mean a worse outcome.	Baseline, before the study
Unified Wilson's Disease Rating Scale(UWDRS)	Unified Wilson's Disease Rating Scale(UWDRS). Higher scores mean a worse outcome.	4 weeks
Unified Wilson's Disease Rating Scale(UWDRS)	Unified Wilson's Disease Rating Scale(UWDRS).Higher scores mean a worse outcome.	12 weeks
Unified Wilson's Disease Rating Scale(UWDRS)	Unified Wilson's Disease Rating Scale(UWDRS).Higher scores mean a worse outcome.	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
24-hour urinary copper	24-hour urinary copper.Higher scores mean a worse outcome.	Baseline, before the study
24-hour urinary copper	24-hour urinary copper.Higher scores mean a worse outcome.	4 weeks
24-hour urinary copper	24-hour urinary copper.Higher scores mean a worse outcome.	12 weeks
24-hour urinary copper	24-hour urinary copper.Higher scores mean a worse outcome.	24 weeks
non-ceruloplasmin bound copper (NCC)	non-ceruloplasmin bound copper (NCC).Higher scores mean a worse outcome.	Baseline, before the study
non-ceruloplasmin bound copper (NCC)	non-ceruloplasmin bound copper (NCC).Higher scores mean a worse outcome.	4 weeks
non-ceruloplasmin bound copper (NCC)	non-ceruloplasmin bound copper (NCC).Higher scores mean a worse outcome.	12 weeks
non-ceruloplasmin bound copper (NCC)	non-ceruloplasmin bound copper (NCC).Higher scores mean a worse outcome.	24 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Global Assessment Scale (GAS)	Global Assessment Scale (GAS).Higher scores mean a worse outcome.	Baseline, before the study
Global Assessment Scale (GAS)	Global Assessment Scale (GAS).Higher scores mean a worse outcome.	4 weeks
Global Assessment Scale (GAS)	Global Assessment Scale (GAS).Higher scores mean a worse outcome.	12 weeks
Global Assessment Scale (GAS)	Global Assessment Scale (GAS).Higher scores mean a worse outcome.	24 weeks
Short Form-36 Questionnaire (SF-36)	Short Form-36 Questionnaire (SF-36).Higher scores mean a worse outcome.	Baseline, before the study
Short Form-36 Questionnaire (SF-36)	Short Form-36 Questionnaire (SF-36).Higher scores mean a worse outcome.	4 weeks
Short Form-36 Questionnaire (SF-36)	Short Form-36 Questionnaire (SF-36).Higher scores mean a worse outcome.	12 weeks
Short Form-36 Questionnaire (SF-36)	Short Form-36 Questionnaire (SF-36).Higher scores mean a worse outcome.	24 weeks

Collaborators and Investigators

• Sponsor: Jun Li

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2023
• Primary Completion (Actual): March 1, 2023
• Study Completion (Estimated): December 31, 2024

Study Registration Dates

• First Submitted: November 24, 2021
• First Submitted That Met QC Criteria: March 23, 2022
• First Posted (Actual): March 31, 2022

Study Record Updates

• Last Update Posted (Actual): February 9, 2024
• Last Update Submitted That Met QC Criteria: February 8, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Liver Diseases; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Metabolism, Inborn Errors; Heredodegenerative Disorders, Nervous System; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Metal Metabolism, Inborn Errors; Hepatolenticular Degeneration
• Other Study ID Numbers: GWPS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No