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Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure

Primary Purpose

Wilson Disease, Liver Failure

Status
Unknown status
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
artificial liver support system
Sponsored by
Third Affiliated Hospital, Sun Yat-Sen University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Wilson Disease focused on measuring Wilson Disease, Liver Failure, plasma exchange, continuous hemodiafiltration

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Clinical diagnosis of Wilson Disease: serum ceruloplasmin < 0.2g/L, 24 hours urinary copper > 40μg, Kayser-Fleischer ring present;
  2. Do not receive drugs to promote the excretion of copper ever before;
  3. Serum total bilirubin > 10 times upper limit of normal, prothrombin time activity < 40% or prothrombin time international ratio > 1.5.

Exclusion Criteria:

  1. Other active liver diseases;
  2. Hepatocellular carcinoma or other malignancy;
  3. Pregnancy or lactation;
  4. Human immunodeficiency virus infection or congenital immune deficiency diseases;
  5. Severe diabetes, autoimmune diseases;
  6. Other important organ dysfunctions.

Sites / Locations

  • The Third Affiliated Hospital of Sun Yat-sen University

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

No Intervention

Arm Label

Artificial liver support system group

Control group

Arm Description

30 patients will receive treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine.

30 patients will receive treatment of internal medicine.

Outcomes

Primary Outcome Measures

survival rate
Whether patients will survive is observed in the follow-up.

Secondary Outcome Measures

liver function improvement
Symptoms (i.e. fatigue, appetite, nausea, vomiting, jaundice, consciousness) and laboratory tests (i.e. blood cells, alanine transaminase, total bilirubin, prothrombin time, creatinine, ceruloplasmin, serum copper) are observed in the follow-up.

Full Information

First Posted
July 5, 2018
Last Updated
July 17, 2018
Sponsor
Third Affiliated Hospital, Sun Yat-Sen University
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1. Study Identification

Unique Protocol Identification Number
NCT03589820
Brief Title
Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure
Official Title
Clinical Efficacy of Artificial Liver Support System Using Combination of Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease - Related Liver Failure
Study Type
Interventional

2. Study Status

Record Verification Date
July 2018
Overall Recruitment Status
Unknown status
Study Start Date
June 1, 2018 (Actual)
Primary Completion Date
June 28, 2020 (Anticipated)
Study Completion Date
July 31, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Third Affiliated Hospital, Sun Yat-Sen University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study is to investigate the clinical efficacy of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration in treatment of Wilson's Disease - related liver failure. 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine. 30 patients will receive treatment of internal medicine.
Detailed Description
Wilson's disease (WD) is an inherited disorder in which defective biliary excretion of copper leads to its accumulation, particularly in liver and brain. Presenting symptoms of liver disease can be highly variable, ranging from asymptomatic, with only biochemical abnormalities, to overt cirrhosis with all its complications. Acute liver failure (ALF) presentation is the most dramatic and may arise with catastrophic suddenness. It is considered rare, constituting only 3% of ALF cases in the pediatric ALF series in USA and associated with a high mortality reaching 95%. Patients with acute liver failure due to WD require liver transplantation, which is lifesaving. Kido J et al reported clinical outcomes of patients with liver failure were improved by artificial liver support system using combination of plasma exchange and continuous hemodiafiltration. So the investigators design this protocol: 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine, 30 patients will receive treatment of internal medicine.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson Disease, Liver Failure
Keywords
Wilson Disease, Liver Failure, plasma exchange, continuous hemodiafiltration

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Artificial liver support system group
Arm Type
Active Comparator
Arm Description
30 patients will receive treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine.
Arm Title
Control group
Arm Type
No Intervention
Arm Description
30 patients will receive treatment of internal medicine.
Intervention Type
Other
Intervention Name(s)
artificial liver support system
Intervention Description
Patients will receive the treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine. The volume of fresh frozen plasma used in plasma exchange is 2000 millilitre. The time for continuous hemodiafiltration is about 8 hours.
Primary Outcome Measure Information:
Title
survival rate
Description
Whether patients will survive is observed in the follow-up.
Time Frame
48 weeks
Secondary Outcome Measure Information:
Title
liver function improvement
Description
Symptoms (i.e. fatigue, appetite, nausea, vomiting, jaundice, consciousness) and laboratory tests (i.e. blood cells, alanine transaminase, total bilirubin, prothrombin time, creatinine, ceruloplasmin, serum copper) are observed in the follow-up.
Time Frame
48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Clinical diagnosis of Wilson Disease: serum ceruloplasmin < 0.2g/L, 24 hours urinary copper > 40μg, Kayser-Fleischer ring present; Do not receive drugs to promote the excretion of copper ever before; Serum total bilirubin > 10 times upper limit of normal, prothrombin time activity < 40% or prothrombin time international ratio > 1.5. Exclusion Criteria: Other active liver diseases; Hepatocellular carcinoma or other malignancy; Pregnancy or lactation; Human immunodeficiency virus infection or congenital immune deficiency diseases; Severe diabetes, autoimmune diseases; Other important organ dysfunctions.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Liang Peng, Doctor
Organizational Affiliation
Third Affiliated Hospital, Sun Yat-Sen University
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Third Affiliated Hospital of Sun Yat-sen University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510630
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure

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