MedPath

A Paediatric, Open, Follow up Study With Modigraf Examining Safety and Efficacy in de Novo Allograft Recipients

Phase 4
Terminated
Conditions
Heart Transplantation
Kidney Transplantation
Liver Transplantation
Interventions
Registration Number
NCT01371344
Lead Sponsor
Astellas Pharma Europe Ltd.
Brief Summary

The purpose of this study, a follow up to study FG506-CL-0403, is to see how safe and effective Modigraf® is (Part A) and to see how safe and effective it is to change your child's medication from Modigraf® to Prograf® (Part B).

Detailed Description

To monitor the safety and efficacy of Modigraf® (tacrolimus granules) in stable paediatric allograft recipients (Part A) and to monitor dose changes and tacrolimus whole blood trough levels after conversion from a Modigraf based Immunosuppression regimen to a Prograf® based Immunosuppression regimen (Part B).

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
47
Inclusion Criteria

F506-CL-0404 Part A

  • Subject was ≤12 years of age at enrolment into study F506-CL-0403
  • Subject received at least one dose of Modigraf in the F506-CL-0403 study

F506-CL-0404 Part B

  • Subject received at least one dose of Modigraf in the F506-CL-0403 study
  • Subject participated in F506-CL-0404 Part A
  • Subject has continuously been dosed with Twice daily (BID) Modigraf since the End of Study Visit for Part A (ESVA) from F506-CL-0404 Part A
  • Subject is stable and has had no dose changes in the preceding 2 weeks
Exclusion Criteria

F506-CL-0404 Part A

  • As all subjects included in this study conform to the exclusion criteria in study F506-CL-0403, hence no specific exclusion criteria are relevant for this study

F506-CL-0404 Part B

  • There are no specific exclusion criteria for this study

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Part B: All Participants (Tacrolimus capsules)Tacrolimus capsulesIn Part B of the study, participants who are heart, kidney or liver transplant recipients and who are converted from tacrolimus granules-based immunosuppression regimen, receive tacrolimus capsules twice daily for 1 month and thereafter receive commercially available tacrolimus capsules.
Part A: Heart Transplant (Tacrolimus granules)Tacrolimus granulesIn Part A of the study, participants who are heart transplant recipients receive tacrolimus granules-based immunosuppressive regimen twice daily for a maximum of 1 year or until commercial availability of tacrolimus granules in the participant's country.
Part A: Liver Transplant (Tacrolimus granules)Tacrolimus granulesIn Part A of the study, participants who are liver transplant recipients receive tacrolimus granules-based immunosuppressive regimen twice daily for a maximum of 1 year or until commercial availability of tacrolimus granules in the participant's country.
Part A: Kidney Transplant (Tacrolimus granules)Tacrolimus granulesIn Part A of the study, participants who are kidney transplant recipients receive tacrolimus granules-based immunosuppressive regimen twice daily for a maximum of 1 year or until commercial availability of tacrolimus granules in the participant's country.
Primary Outcome Measures
NameTimeMethod
Part A: Number of Participants with Acute Rejection EpisodesUp to 12 months

Rejection episodes/acute rejections are indicated by clinical and/or laboratory signs, and are classified according to their rejection specific treatment: •Spontaneously Resolving Acute Rejection: not treated with new or increased corticosteroid medication, antibodies or any other medication and resolved, irrespective of any tacrolimus dose changes; •Corticosteroid Sensitive Acute Rejection: treated with new or increased corticosteroid medication only and which has resolved, irrespective of any tacrolimus dose changes; •Corticosteroid Resistant Acute Rejection: did not resolve following treatment with corticosteroids; - Resolved with further treatment: any acute rejection with an end date AND a treatment other than corticosteroid used; - Unresolved with further treatment: any acute rejection with no end date AND a treatment other than corticosteroid used; - Unresolved with no further treatment: any acute rejection with no end date AND ONLY corticosteroid treatment used.

Part A; Number of Participants with Biopsy-proven Acute Rejection Episodes (BPARs)Up to 12 months

BPAR episodes are defined as acute rejection episodes confirmed by biopsy, and are classified according to their rejection specific treatment: •Spontaneously Resolving Acute Rejection: not treated with new or increased corticosteroid medication, antibodies or any other medication and resolved, irrespective of any tacrolimus dose changes; •Corticosteroid Sensitive Acute Rejection: treated with new or increased corticosteroid medication only and which has resolved, irrespective of any tacrolimus dose changes; •Corticosteroid Resistant Acute Rejection: did not resolve following treatment with corticosteroids; - Resolved with further treatment: any acute rejection with an end date AND a treatment other than corticosteroid used; - Unresolved with further treatment: any acute rejection with no end date AND a treatment other than corticosteroid used; - Unresolved with no further treatment: any acute rejection with no end date AND ONLY corticosteroid treatment used.

