MULTICENTER RANDOMIZED CLINICAL TRIAL OF FSGS IN CHILDREN

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MULTICENTER RANDOMIZED CLINICAL TRIAL OF FSGS IN CHILDREN AND YOUNG ADULTS J Gassman, R

MULTICENTER RANDOMIZED CLINICAL TRIAL OF FSGS IN CHILDREN AND YOUNG ADULTS J Gassman, R Fine, A Friedman, D Gipson, T Greene, R Hogg, F Kaskel, M Moxey-Mims, M Radeva, N Siegel*, H Trachtman, S Watkins NIH NIDDK, Bethesda MD *deceased

Objectives • Randomized trial to compare 2 therapeutic interventions for patients 2 – 40

Objectives • Randomized trial to compare 2 therapeutic interventions for patients 2 – 40 yrs of age with steroid resistant FSGS • Facilitate the investigation of complex pathobiology of FSGS • Target sample size is 207 randomized patients to achieve 80% power to detect 17. 9% absolute difference in remission rate

Outcomes Primary Outcome • Attainment of partial or complete remission (6 -level ordinal scale)

Outcomes Primary Outcome • Attainment of partial or complete remission (6 -level ordinal scale) Main Secondary Outcome • No relapse after withdrawal of immunosuppressive agents Secondary Outcomes • Treatment failures • Change in e. GFR • Side effects • Quality of life • Biological materials

Primary Outcome Measures • The primary outcome is defined as achievement of remission from

Primary Outcome Measures • The primary outcome is defined as achievement of remission from proteinuria during the first 52 weeks after randomization • *Complete remission: Decline in UP/C to no > than 0. 2 • *Partial remission: 50% or > decline to a level between 0. 2 and 2. 0 * Mean of 2 baseline measurements of first morning urine

Definition of Primary Outcome at 52 Weeks for Participants Achieving a Partial or Complete

Definition of Primary Outcome at 52 Weeks for Participants Achieving a Partial or Complete Remission at 26 Weeks Remissi on Sustained Categor complete y remission Level Definitio n 1 Limited complete remission Partial remission No remissio n 2 3 4 Partial remission at W 52 No remissio n at W 52 Complete remission at remission W 52 but througho Up/c > 0. 2 ut weeks between 26 -52 weeks 26 and 52

Main Secondary Outcome Measures • 5 -level ordinal categorical outcome based on the participant’s

Main Secondary Outcome Measures • 5 -level ordinal categorical outcome based on the participant’s level of proteinuria during the period from W 52 through W 78 following withdrawal of CSA or MMF/Pulse steroids • Participants remain on ACEi / ARB

Definition of Main Secondary Outcome at Week 78 for Participants with a Primary Outcome

Definition of Main Secondary Outcome at Week 78 for Participants with a Primary Outcome Level of at least 3 Remission Category Level Definition Full persistence of complete remission Limited persistence of complete remission Partial remission No remission A B C D Complete remission throughout weeks 52 -78 Complete remission at week 78, but Up/c between 0. 2 and 2. 0 between weeks 52 and 78 Partial remission Failure to at week 78, and maintain at partial or complete least a partial remission between remission weeks 52 and 78 after week 52 Note: Participants who were assigned to levels 4, 5, or 6 for the primary outcome at the 26 and/or 52 week assessment will be assigned to level E for the main secondary outcome.