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Tacrolimus, Sirolimus and Ustekinumab vs. Tacrolimus and Sirolimus for the Prevention of Acute Graft-Versus-Host Disease (Ustekinumab)

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ClinicalTrials.gov Identifier: NCT01713400
Recruitment Status : Completed
First Posted : October 24, 2012
Results First Posted : September 10, 2015
Last Update Posted : March 4, 2020
Sponsor:
Collaborator:
Gateway for Cancer Research
Information provided by (Responsible Party):
H. Lee Moffitt Cancer Center and Research Institute

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Graft vs. Host Disease
Interventions Drug: Ustekinumab
Drug: Placebo
Drug: Tacrolimus (TAC)
Drug: Sirolimus
Enrollment 54
Recruitment Details 54 participants (potential donors and recipients) were enrolled at Moffitt Cancer Center from 3/15/2013 through 5/22/2014.
Pre-assignment Details 30 eligible recipients were randomly assigned 1:1 (Ustekinumab n=15, Placebo n=15) with stratification for donor type. Results Data pertains to recipients only.
Arm/Group Title Ustekinumab Placebo
Hide Arm/Group Description Ustekinumab, Tacrolimus and Sirolimus. Ustekinumab: 45 mg for adults who weight 100 kg or less; 90 mg for adults who weight greater than 100 kg. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program. Placebo, Tacrolimus, and Sirolimus. Placebo: Identical volume to that of ustekinumab. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
Period Title: Overall Study
Started 15 15
Completed 15 15
Not Completed 0 0
Arm/Group Title Ustekinumab Placebo Total
Hide Arm/Group Description Ustekinumab, Tacrolimus and Sirolimus. Ustekinumab: 45 mg for adults who weight 100 kg or less; 90 mg for adults who weight greater than 100 kg. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program. Placebo, Tacrolimus, and Sirolimus. Placebo: Identical volume to that of ustekinumab. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program. Total of all reporting groups
Overall Number of Baseline Participants 15 15 30
Hide Baseline Analysis Population Description
All participating recipients
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 30 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
13
  86.7%
11
  73.3%
24
  80.0%
>=65 years
2
  13.3%
4
  26.7%
6
  20.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 30 participants
Female
7
  46.7%
4
  26.7%
11
  36.7%
Male
8
  53.3%
11
  73.3%
19
  63.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 15 participants 15 participants 30 participants
15 15 30
1.Primary Outcome
Title T Regulatory Cell (Treg)/Total Cluster of Differentiation 4 (CD4)+ Ratio
Hide Description Median Blood Treg/Total CD4+ Ratio at day 30 following hematopoietic cell transplantation (HCT). Comparison between study arms: Ustekinumab vs. Placebo. From NCI Dictionary: "T reg" - A type of immune cell that blocks the actions of some other types of lymphocytes, to keep the immune system from becoming over-active. T regs are being studied in the treatment of cancer. A T reg is a type of white blood cell and a type of lymphocyte. Also called regulatory T cell, suppressor T cell, and T-regulatory cell.
Time Frame 30 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
All participating recipients
Arm/Group Title Ustekinumab Placebo
Hide Arm/Group Description:
Ustekinumab, Tacrolimus and Sirolimus. Ustekinumab: 45 mg for adults who weight 100 kg or less; 90 mg for adults who weight greater than 100 kg. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
Placebo, Tacrolimus, and Sirolimus. Placebo: Identical volume to that of ustekinumab. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
Overall Number of Participants Analyzed 15 15
Median (Full Range)
Unit of Measure: ratio
13
(2 to 22)
11
(3 to 20)
2.Secondary Outcome
Title Incidence of Acute Graft vs. Host Disease (AGVHD)
Hide Description Cumulative incidence of Grade II - IV AGVHD to be characterized weekly from day of transplant to day 100 using the 1995 updated grading scheme for Graft vs. Host Disease (GVHD) developed by Glucksberg, et al.
