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Safety of Immunosuppression Minimization in Children and Adolescents After Kidney Transplantation

Phase 1
Completed
Conditions
Kidney Failure, Chronic
Kidney Transplantation
Immunosuppression
Interventions
Registration Number
NCT00768729
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

Kidney transplantation is widely considered to be the treatment of choice for children with End Stage Renal Disease (ESRD). The purpose of this study is to determine the safety of sirolimus monotherapy for long-term immunosuppression in children and adolescents after kidney transplantation.

Detailed Description

Improvements in surgical techniques, donor selection, immunosuppression practices, and the enhanced experience of specialized pediatric transplant teams have all led to marked improvements in patient and kidney graft survival in infants and young children Long-term graft survival rates decrease in adolescents 11 to 17 years of age. Several studies have suggested this decrease may be the result of noncompliance with immunosuppressive medications in this age group. Therefore, protocols that minimize the use of immunosuppressive medications, while retaining kidney function are necessary for improving graft and patient survival in children. The purpose of this study is to determine the safety of sirolimus monotherapy for long-term immunosuppression in children and adolescents after kidney transplantation.

This study will enroll 10 participants who previously completed the CCTPT-PC01 study. The accrual period is scheduled for 12 months. The study follow-up period will last 96 weeks. Patients from the CCTPT-PC01 study have been maintained on sirolimus and mycophenolate mofetil (MMF) since 2-3 months post transplant. Enrolled participants receiving (MMF) or Azathioprine at study entry will have their doses withdrawn gradually over a period of 6 months. Dosage will be reduced by 25% initially and by 25% every 2 months resulting in complete withdrawal by 6 months.

This study will consist of 11 study visits after screening and study entry. Study visits will occur at weeks 1, 8, 16, 24, 32, 40, 48, 60, 72, 84, and 96. A physical exam, vital signs, sirolimus levels, as well as blood and urine collection will occur at all visits. A renal biopsy will be performed at week 96.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
7
Inclusion Criteria
  • Participant and/or parent guardian able to understand and willing to provide informed consent
  • Previously enrolled and completed the CCTPT-PC01 study and within the 36 months post-completion timeframe prior to study entry
  • Currently receiving sirolimus and MMF or azathioprine therapy
  • No history of acute rejection episodes
  • No evidence of acute or chronic rejection on the 24 month CCTPT-PC01 protocol biopsy or any subsequent biopsy obtained after that time prior to study entry
  • PRA (Class I and II) less than 5% at study entry
  • No evidence of donor specific antibody at study entry
  • Stable renal function with GFR greater than 60 cc/min 1.73M^2 using the Schwartz calculated method
  • A negative pregnancy test for female participants of childbearing potential at study entry
  • Agreement by female and male participants to use FDA approved methods of contraception.
Exclusion Criteria
  • Total lymphocyte count less than 400 mm^3
  • Acute or chronic infection at study entry
  • Treatment with investigational drug within 1 month prior to study entry
  • Mental illness or history of drug or alcohol abuse that, in the opinion of the investigator, would interfere with the study
  • History of allergic reaction to Iodine GFR assay
  • History of malignancy within the past 12 months
  • Inability or unwillingness to give informed consent or comply with the study protocol

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
1SirolimusParticipants who have been maintained on MMF at study entry will start the study on 600 mg/m2 MMF orally daily. Participants who have been maintained on Azathioprine due to MMF intolerance will receive 1 mg/kg Azathioprine orally daily. Participants will continue receiving sirolimus throughout the study. However, MMF or Azathioprine will be withdrawn gradually over a period of at least 6 months. Dosage will be reduced by 25% initially and by 25% every subsequent 2 months resulting in complete withdrawal by 6 months.
1MMF or AzathioprineParticipants who have been maintained on MMF at study entry will start the study on 600 mg/m2 MMF orally daily. Participants who have been maintained on Azathioprine due to MMF intolerance will receive 1 mg/kg Azathioprine orally daily. Participants will continue receiving sirolimus throughout the study. However, MMF or Azathioprine will be withdrawn gradually over a period of at least 6 months. Dosage will be reduced by 25% initially and by 25% every subsequent 2 months resulting in complete withdrawal by 6 months.
Primary Outcome Measures
NameTimeMethod
Per-person incidence of acute rejection episodes and death or graft lossThroughout study
Secondary Outcome Measures
NameTimeMethod
Incidence of chronic allograft dysfunctionThroughout study
Incidence of sub-clinical rejectionThroughout study
Incidence of hospitalizationsThroughout study
Incidence of surgical complicationsThroughout study
Resumption of MMF or other therapyThroughout study
Incidence, severity, and treatment of anemia, hypertension, hyperlipidemia, proteinuria, thrombocytopenia, and leukopeniaThroughout study
Incidence, severity, and treatment of opportunistic infectionsThroughout study
Incidence of biopsy proven PTLDThroughout study
Renal function assessed by measured GFRAt baseline, week 48 and week 96
Development of donor-specific or non-specific anti-HLA antibodiesThroughout study
Evolution of immune response in cellular, humoral, and molecular assays from baseline through week 96Throughout study

Trial Locations

Locations (5)

Children's Hospital, Boston

🇺🇸

Boston, Massachusetts, United States

Children's Hospital, Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

Children's Hospital of Central California

🇺🇸

Madera, California, United States

UCSF Children's Hospital

🇺🇸

San Francisco, California, United States

Children's Hospital and Regional Medical Center, Seattle

🇺🇸

Seattle, Washington, United States

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