Trial Outcomes & Findings for Safety and Efficacy of Alemtuzumab in Pediatric Intestinal Transplantation (NCT NCT01208337)

NCT ID: NCT01208337

Last Updated: 2023-03-08

Results Overview

Asses safety of Alemtuzumab in combination with Tacrolimus and steroids in twenty-three pediatric intestine allograft recipients by calculating the rate in which PTLD occurred amongst the study population.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

5 Year

Results posted on

2023-03-08

Participant Flow

Participant milestones

Participant milestones
Measure
Alemtuzumab Induction
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Overall Study
STARTED
23
Overall Study
One-year Follow-up
23
Overall Study
Five-year Follow-up
20
Overall Study
COMPLETED
20
Overall Study
NOT COMPLETED
3

Reasons for withdrawal

Reasons for withdrawal
Measure
Alemtuzumab Induction
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Overall Study
Death
3

Baseline Characteristics

Safety and Efficacy of Alemtuzumab in Pediatric Intestinal Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Alemtuzumab Induction
n=23 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Age, Continuous
5.7 years
n=99 Participants
Sex: Female, Male
Female
6 Participants
n=99 Participants
Sex: Female, Male
Male
17 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
21 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
5 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
5 Participants
n=99 Participants
Race (NIH/OMB)
White
13 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Primary Isolated Intestine Transplant
21 participants
n=99 Participants

PRIMARY outcome

Timeframe: 5 Year

Population: Alemtuzumab induction at the time of transplant may reduce the rate of early acute cellular rejection compared with historical controls, but may increase rate of alternate post-transplant complications, such as Post Transplant Lymphoproliferative Disorder (PTLD).

Asses safety of Alemtuzumab in combination with Tacrolimus and steroids in twenty-three pediatric intestine allograft recipients by calculating the rate in which PTLD occurred amongst the study population.

Outcome measures

Outcome measures
Measure
Alemtuzumab Induction
n=23 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Incidence of Post Transplant Lymphoproliferative Disorder (PTLD)
6 participants

SECONDARY outcome

Timeframe: 1 Year

Population: Alemtuzumab induction at the time of transplant may reduce the rate of early acute cellular rejection compared with historical controls, but may increase rate of alternate post-transplant complications, such as Post Transplant Lymphoproliferative Disorder (PTLD).

Biopsy-proven incidence of acute cellular rejection

Outcome measures

Outcome measures
Measure
Alemtuzumab Induction
n=23 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Incidence of Biopsy-proven Acute Cellular Rejection
12 participants

SECONDARY outcome

Timeframe: 1 year

Population: Induction of Alemtuzumab at time of transplant may increase the likelihood of weaning off steroids sooner after transplant than patients who did not receive Alemtuzumab as induction immunosuppression medication.

As part of analysis for assessing effectiveness and safety of Alemtuzumab Induction at the time of transplant, it is important to assess the incidence in which patients enrolled were able to wean off of steroids post-transplant compared to historical controls.

Outcome measures

Outcome measures
Measure
Alemtuzumab Induction
n=21 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Incidence of Patients in Whom Steroids Are Not Used
5 participants

SECONDARY outcome

Timeframe: 1 year

Population: It is believed that patients whom received Alemtuzumab at the time of transplant and continue post-transplant with functioning grafts will have Tacrolimus whole blood concentrations less than 10ng/ml.

Tacrolimus whole blood concentrations less than 10 ng/ml

Outcome measures

Outcome measures
Measure
Alemtuzumab Induction
n=21 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Incidence of Patients in Whom Tacrolimus Whole Blood Concentration Less Than 10 ng/ml Are Being Used at 1-year Follow-up.
16 participants

SECONDARY outcome

Timeframe: 5 year

Population: Induction of Alemtuzumab at time of transplant may increase the likelihood of weaning off steroids sooner after transplant than patients who did not receive Alemtuzumab as induction immunosuppression medication.

As part of analysis for assessing effectiveness and safety of Alemtuzumab Induction at the time of transplant, it is important to assess the incidence in which patients enrolled were able to wean off of steroids post-transplant compared to historical controls.

Outcome measures

Outcome measures
Measure
Alemtuzumab Induction
n=21 Participants
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Incidence of Patients in Whom Steroids Are Not Used
9 participants

Adverse Events

Alemtuzumab Induction

Serious events: 6 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Alemtuzumab Induction
n=23 participants at risk
Intestine transplant recipients who receive induction with alemtuzumab prior to transplantation. Alemtuzumab: Alemtuzumab is a monoclonal antibody directed against the CD52 antigen. A single dose of 0.3-0.4 mg/kg is given to enrolled subjects at the time of intestine transplantation, 30 minutes after premedication with acetaminophen, diphenhydramine and methylprednisolone
Infections and infestations
PTLD
26.1%
6/23 • Number of events 6 • 5 years from time of enrollment

Other adverse events

Adverse event data not reported

Additional Information

Dr. Rakesh Sindhi

University of Pittsburgh

Phone: 412-692-6110

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place