Time Frame 100 days post transplant
Hide Outcome Measure Data
Hide Analysis Population Description
All participating recipients
Arm/Group Title Ustekinumab Placebo
Hide Arm/Group Description:
Ustekinumab, Tacrolimus and Sirolimus. Ustekinumab: 45 mg for adults who weight 100 kg or less; 90 mg for adults who weight greater than 100 kg. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
Placebo, Tacrolimus, and Sirolimus. Placebo: Identical volume to that of ustekinumab. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
Overall Number of Participants Analyzed 15 15
Measure Type: Number
Unit of Measure: percentage of participants
33.3 40.0
Time Frame 1 year, 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ustekinumab Placebo
Hide Arm/Group Description Ustekinumab, Tacrolimus and Sirolimus. Ustekinumab: 45 mg for adults who weight 100 kg or less; 90 mg for adults who weight greater than 100 kg. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program. Placebo, Tacrolimus, and Sirolimus. Placebo: Identical volume to that of ustekinumab. Tacrolimus: Level determined according to Blood and Marrow Transplant (BMT) Program standard operating procedures. Sirolimus: The dose for both loading and ongoing administration to be dictated by the standard operating procedures of the BMT program.
All-Cause Mortality
Ustekinumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Ustekinumab Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/15 (26.67%)      4/15 (26.67%)    
Blood and lymphatic system disorders     
Blood and lymphatic system disorders - Other, TMA  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Febrile neutropenia  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Cardiac disorders     
Cardiac disorders - Other, Hypotension  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Gastrointestinal disorders     
Gastric hemorrhage  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Gastrointestinal disorders - Other, Acute cholecystitis  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Gastrointestinal disorders - Other, GVHD of gut  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Hepatobiliary disorders     
Hepatobiliary disorders - Other, VOD  1  2/15 (13.33%)  2 1/15 (6.67%)  2
Portal hypertension  1  2/15 (13.33%)  2 0/15 (0.00%)  0
Infections and infestations     
Bladder infection  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Infections and infestations - Other, HHV6 infection affecting CNS  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Skin infection  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Psychiatric disorders     
Confusion  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Hallucinations  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Renal and urinary disorders     
Acute kidney injury  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Renal and urinary disorders - Other, elevated creatinine  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Renal and urinary disorders - Other, renal failure-dialysis  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Respiratory, thoracic and mediastinal disorders     
Respiratory failure  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Skin and subcutaneous tissue disorders     
Skin and subcutaneous tissue disorders - Other, GVHD of skin  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Ustekinumab Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   8/15 (53.33%)      8/15 (53.33%)    
Blood and lymphatic system disorders     
Blood and lymphatic disorders - Other  1  1/15 (6.67%)  1 1/15 (6.67%)  1
Cardiac disorders     
Cardiac disorders - Other  1  1/15 (6.67%)  1 2/15 (13.33%)  7
Gastrointestinal disorders     
Mucositis oral  1  1/15 (6.67%)  5 2/15 (13.33%)  8
Diarrhea  1  1/15 (6.67%)  6 2/15 (13.33%)  2
Hepatobiliary disorders     
Hepatobiliary disorders - Other  1  2/15 (13.33%)  2 3/15 (20.00%)  4
Infections and infestations     
Rhinitis infective  1  0/15 (0.00%)  0 3/15 (20.00%)  3
Upper respiratory infection  1  1/15 (6.67%)  3 0/15 (0.00%)  0
Urinary tract infection  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Investigations     
Alanine aminotransferase increased  1  1/15 (6.67%)  6 1/15 (6.67%)  1
Alkaline phosphatase increased  1  1/15 (6.67%)  1 1/15 (6.67%)  6
Aspartate aminotransferase increased  1  1/15 (6.67%)  4 1/15 (6.67%)  5
Blood bilirubin increased  1  1/15 (6.67%)  3 0/15 (0.00%)  0
GGT increased  1  1/15 (6.67%)  2 0/15 (0.00%)  0
Nervous system disorders     
Headache  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Bronchopulmonary hemorrhage  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Laryngeal mucositis  1  0/15 (0.00%)  0 1/15 (6.67%)  1
Respiratory, thoracic and mediastinal disorders - Other  1  1/15 (6.67%)  1 0/15 (0.00%)  0
Vascular disorders     
Hypertension  1  2/15 (13.33%)  2 0/15 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Joseph Pidala
Organization: H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-2556
EMail: joseph.pidala@moffitt.org
Layout table for additonal information
Responsible Party: H. Lee Moffitt Cancer Center and Research Institute
ClinicalTrials.gov Identifier: NCT01713400    
Other Study ID Numbers: MCC-16743
First Submitted: October 22, 2012
First Posted: October 24, 2012
Results First Submitted: July 15, 2015
Results First Posted: September 10, 2015
Last Update Posted: March 4, 2020