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Everolimus vs Tacrolimus in Pediatric Heart Transplant: Phase III Trial

This study aims to compare the efficacy and safety of everolimus and low-dose tacrolimus to tacrolimus and mycophenolate mofetil in preventing long-term complications after pediatric heart transplantation. The trial will involve 242 children at 20 sites, with a follow-up period of 30 months.

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Everolimus vs Tacrolimus in Pediatric Heart Transplant: Phase III Trial

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  1. Phase III Multi-Center Open-Label Randomized Trial Comparing Everolimus and Low-Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil Initiated at 6 Months Post-Heart Transplant to Prevent Long-term Complications after Pediatric Heart TransplantationIND #127980 Kevin Daly MD Christopher Almond MD MPH PHTS Annual Meeting Tuesday April 26, 2016 Washington, DC

  2. Developments Since the AHA Oct 2015 Pre-IND meeting with FDA Nov 2015 AHA meeting, feedback ped HT community Dec 2015 Protocol finalized with FDA/MD input Jan 2016 IND submitted to FDA Feb 2016 OOPD Grant (R01) Submitted to FDA ($2M) Feb 2016 IND officially approved by FDA Mar 2016 Novartis indicates willingness to provide drug Mar 2016 NHLBI consultation for funding (>500k/year) Apr 2016 Submit formal LOI to NHLBI ($9M) Apr 2016 ‘Enduring Hearts’ funding consultation

  3. Study Overview • RCT at 6 months post HT comparing TAC/MMF and EVL/LDTAC in children <21 years who are rejection free at 6 months post-HT • Study will randomize 242 children at 20 sites with a follow-up period 30 months. • Primary efficacy endpoint is a reduction in the MATE score for CAV, CKD and BPACR (three adverse events). • Primary safety endpoint is not increase in the predicted graft loss based on the overall MATE score (all six adverse events)

  4. MATE Score

  5. Science to Date • Survey of Pediatric Transplant Center Clinical Practice led by Daly and Castleberry: • Abstract presented at 2015 AHA Scientific Sessions • Manuscript submitted to Pediatric Transplantation • MATE Score Validation led by Almond, Daly and Desai • Abstract to be presented on Fri, Apr 29 at 3 PM • Manuscript being split into a statistical and clinical paper • Survey regarding safety concerns with extemporaneous compounding of tacrolimus • Identified important safety signal which would support the development of a commercially available compounded form of tacrolimus • Abstract to be submitted 2016

  6. Timeline Going Forward Jun 2016: Submit NHLBI R01 (Stanford CCC/Boston DCC) Nov 2016: Funding decision on FDA and NIH grant applications If funded – Dec 2016: IRB submissions Feb 2017: FDA funding commences Apr 2017: NIH funding commences May 2017: Trial accrual begins April 2019: Trial accrual complete April 2022: 3-year follow-up complete

  7. Study Sites • 8 core centers • 12 auxiliary centers (April-May 2016) • US, UK, ?Canada

  8. Steering Committee Kevin Daly MD Boston Christopher Almond MD MPH Stanford Lynn Sleeper ScD Boston (PI DCC) Joe Rossano MD MS CHOP Elfi Pahl MD Chicago Melanie Everitt MD Denver Scott Auerbach MD Denver Seth Hollander MD Stanford Chesney Castleberry MD Wash U/St. Louis Ashwin Lal MD Primary/Utah Matt Bock MD Loma Linda Mathew Fenton MD GOSH

  9. Thanks!Questions?

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