Part A: Severity of BPARsUp to 12 months

The severity of BPARs is categorized with specific criteria by organ: For kidney transplant participants, according to Banff '97 Diagnostic categories for renal allograft biopsies - Banff '07 update (C4d deposition, Acute antibody-mediated rejection I, II, and III, Acute T cell mediated rejection IA, IB, IIA, IIB and III); for liver transplant participants, according to 1997 Banff Schema for Grading of Liver Allograft Rejection - Rejection Activity Index score (sum of grades: 1-mild, 2-moderate, 3-severe; range from 0-9); for heart, according to Standardized Nomenclature of the International Society of Heart and Lung Transplantation - Standardised Cardiac Biopsy Grading: Acute Cellular Rejection 2004 (mild, moderate, severe).

Part A: Patient SurvivalUp to 12 months

Patient survival is reported as the number of deaths that occurred during Part A of the study.

Part A: Graft SurvivalUp to 12 months

Graft survival is reported as the number of participants who experienced graft loss. Graft loss is defined as retransplantation or death or return to pretransplantation treatment modality for 6 weeks or longer. Additionally, kidney transplanted participants with ongoing dialysis at the end of study is counted as participants with graft loss.

Part A: Number of Participants with Adverse Events (AEs)From first dose of study drug up to 30 days after last dose of study drug (up to 13 months)

Safety is assessed by AEs, which includes abnormalities identified during a medical test (e.g. clinical laboratory tests, vital signs, etc.) if the abnormality induces clinical signs or symptoms, needs active intervention, interruption or discontinuation of study medication or is clinically significant. A serious AE (SAE) is an event resulting in death, persistent or significant disability/incapacity or congenital anomaly or birth defect, is life-threatening, requires or prolongs hospitalization or is considered medically important. A treatment emergent adverse event (TEAE) is defined as an AE observed after investigational drug administration.

Part A: Tacrolimus Mean Trough LevelsDay 1, months 1, 2, 3, 6, 9, 12 (prior to each study drug dosing)
Part A: Number of Dose AdjustmentsMonths 1, 2, 3, 6, 9, 12

Study drug doses are adjusted based on clinical evidence of efficacy and occurrence of adverse events, and taking into consideration the recommended whole blood trough level range of 5-20 ng/ml.

Part B: Number of Participants with AEsFrom first dose of study drug (tacrolimus capsules) up to 7 days after last dose (up to 38 days)

Safety is assessed by AEs, which included abnormalities identified during a medical test (e.g. clinical laboratory tests, vital signs, etc.) if the abnormality induces clinical signs or symptoms, needs active intervention, interruption or discontinuation of study medication or is clinically significant. A SAE is an event resulting in death, persistent or significant disability/incapacity or congenital anomaly or birth defect, is life-threatening, requires or prolongs hospitalization or is considered medically important. A TEAE is defined as an AE observed after investigational drug administration.

Part B: Tacrolimus Trough Levels Prior to and After ConversionDay -1 up to 1 month

Values prior to conversion are the last trough level prior to first dose of study drug (tacrolimus capsules). Values after conversion are the first trough level after first dose of study drug (tacrolimus capsules).

Part B: Number of Dose AdjustmentsFrom first dose of study drug up to 1 month
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (12)

Site BE40 Clinique Univ. Saint Luc

🇧🇪

Brussels, Belgium

Site FR60 Groupement Hospitalier EST

🇫🇷

Bron, France

Site DE31 Kliniken der Medizinischen Hoc

🇩🇪

Hannover, Germany

Site DE30 Universitätsklin Heidelberg

🇩🇪

Heidelberg, Germany

Site FR61 Hopital Robert Debre

🇫🇷

Paris Cedex 19, France

Site PL50 Centrum Zdrowia Dziecka

🇵🇱

Warsaw, Poland

Site ES20 Hospital Universitario La Paz

🇪🇸

Madrid, Spain

Site ES22 H.U. Gregorio Maranon

🇪🇸

Madrid, Spain

Site ES23 Hospital Universitario La Paz

🇪🇸

Madrid, Spain

Site GB14 Alder Hey Children Hospital

🇬🇧

Liverpool, United Kingdom

Site GB13 Cent. Manchester Uni. Hospital

🇬🇧

Manchester, United Kingdom

Site ES21 Hospital Universitario La Paz

🇪🇸

Madrid, Spain

© Copyright 2025. All Rights Reserved by MedPath