The Individual Therapy for Patients With Wilson’s Disease

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Wilson disease
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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