Maraviroc as GVHD Prophylaxis in Transplant Recipients
Primary Purpose
Diagnoses That Require Stem Cell Transplant, Graft Versus Host Disease (GVHD)
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Maraviroc
Sponsored by
About this trial
This is an interventional prevention trial for Diagnoses That Require Stem Cell Transplant focused on measuring stem cell transplant, GVHD prevention
Eligibility Criteria
Inclusion Criteria:
- Ages 5 years and </= 40 years
- All diagnoses
- Peripheral blood stem cells, marrow or cord blood
- All conditioning regimens
- Patient must be planned to receive a calcineurin inhibitor (cyclosporine or tacrolimus) together with steroid, methotrexate or mycophenolate mofetil as GVHD prophylaxis.
Exclusion Criteria:
- Documented anaphylaxis to Maraviroc
- Ex vivo T-cell (type of white blood cell) depleted grafts
- Abnormal Alanine Aminotransferase (ALT) (>/=10X ULN) on day -3. (Assessed at study enrollment and confirmed again prior to the first dose of maraviroc.)
Sites / Locations
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Maraviroc
Arm Description
Maraviroc administration will start on day -3 and will end on day +30 after stell cell transplant, making the total number of days of drug administration 34 days. Maraviroc will be administered twice daily orally or via enteral tube. Dosing of Maraviroc will be based on body surface area (starting with 100mg twice a day for BSA of 0.2 and up to 300mg twice a day for BSA greater than 1.73).
Outcomes
Primary Outcome Measures
Feasibility of Maraviroc
The ability to administer twice daily oral maraviroc in children and adults undergoing stem cell transplant in addition to routine standard graft versus host disease prophylaxis.
GVHD Incidence
Incidence of GVHD by day+100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
pK target >100ng/ml at day zero at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Incidence of Visceral GVHD
determine the number of patients who develop visceral GVHD by day+100
Area Under The Concentration-Time Curve (AUC) of Maraviroc
pK target >100ng/ml at day 10 at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Secondary Outcome Measures
Overall Survival
Overall survival for patients who were enrolled and received maraviroc
Graft Failure
Failure to engraft and loss of graft.
Primary Disease Relapse
Toxicities
Incidence of toxicities due to drug
Infectious Complications
Infections complications which include asymptomatic viremias for EBV, Adenovirus, CMV, and/or viral disease, bacterial and fungal infections as documented by blood cultures.
Time to Neutrophil
Neutrophil engraftment is defined as the first of three consecutive measurements of ANC>500mcL over 3 or more days.
Time to Platelet Engraftment
Time to achieve platelets count of 20,000 without transfusions
Full Information
NCT ID
NCT02167451
First Posted
June 13, 2014
Last Updated
March 2, 2020
Sponsor
Children's Hospital Medical Center, Cincinnati
1. Study Identification
Unique Protocol Identification Number
NCT02167451
Brief Title
Maraviroc as GVHD Prophylaxis in Transplant Recipients
Official Title
Maraviroc as Graft Versus Host Disease Prophylaxis in Pediatric and Adult Stem Cell Transplant Recipients
Study Type
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Terminated
Why Stopped
slow recruitment and no further drug supply
Study Start Date
July 2014 (undefined)
Primary Completion Date
September 2018 (Actual)
Study Completion Date
September 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose is to determine if the addition of Maraviroc to a standard transplant regimen will reduce the incidence of graft versus host disease in children and young adults after a stem cell transplant.
Detailed Description
In the first stage, drug levels will be obtained to establish appropriate dosing. In the second stage of the study the investigators will study the effects of using Maraviroc in these patients.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diagnoses That Require Stem Cell Transplant, Graft Versus Host Disease (GVHD)
Keywords
stem cell transplant, GVHD prevention
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
31 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Maraviroc
Arm Type
Experimental
Arm Description
Maraviroc administration will start on day -3 and will end on day +30 after stell cell transplant, making the total number of days of drug administration 34 days. Maraviroc will be administered twice daily orally or via enteral tube. Dosing of Maraviroc will be based on body surface area (starting with 100mg twice a day for BSA of 0.2 and up to 300mg twice a day for BSA greater than 1.73).
Intervention Type
Drug
Intervention Name(s)
Maraviroc
Other Intervention Name(s)
Selzentry
Primary Outcome Measure Information:
Title
Feasibility of Maraviroc
Description
The ability to administer twice daily oral maraviroc in children and adults undergoing stem cell transplant in addition to routine standard graft versus host disease prophylaxis.
Time Frame
Up to day +100
Title
GVHD Incidence
Description
Incidence of GVHD by day+100
Time Frame
By day +100
Title
Area Under The Concentration-Time Curve (AUC) of Maraviroc
Description
pK target >100ng/ml at day zero at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Time Frame
Day 0
Title
Incidence of Visceral GVHD
Description
determine the number of patients who develop visceral GVHD by day+100
Time Frame
day+100
Title
Area Under The Concentration-Time Curve (AUC) of Maraviroc
Description
pK target >100ng/ml at day 10 at the following time points : before the dose and 1, 2, 4, 6, 8, and 12 hours after maraviroc administration
Time Frame
Day 10
Secondary Outcome Measure Information:
Title
Overall Survival
Description
Overall survival for patients who were enrolled and received maraviroc
Time Frame
By day +100
Title
Graft Failure
Description
Failure to engraft and loss of graft.
Time Frame
By day +100
Title
Primary Disease Relapse
Time Frame
By day +100
Title
Toxicities
Description
Incidence of toxicities due to drug
Time Frame
Up to day +100
Title
Infectious Complications
Description
Infections complications which include asymptomatic viremias for EBV, Adenovirus, CMV, and/or viral disease, bacterial and fungal infections as documented by blood cultures.
Time Frame
Up to day +100
Title
Time to Neutrophil
Description
Neutrophil engraftment is defined as the first of three consecutive measurements of ANC>500mcL over 3 or more days.
Time Frame
Up to day +100
Title
Time to Platelet Engraftment
Description
Time to achieve platelets count of 20,000 without transfusions
Time Frame
days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
40 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Ages 5 years and </= 40 years
All diagnoses
Peripheral blood stem cells, marrow or cord blood
All conditioning regimens
Patient must be planned to receive a calcineurin inhibitor (cyclosporine or tacrolimus) together with steroid, methotrexate or mycophenolate mofetil as GVHD prophylaxis.
Exclusion Criteria:
Documented anaphylaxis to Maraviroc
Ex vivo T-cell (type of white blood cell) depleted grafts
Abnormal Alanine Aminotransferase (ALT) (>/=10X ULN) on day -3. (Assessed at study enrollment and confirmed again prior to the first dose of maraviroc.)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pooja Khandelwal, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
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Maraviroc as GVHD Prophylaxis in Transplant Recipients
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