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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01598987




Registration number
NCT01598987
Ethics application status
Date submitted
1/03/2012
Date registered
15/05/2012
Date last updated
16/05/2017

Titles & IDs
Public title
Efficacy, Safety and Tolerability of Everolimus in Combination With Reduced Exposure Cyclosporine or Tacrolimus in Paediatric Liver Transplant Recipients.
Scientific title
A 24-month, Multi-center, Single Arm, Prospective Study to Evaluate Renal Function, Efficacy, Safety and Tolerability of Everolimus in Combination With Reduced Exposure Cyclosporine or Tacrolimus in Paediatric Liver Transplant Recipients.
Secondary ID [1] 0 0
2011-003069-14
Secondary ID [2] 0 0
CRAD001H2305
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Renal Function 0 0
Liver Transplant 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Introduction of everolimus with reduced cyclosporine or tacrolimus dose, the earliest 1 month and the latest 6 months after liver transplantation.

Experimental: Everolimus based regimen - Conversion at Baseline from an immunosuppressive regimen which contains either cyclosporine (CsA) or tacrolimus (TAC) with or without mycophenolic acid (MPA), with or without corticosteroids in a regimen which contains everolimus combined reduced dose of either cyclosporine (CsA) or tacrolimus (TAC).

The dosing schedule was twice daily, 12 hours apart.


Treatment: Drugs: Introduction of everolimus with reduced cyclosporine or tacrolimus dose, the earliest 1 month and the latest 6 months after liver transplantation.
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Estimated Glomerular Filtration Rate - Month 12
Timepoint [1] 0 0
Baseline, Month 12
Secondary outcome [1] 0 0
Kaplan-Meier Estimates for Failure Rates of Efficacy Endpoints
Timepoint [1] 0 0
At 12-month and 24-month after start of study drug
Secondary outcome [2] 0 0
Change From Baseline in Estimated Glomerular Filtration Rate - Month 24
Timepoint [2] 0 0
Baseline, Month 24
Secondary outcome [3] 0 0
Growth Development - Height at Baseline and Month 12
Timepoint [3] 0 0
Baseline, Month 12
Secondary outcome [4] 0 0
Growth Development - Weight at Baseline and Month 12
Timepoint [4] 0 0
Baseline, Month 12
Secondary outcome [5] 0 0
Growth Development - Weight at Baseline and Month 24
Timepoint [5] 0 0
Baseline, Month 24
Secondary outcome [6] 0 0
Growth Development - Height at Baseline and Month 24
Timepoint [6] 0 0
Baseline, Month 24

Eligibility
Key inclusion criteria
Key

Signed informed consent from both parents or legal guardian(s) prior to patient participation in the study.

Paediatric liver transplant recipients aged greater than or equal to 1 month and younger than 18 years of age.

Paediatric recipients at the earliest 1 month and latest 6 month after liver transplantation.

Key
Minimum age
1 Month
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients with hepato-biliary malignancies and/or patients transplanted due to fulminant hepatitis /acute liver failure.

Presence of thrombosis of any major hepatic arteries, major/reconstructed hepatic veins, portal vein or inferior vena cava at any time prior to the start of study drug.

Patients with serum creatinine value >2 times age-related ULN at Baseline or who received renal replacement therapy within one week prior to the start of study drug and patients with a confirmed spot urine protein/creatinine ratio indicating a urinary protein excretion >500 mg/m2/24 hrs, at Baseline.

Patients with clinically significant systemic infection and/or in a critical care setting requiring life support measures such as mechanical ventilation, dialysis, or vasopressor agents.

Patients with a known hypersensitivity to the drugs used on study or their class, or to any of the excipients.

Pregnant or nursing (lactating) female patients, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive ßHCG laboratory test (>9 mIU/mL) at Baseline.

Female patients of child-bearing potential, defined as all women physiologically capable of becoming pregnant, UNLESS they agree for abstinence from sexual activity.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/10/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/06/2016
Sample size
Target
Accrual to date
Final
56
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
South Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
United States of America
State/province [9] 0 0
Wisconsin
Country [10] 0 0
Belgium
State/province [10] 0 0
Bruxelles
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Denmark
State/province [12] 0 0
København Ø
Country [13] 0 0
France
State/province [13] 0 0
Bron
Country [14] 0 0
Germany
State/province [14] 0 0
Bonn
Country [15] 0 0
Germany
State/province [15] 0 0
Essen
Country [16] 0 0
Germany
State/province [16] 0 0
Hamburg
Country [17] 0 0
Germany
State/province [17] 0 0
Hannover
Country [18] 0 0
Germany
State/province [18] 0 0
Regensburg
Country [19] 0 0
Germany
State/province [19] 0 0
Tübingen
Country [20] 0 0
Hungary
State/province [20] 0 0
Budapest
Country [21] 0 0
Italy
State/province [21] 0 0
BG
Country [22] 0 0
Italy
State/province [22] 0 0
ITA
Country [23] 0 0
Italy
State/province [23] 0 0
PD
Country [24] 0 0
Italy
State/province [24] 0 0
TO
Country [25] 0 0
Spain
State/province [25] 0 0
Catalunya
Country [26] 0 0
Spain
State/province [26] 0 0
Madrid
Country [27] 0 0
Sweden
State/province [27] 0 0
Stockholm
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Birmingham
Country [29] 0 0
United Kingdom
State/province [29] 0 0
Leeds
Country [30] 0 0
United Kingdom
State/province [30] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01598987
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01598987