This study was designed to assess the evolution of renal function and to collect efficacy, safety, and tolerability data of everolimus in co-exposure with reduced CNI in paediatric liver transplant recipients.
Study is completed (was active and ongoing but no longer recruiting since December 2014). The study Data Monitoring Committee meeting communicated to Novartis the following safety findings in the study population: high rate of premature discontinuation of study medication, high rate of post-transplant lymphoproliferative disease and high rate of related serious infections leading to hospitalization. In light of the safety findings, Novartis followed the DMC recommendation to discontinue the study medication in this age group and to stop enrolling new patients in this study (regardless of age).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
56
Immunosuppression after liver transplantation. Pediatric transplant recipients received a starting dose of 0.8 mg/m\^2/dose in combination wit Cyclosporine A or 2.0 mg/m\^2/dose in combination with tacrolimus, twice-daily. Thereafter, doses were adjusted to achieve everolimus C-0h blood trough level between 3 to 8 ng/ml.
Change From Baseline in Estimated Glomerular Filtration Rate - Month 12
Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 12.
Time frame: Baseline, Month 12
Kaplan-Meier Estimates for Failure Rates of Efficacy Endpoints
The proportion of patients with composite efficacy failure (treated biopsy proven acute rejection\[tBPAR\], graft loss \[GL\] , death \[D\]) before/at Month 12 and Month 24, estimated with Kaplan-Meier (KM) methods and the proportion of patients who experienced any of the components of composite efficacy failure (tBPAR, GL, D) before/at Month 12 and Month 24, separately for each component. AR: acute rejection; BPAR: biopsy proven acute rejection. Rate = Kaplan-Meier estimate for failure in %; CI = confidence interval for failure rate.
Time frame: At 12-month and 24-month after start of study drug
Change From Baseline in Estimated Glomerular Filtration Rate - Month 24
Evolution of renal function assessed by estimated Glomerular Filtration Rate (eGFR) calculated by the Chronic Kidney Disease in Children (CKiD) Schwartz formula (Schwartz 2009), expressed in mean change in eGFR of CKiD between start of study (baseline assessment) and Month 24.
Time frame: Baseline, Month 24
Growth Development - Height at Baseline and Month 12
Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (\<=5, \>5-25, \>25-50, \>50-75, \>75-95 and \>95% percentile).
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Novartis Investigative Site
Los Angeles, California, United States
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New Haven, Connecticut, United States
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Chicago, Illinois, United States
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St Louis, Missouri, United States
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New York, New York, United States
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Charleston, South Carolina, United States
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Salt Lake City, Utah, United States
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Madison, Wisconsin, United States
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Parkville, Victoria, Australia
...and 22 more locations
Time frame: Baseline, Month 12
Growth Development - Weight at Baseline and Month 12
Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (\<=5, \>5-25, \>25-50, \>50-75, \>75-95 and \>95% percentile).
Time frame: Baseline, Month 12
Growth Development - Weight at Baseline and Month 24
Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (\<=5, \>5-25, \>25-50, \>50-75, \>75-95 and \>95% percentile).
Time frame: Baseline, Month 24
Growth Development - Height at Baseline and Month 24
Individual growth measurements were compared with the gender and age-specific growth percentiles in the CDC growth charts for the US population. Each value observed is thus represented by the (approximated) percentage of subjects with a lower value in the reference population. Changes were calculated on this scale and thus express the change in growth measurements relative to the percentiles in the CDC growth charts. Patients were classified into growth percentile categories (\<=5, \>5-25, \>25-50, \>50-75, \>75-95 and \>95% percentile).
Time frame: Baseline, Month 24