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The Individual Therapy for Patients With Wilson's Disease

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ClinicalTrials.gov Identifier: NCT03957720
Recruitment Status : Recruiting
First Posted : May 21, 2019
Last Update Posted : May 21, 2019
Sponsor:
Information provided by (Responsible Party):
Second Affiliated Hospital, School of Medicine, Zhejiang University

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.

Condition or disease Intervention/treatment Phase
Wilson's Disease Drug: DMPS Drug: Penicillamine Drug: DMSA Drug: Zinc gluconate Early Phase 1

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 400 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Official Title: The Individual Therapy for Patients With Wilson's Disease
Actual Study Start Date : March 15, 2019
Estimated Primary Completion Date : December 31, 2024
Estimated Study Completion Date : December 31, 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease

Arm Intervention/treatment
Experimental: homo-R778L
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;
Drug: DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Other Name: Sodium Dimercaptosulphonate

Drug: 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;
Other Name: Dimercaptosuccinic Acid

Experimental: R778L+truncation mutation

When patients carrying R778L and truncation 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;

Drug: DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Other Name: Sodium Dimercaptosulphonate

Drug: 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;
Other Name: Dimercaptosuccinic Acid

Experimental: Homo-P992L

When patients carrying Homo-P992L 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;

Drug: DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Other Name: Sodium Dimercaptosulphonate

Drug: 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;
Other Name: Dimercaptosuccinic Acid

Experimental: P992L+truncation mutation

When patients carrying P992L and truncation 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;

Drug: DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Other Name: Sodium Dimercaptosulphonate

Drug: 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;
Other Name: Dimercaptosuccinic Acid

Experimental: T935M+other point mutations
When patients carrying T935M and other point mutations are in hospital, they randomly receive DMPS or penicillamine treatment; Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days; Dosage Form: penicillamine: 250-1500mg per day, Frequency:TID,Duration: 5 years; When being off hospital, they receive DMSA treatment or penicillamine. 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;
Drug: DMPS
Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Other Name: Sodium Dimercaptosulphonate

Drug: Penicillamine
Dosage Form: Penicillamine: 250-1500mg per day, Frequency:TID,Duration: 5 years;

Drug: 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;
Other Name: Dimercaptosuccinic Acid

Experimental: Presymptomatic patients with Wilson's disease

According to different age group, they receive various dosage of Zinc Gluconate treatment.

Patient aged≤6 years,Dosage Form: Zinc Gluconate: 140mg once, Zinc Gluconate Frequency:BID, Zinc Gluconate Duration: 5 years; Patient aged from 6 to 14 years,Dosage Form: Zinc Gluconate: 140mg once, Zinc Gluconate Frequency:TID, Zinc Gluconate Duration: 5 years; Patient aged≥14 years,Dosage Form: Zinc Gluconate: 210mg once, Zinc Gluconate Frequency:TID, Zinc Gluconate Duration: 5 years;

Drug: 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;




Primary Outcome Measures :
  1. Serum ceruloplasmin [ Time Frame: Five years ]
    Serum ceruloplasmin concentration will be analysed using a validated assay

  2. 24-hour urine copper [ Time Frame: Five years ]
    24-hour urinary copper excretion is to be measured

  3. serum copper [ Time Frame: Five years ]
    serum copper will be analysed using a validated assay

  4. White blood cell [ Time Frame: Five years ]
    The white blood cell will be evaluated using a validated assay

  5. Platelet count [ Time Frame: Five years ]
    The platelet count will be evaluated using a validated assay

  6. urine protein level [ Time Frame: Five years ]
    The urine protein level will be collected using a validated assay

  7. Alanine transaminase [ Time Frame: Five years ]
    The concentration of alanine transaminase will be collected using a validated assay

  8. blood creatinine [ Time Frame: Five years ]
    The concentration of blood creatinine will be tested using a validated assay

  9. international normalized ratio [ Time Frame: Five years ]
    The international normalized ratio will be analyzed

  10. hepatic fibrosis markers test [ Time Frame: Five years ]
    The concentrations of procollagen III, collage IV and hyaluronidase will be evaluated using a validated assay

  11. bone mineral density test [ Time Frame: Five years ]
    The bone mineral density will be tested using the dual energy X ray absorptiometry

  12. Abdominal ultrasound [ Time Frame: Five years ]
    The abdominal ultrasound will be collected

  13. Urinary ultrasound [ Time Frame: Five years ]
    The urinary ultrasound will be analyzed in patients

  14. Cranial MRI scan [ Time Frame: Five years ]
    The cranial MRI scan will be analyzed in patients

  15. Unified Wilson's disease rating scale [ Time Frame: Five years ]
    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



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03957720


Contacts
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Contact: Zhi-Ying Wu, MD&PhD +86-571-87783569 zhiyingwu@zju.edu.cn
Contact: Yi Dong, MD&PhD +8618367129345 dongyi720@zju.edu.cn

Locations
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China, Zhejiang
econd Affiliated Hospital,Zhejiang University School of Medicine Recruiting
Hangzhou, Zhejiang, China, 310009
Contact: Zhi-Ying Wu, MD&PhD    +86-571-87783569    zhiyingwu@zju.edu.cn   
Sponsors and Collaborators
Second Affiliated Hospital, School of Medicine, Zhejiang University
Investigators
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Principal Investigator: Zhi-Ying Wu, MD&PhD Zhejiang University

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Responsible Party: Second Affiliated Hospital, School of Medicine, Zhejiang University
ClinicalTrials.gov Identifier: NCT03957720     History of Changes
Other Study ID Numbers: WuZYLab-WD
First Posted: May 21, 2019    Key Record Dates
Last Update Posted: May 21, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases
Zinc
Penicillamine
Succimer
Trace Elements
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Antidotes
Protective Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Antirheumatic Agents