Dear PALISI members,
Happy holidays! Please review the updates below regarding the following topics:
1. PALISI Platform Trial
2. Spring Meeting Hotel Reservation Link
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PALISI Platform Trial
Over the past several months the PALISI Platform Trial Steering Group (PTSG) has continued to meet and has reached consensus on several critical points that we wanted to share with you. We are writing to you today to solicit candidate initial Phase 3 trials that are ready to accompany our funding proposal.
Our purpose is to build a single integrated adaptive platform trial (APT) to efficiently evaluate multiple therapies, individually or in combination, focused on critically ill children in the pediatric intensive care unit. An APT allows for the concurrent evaluation of multiple groups of mutually exclusive treatments (referred to as “Domains”; e.g., high vs low target glucose control or high vs low PEEP) across several different therapeutic clusters (e.g., immune/endocrine modulation, respiratory support). We intend to enroll PICU patients and randomly assign each to a single treatment regimen that includes a combination of one or several treatment domains.
Our inclusion criteria will potentially include children admitted to the PICU for either primary or secondary cardiovascular or respiratory dysfunction (i.e., heart or lung failure; cardiac dysfunction would include children supported on inotropes or vasopressors, ECMO/VAD; respiratory dysfunction would include children supported on HHFNC, NIV, IMV, ECMO).
Our Primary Outcome variable (inferential whole) is ICU-free days.
Our Secondary Outcomes will include Mortality, MODS, plus Domain-Specific outcomes.
We have also outlined an organizational structure that will include a large interprofessional Steering Committee and several Cores.
At this time, we would like to identify interventions that YOU think are ready for a Phase 3 RCT that could become integrated into the APT as described above, including the intended patient population, and delineated outcomes.
If you or your group has an idea, we ask that you please provide a one-page outline including the following points. (Note that we are not looking for a Specific Aims page.)
Draft Title
Lead Investigator(s): Name(s) and contact information
PALISI subgroup: Has the study been vetted in a PALISI subgroup?
Therapeutic cluster: e.g., Immune/endocrine modulation, Respiratory support,
Transfusions, ECMO, other Critical Care therapies
Brief overview of the compelling rationale for the proposed study
o Importance of the research (Significance)
o Preliminary data supporting a Phase 3 trial
Domain – what intervention arms will be compared?
o Treatment arms
o Inclusion/exclusion criteria (major)
o Timing of randomization (if important)
To make sure that we are conducting important Phase 3 trials on the platform, we will curate the submitted ideas and vet study prioritization with the PALISI membership before/during the Spring meeting. Note that the lead investigator(s) submitting the
study idea, if it is prioritized, will become a member of the steering committee. Finally, this is intended as the starting point. We are seeking near-ready Phase III proposals to maximize our chances with funding opportunities. If this first phase is successful, then the PLATFORM will be expanded to accommodate earlier phase studies (i.e. Phase I/II) and potentially more therapeutic clusters and more domains. If you have any questions or concerns or other suggestions, please reach out to any member of the PTSG. Please send your outline to Lou Ventura at VENTURAL@chop.edu by January 31, 2024. If that timing doesn’t work for you, please drop us a note with a timeframe that would work.
Our best,
Martha A.Q. Curley (Curley@UPENN.edu)
Michael Agus (Michael.Agus@childrens.harvard.edu)
Roby Khemani (RKhemani@chla.usc.edu)
Tell Bennett (TELL.BENNETT@CUANSCHUTZ.EDU)
David Wypij (David.Wypij@cardio.chboston.org)
Roger J. Lewis (roger@berryconsultants.net)
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Palisi Spring Meeting Hotel Reservations Instruction
Hotel Booking Information is now available for the Spring Meeting. Please use one of the three options below to secure your reservation. Please be aware that a limited number of rooms are available on a first-come, first-served basis.
Option #1:
Attendees can make their reservations directly with the reservation office at 1-888-722-1274. Each person must identify themselves as part of the Palisi Spring Meeting PLSI24 to receive the group rates.
Option #2:
Personalized web page address: https://group.hilton.com/djos71
Option #3:
Hotel Web Page:
I. Select the desired arrival and departure dates
II. Select special rates
III. Add Group Code: PLSI24
IV. Continue with the reservation process
