An Early Phase 1 interventional study of DMPS and Penicillamine in Wilson's Disease, sponsored by Second Affiliated Hospital, School of Medicine, Zhejiang University. Status unknown at 1 site in China. Per ClinicalTrials.gov, last updated 2019-05-21.
Sponsored by Second Affiliated Hospital, School of Medicine, Zhejiang University · Early Phase 1, Interventional, and Treatment
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.
The purpose of the study is to evaluate treatment regimen efficacy based on different mutations, and Zinc treatment in presymptomatic WD patients.
78 studies on the registry are indexed under Hepatolenticular Degeneration; 30 are open to participants now.
This study's planned enrollment of 400 is above the median of 30 across 42 interventional studies indexed under Hepatolenticular Degeneration.
Browse Hepatolenticular Degeneration studies →Second Affiliated Hospital, School of Medicine, Zhejiang University is the lead sponsor of 1,058 studies on the registry; 511 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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 · Drug: DMSA
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 · Drug: DMSA
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 · Drug: DMSA
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 · Drug: DMSA
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 · Drug: Penicillamine · Drug: DMSA
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: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;
Also known as: Sodium Dimercaptosulphonate
Dosage Form: Penicillamine: 250-1500mg per day, Frequency:TID,Duration: 5 years;
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;
Also known as: Dimercaptosuccinic Acid
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;
Serum ceruloplasmin
Serum ceruloplasmin concentration will be analysed using a validated assay
Time frame: Five years
24-hour urine copper
24-hour urinary copper excretion is to be measured
Time frame: Five years
serum copper
serum copper will be analysed using a validated assay
Time frame: Five years
White blood cell
The white blood cell will be evaluated using a validated assay
Time frame: Five years
Platelet count
The platelet count will be evaluated using a validated assay
Time frame: Five years
urine protein level
The urine protein level will be collected using a validated assay
Time frame: Five years
Alanine transaminase
The concentration of alanine transaminase will be collected using a validated assay
Time frame: Five years
blood creatinine
The concentration of blood creatinine will be tested using a validated assay
Time frame: Five years
international normalized ratio
The international normalized ratio will be analyzed
Time frame: Five years
hepatic fibrosis markers test
The concentrations of procollagen III, collage IV and hyaluronidase will be evaluated using a validated assay
Time frame: Five years
bone mineral density test
The bone mineral density will be tested using the dual energy X ray absorptiometry
Time frame: Five years
Abdominal ultrasound
The abdominal ultrasound will be collected
Time frame: Five years
Urinary ultrasound
The urinary ultrasound will be analyzed in patients
Time frame: Five years
Cranial MRI scan
The cranial MRI scan will be analyzed in patients
Time frame: Five years
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
Time frame: Five years
Plan to share: No
No publications or documents are linked to this record.
This study is status unknown, as verified in May 2019. You cannot join it, but the record below documents what was studied.
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Hepatolenticular Degeneration→
Second Affiliated Hospital, School of Medicine, Zhejiang University