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Immunosuppression Withdrawal for Stable Pediatric Liver Transplant Recipients (iWITH)

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ClinicalTrials.gov Identifier: NCT01638559
Recruitment Status : Completed
First Posted : July 11, 2012
Results First Posted : June 8, 2017
Last Update Posted : August 14, 2018
Sponsor:
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Immune Tolerance Network (ITN)
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Liver Transplant Recipients
Liver Transplantation
Immunosuppression
Intervention Drug: Immunosuppression withdrawal
Enrollment 88
Recruitment Details After institutional review board approval and informed consent, 161 subjects were screened and 88 subjects were enrolled. Eleven sites are in the U.S. and 1 site is in Canada. Recruitment was active from August 2012 through April 2014.
Pre-assignment Details Participants underwent a screening biopsy to assess study eligibility prior to beginning withdrawal of immunosuppression medications.
Arm/Group Title Participants That Initiated Withdrawal
Hide Arm/Group Description Pediatric liver transplant recipients with stable liver function tests, no evidence of rejection in the past 2 years, and at least 4 years post-transplant underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
Period Title: Overall Study
Started 88
Completed 88 [1]
Not Completed 0
[1]
Completion through the time of the primary endpoint.
Arm/Group Title Participants That Initiated Withdrawal
Hide Arm/Group Description Pediatric liver transplant recipients with stable liver function tests, no evidence of rejection in the past 2 years, and at least 4 years post-transplant underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
Overall Number of Baseline Participants 88
Hide Baseline Analysis Population Description
Intent to Treat
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants
<=18 years
88
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 88 participants
10.4  (3.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants
Female
49
  55.7%
Male
39
  44.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants
Hispanic or Latino
12
  13.6%
Not Hispanic or Latino
74
  84.1%
Unknown or Not Reported
2
   2.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 88 participants
American Indian or Alaska Native
0
   0.0%
Asian
4
   4.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
4
   4.5%
White
76
  86.4%
More than one race
0
   0.0%
Unknown or Not Reported
4
   4.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 88 participants
Canada 6
United States 82
1.Primary Outcome
Title Number of Operationally Tolerant Participants
Hide Description The number of participants that are operationally tolerant, defined as those who successfully withdraw from immunosuppression and maintain normal allograft status as assessed by liver biopsy and liver tests 12 months after complete immunosuppression withdrawal.
Time Frame 12 Months after Complete immunosuppression withdrawal
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol
Arm/Group Title Participants That Initiated Withdrawal
Hide Arm/Group Description:
Pediatric liver transplant recipients with stable liver function tests, no evidence of rejection in the past 2 years, and at least 4 years post-transplant underwent gradual Immunosuppression withdrawal in no less than 36 weeks and no more than 52 weeks with frequent monitoring of liver tests. All participants were followed for 48 months ensuring a minimum of 36 months of follow-up after successful Immunosuppression withdrawal.
Overall Number of Participants Analyzed 88
Measure Type: Count of Participants
Unit of Measure: Participants
33
  37.5%
Time Frame From screening through 31 MAR 2016 (up to 3 years and 8 months)
Adverse Event Reporting Description Adverse events are presented through 31 MAR 2016, when the last patient completed their primary endpoint visit. Screen failures are included in the safety population.
 
Arm/Group Title All Screened Participants
Hide Arm/Group Description All participants that were screened for the study.
All-Cause Mortality
All Screened Participants
Affected / at Risk (%)
Total   0/161 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
All Screened Participants
Affected / at Risk (%) # Events
Total   19/161 (11.80%)    
Gastrointestinal disorders   
Abdominal pain  1  1/161 (0.62%)  2
Hepatobiliary disorders   
Bile duct stenosis  1  2/161 (1.24%)  2
Cholangitis  1  2/161 (1.24%)  2
Immune system disorders   
Transplant rejection  1  3/161 (1.86%)  4
Infections and infestations   
Cellulitis  1  1/161 (0.62%)  1
Gastroenteritis  1  2/161 (1.24%)  2
Gastroenteritis viral  1  2/161 (1.24%)  3
Pneumonia  1  1/161 (0.62%)  1
Post procedural cellulitis  1  1/161 (0.62%)  1
Varicella  1  1/161 (0.62%)  1
Viral infection  1  2/161 (1.24%)  2
Injury, poisoning and procedural complications   
Post procedural bile leak  1  1/161 (0.62%)  1
Procedural pain  1  2/161 (1.24%)  2
Nervous system disorders   
Grand mal convulsion  1  1/161 (0.62%)  1
Psychiatric disorders   
Oppositional defiant disorder  1  1/161 (0.62%)  1
Suicidal ideation  1  1/161 (0.62%)  1
Reproductive system and breast disorders   
Menorrhagia  1  1/161 (0.62%)  1
Respiratory, thoracic and mediastinal disorders   
Asthma  1  2/161 (1.24%)  2
Skin and subcutaneous tissue disorders   
Eczema  1  1/161 (0.62%)  1
1
Term from vocabulary, MedDRA 14.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
All Screened Participants
Affected / at Risk (%) # Events
Total   89/161 (55.28%)    
Gastrointestinal disorders   
Abdominal pain  1  14/161 (8.70%)  19
Diarrhoea  1  14/161 (8.70%)  19
General disorders   
Influenza like illness  1  16/161 (9.94%)  30
Pyrexia  1  13/161 (8.07%)  16
Immune system disorders   
Transplant rejection  1  33/161 (20.50%)  37
Infections and infestations   
Ear infection  1  12/161 (7.45%)  22
Gastroenteritis  1  11/161 (6.83%)  13
Gastroenteritis viral  1  20/161 (12.42%)  25
Influenza  1  12/161 (7.45%)  14
Nasopharyngitis  1  34/161 (21.12%)  102
Pharyngitis streptococcal  1  14/161 (8.70%)  17
Sinusitis  1  13/161 (8.07%)  24
Upper respiratory tract infection  1  17/161 (10.56%)  31
Viral infection  1  17/161 (10.56%)  36
Investigations   
Liver function test abnormal  1  22/161 (13.66%)  31
Nervous system disorders   
Headache  1  14/161 (8.70%)  27
Respiratory, thoracic and mediastinal disorders   
Cough  1  12/161 (7.45%)  17
1
Term from vocabulary, MedDRA 14.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Director, Clinical Research Operations Program
Organization: DAIT/NIAID
Phone: 301-594-7669
Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT01638559     History of Changes
Other Study ID Numbers: DAIT iWITH
5U01AI100807-04 ( U.S. NIH Grant/Contract )
RTB-001 ( Other Identifier: DAIT NIAID )
First Submitted: July 9, 2012
First Posted: July 11, 2012
Results First Submitted: May 17, 2017
Results First Posted: June 8, 2017
Last Update Posted: August 14, 2018