The Individual Therapy for Patients With Wilson's Disease

Wilson's Disease Clinical Trial

Official title:

The Individual Therapy for Patients With Wilson's Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03957720
• Other study ID #: WuZYLab-WD
• Status: Recruiting
• Phase: Early Phase 1
• Start date: March 15, 2019
• Est. completion date: December 31, 2024

Study information

• Verified date: May 2019
• Source: Second Affiliated Hospital, School of Medicine, Zhejiang University
• Contact: Zhi-Ying Wu, MD&PhD
• Phone: +86-571-87783569
• Email: zhiyingwu[@]zju.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Based on the genotype characteristics and genotype-phenotype-treatment prognosis data of Chinese WD patients, this study intends to further optimize the treatment regimen of Chinese WD patients and formulate individualized treatment regimens for each genotype, so as to further improve the prognosis of patients.

Description:

The purpose of the study is to evaluate treatment regimen efficacy based on different mutations, and Zinc treatment in presymptomatic WD patients.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: December 31, 2024
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Genetic diagnosis of Wilson's disease

• Presymptomatic patients with Wilson's disease

Exclusion Criteria:

• Movement disorder due to other definite causes instead of Wilson's disease

• Severe Lung, kidney or liver disease

• Neoplastic Disease

Related Conditions & MeSH terms

• Wilson's Disease

Intervention

Drug:
• DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
• Penicillamine
Dosage Form: Penicillamine: 250-1500mg per day, Frequency:TID,Duration: 5 years;
• DMSA
Dosage Form: DMSA: 750-1000mg per day,DMSA Frequency:BID,DMSA Duration: 5 years; Dosage Form: DMSA: 35mg/kg per day,DMSA Frequency:BID,DMSA Duration: 5 years;
• Zinc gluconate
Dosage Form: Zinc gluconate: 140mg per time,Zinc Frequency:BID,Zinc Duration: 5 years; Dosage Form: Zinc gluconate: 140mg per time,Zinc Frequency:TID,Zinc Duration: 5 years; Dosage Form: Zinc gluconate: 210mg per time,Zinc Frequency:TID,Zinc Duration: 5 years;

Locations

Country	Name	City	State
China	econd Affiliated Hospital,Zhejiang University School of Medicine	Hangzhou	Zhejiang

Sponsors (1)

Lead Sponsor	Collaborator
Second Affiliated Hospital, School of Medicine, Zhejiang University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Serum ceruloplasmin	Serum ceruloplasmin concentration will be analysed using a validated assay	Five years
Primary	24-hour urine copper	24-hour urinary copper excretion is to be measured	Five years
Primary	serum copper	serum copper will be analysed using a validated assay	Five years
Primary	White blood cell	The white blood cell will be evaluated using a validated assay	Five years
Primary	Platelet count	The platelet count will be evaluated using a validated assay	Five years
Primary	urine protein level	The urine protein level will be collected using a validated assay	Five years
Primary	Alanine transaminase	The concentration of alanine transaminase will be collected using a validated assay	Five years
Primary	blood creatinine	The concentration of blood creatinine will be tested using a validated assay	Five years
Primary	international normalized ratio	The international normalized ratio will be analyzed	Five years
Primary	hepatic fibrosis markers test	The concentrations of procollagen III, collage IV and hyaluronidase will be evaluated using a validated assay	Five years
Primary	bone mineral density test	The bone mineral density will be tested using the dual energy X ray absorptiometry	Five years
Primary	Abdominal ultrasound	The abdominal ultrasound will be collected	Five years
Primary	Urinary ultrasound	The urinary ultrasound will be analyzed in patients	Five years
Primary	Cranial MRI scan	The cranial MRI scan will be analyzed in patients	Five years
Primary	Unified Wilson's disease rating scale	The Unified Wilson's disease rating scale consist of three subscales, including neurological part (0~112), liver functional part (0~36) and mental state part (0~76). Three subscale scores are summed to compute a total score. The higher values represent a worse outcome	Five years