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Wilson's DiseaseEarly Phase 1NCT03957720

The Individual Therapy for Patients With Wilson's Disease

A Early Phase 1 study testing DMPS, Penicillamine and DMSA in people with Wilson's Disease, run by Second Affiliated Hospital, Zhejiang University, School of Medicine at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Wilson's Disease
What is being tested?
DMPS, Penicillamine and DMSA
Could I receive a placebo?
No, everyone receives an active treatment
How many people
400 participants
Where
1 siteChina
Study dates
15 Mar 2019to 31 Dec 2024
Phase
Early Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Genetic diagnosis of Wilson's disease
  • Presymptomatic patients with Wilson's disease

Who cannot join

  • Movement disorder due to other definite causes instead of Wilson's disease
  • Severe Lung, kidney or liver disease
  • Neoplastic Disease

Trial locations and contacts

1 site in 1 country.

Zhi-Ying Wu, MD&PhD contact

+86-571-87783569

zhiyingwu@zju.edu.cn

Yi Dong, MD&PhD contact

+8618367129345

dongyi720@zju.edu.cn

China1 site, 1 recruiting

  • econd Affiliated Hospital,Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310009

    Zhi-Ying Wu, MD&PhD, +86-571-87783569, zhiyingwu@zju.edu.cn

    Recruiting

Other trials in China

About this study

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.

Read the detailed description

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

Being tested
DMPS, Penicillamine, DMSA, Zinc gluconate
Medical subject headings
Hepatolenticular Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DMPSDrug

    Also called Sodium Dimercaptosulphonate

    Dosage Form: DMPS: 500-1000mg per day,DMPS Frequency:BID,DMPS Duration: 6 days;

  • PenicillamineDrug

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

  • DMSADrug

    Also called Dimercaptosuccinic Acid

    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 gluconateDrug

    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;

Study groups in NCT03957720
Study groupRoleWhat they receive
homo-R778LExperimentalWhen 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;
Receives: Drug: DMPS; Drug: DMSA
R778L+truncation mutationExperimentalWhen 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;
Receives: Drug: DMPS; Drug: DMSA
Homo-P992LExperimentalWhen 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;
Receives: Drug: DMPS; Drug: DMSA
P992L+truncation mutationExperimentalWhen 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;
Receives: Drug: DMPS; Drug: DMSA
T935M+other point mutationsExperimentalWhen 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;
Receives: Drug: DMPS; Drug: Penicillamine; Drug: DMSA
Presymptomatic patients with Wilson's diseaseExperimentalAccording 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;
Receives: Drug: Zinc gluconate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Serum ceruloplasmin

    Serum ceruloplasmin concentration will be analysed using a validated assay

    Measured: Five years

  2. 24-hour urine copper

    24-hour urinary copper excretion is to be measured

    Measured: Five years

  3. serum copper

    serum copper will be analysed using a validated assay

    Measured: Five years

  4. White blood cell

    The white blood cell will be evaluated using a validated assay

    Measured: Five years

  5. Platelet count

    The platelet count will be evaluated using a validated assay

    Measured: Five years

  6. urine protein level

    The urine protein level will be collected using a validated assay

    Measured: Five years

  7. Alanine transaminase

    The concentration of alanine transaminase will be collected using a validated assay

    Measured: Five years

  8. blood creatinine

    The concentration of blood creatinine will be tested using a validated assay

    Measured: Five years

  9. international normalized ratio

    The international normalized ratio will be analyzed

    Measured: Five years

  10. hepatic fibrosis markers test

    The concentrations of procollagen III, collage IV and hyaluronidase will be evaluated using a validated assay

    Measured: Five years

  11. bone mineral density test

    The bone mineral density will be tested using the dual energy X ray absorptiometry

    Measured: Five years

  12. Abdominal ultrasound

    The abdominal ultrasound will be collected

    Measured: Five years

  13. Urinary ultrasound

    The urinary ultrasound will be analyzed in patients

    Measured: Five years

  14. Cranial MRI scan

    The cranial MRI scan will be analyzed in patients

    Measured: Five years

  15. 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

    Measured: Five years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 May 2019
  2. Study start15 Mar 2019
  3. Primary completion31 Dec 2024 (estimated)
  4. Study completion31 Dec 2024 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
Single blind
Who is masked
Participant
Enrolment
400 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03957720
Sponsor's protocol ID
WuZYLab-WD
Status last verified
May 2019
First submitted
8 Apr 2019
Last updated
21 May 2019
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing DMPS, Penicillamine and DMSA in people with Wilson's Disease, run by Second Affiliated Hospital, Zhejiang University, School of Medicine at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 21 May 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Zhi-Ying Wu, MD&PhD (+86-571-87783569, zhiyingwu@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Serum ceruloplasmin (time frame: Five years). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03957720.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2019.

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