Brief Title
The Individual Therapy for Patients With Wilson's Disease
Official Title
The Individual Therapy for Patients With Wilson's Disease
Brief 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.
Detailed Description
The purpose of the study is to evaluate treatment regimen efficacy based on different mutations, and Zinc treatment in presymptomatic WD patients.
Study Phase
Early Phase 1
Study Type
Interventional
Primary Outcome
Serum ceruloplasmin
Condition
Wilson's Disease
Intervention
DMPS
Study Arms / Comparison Groups
homo-R778L
Description: When patients carrying homo-R778L mutation are in hospital, they receive DMPS treatment. Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days; When being off hospital, they receive DMSA treatment. Adult patient,Dosage Form: DMSA: 750-1000mg per day,DMSA Frequency:BID,DMSA Duration: 5 years; Pediatric patient,Dosage Form: DMSA: 35mg/kg per day,DMSA Frequency:BID,DMSA Duration: 5 years;
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
400
Start Date
March 15, 2019
Completion Date
December 31, 2024
Primary Completion Date
December 31, 2024
Eligibility Criteria
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
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Zhi-Ying Wu, MD&PhD, +86-571-87783569, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT03957720
Organization ID
WuZYLab-WD
Responsible Party
Sponsor
Study Sponsor
Second Affiliated Hospital, School of Medicine, Zhejiang University
Study Sponsor
Zhi-Ying Wu, MD&PhD, Principal Investigator, Zhejiang University
Verification Date
May 2019