Trachtman 2018.

Trial name or title	A phase II randomised, placebo‐controlled, double‐blind, parallel arms with switchover, pilot study to evaluate the efficacy and safety of intravenous abatacept in treatment resistant nephrotic syndrome (focal segmental glomerulosclerosis/ minimal change disease)
Methods	Randomised placebo controlled trial (quadruple blind)
Participants	90 patients aged ≥ 6 years with TRNS due to MCD or FSGS (Collapsing FSGS excluded), GFR ≥ 45 mL/min/1.73 m2. Patients stratified for age (< 18 and ≥ 18) and APOL1 risk status. Exclusions: Patients with recurrence of disease post transplant, secondary TRNS, DM, CHF, BMI > 40, recent or chronic infections
Interventions	1. 16 week parallel arms comparing IV abatacept and placebo (normal saline) on days 1, 14, 28 and then every 28 days 2. 16 week cross‐over with placebo group receiving abatacept and abatacept group receiving placebo 3. 169 day abatacept extension with all receiving abatacept 4. Weight tiered dose of abatacept from 500 to 1000 mg. Children < 18 years weighing < 75 kg: 10 mg/kg/dose 4. Standard immunosuppression (CNI, MMF, prednisone) unchanged in 1 months, ACEi, ARB
Outcomes	1. Difference in % of participants who achieve a renal response by 113 days (end of first 16 week parallel group study). Renal response defined as a ≥ 50% reduction in Up/C from baseline to day 113 with Up/C < 3g/g and eGFR > 90 mL/min/1.73 m2 (if below normal at baseline, remaining ≥ 75% of baseline. 2. Change in proteinuria, GFR, remission, quality of life (PROMIS), adverse events
Starting date	March 1, 2016. Estimated completion date June 2020
Contact information	Anna Greka: agreka@bwh.harvard.edu
Notes	27 study sites. NCT02592798. Sponsor: Bristol‐Myers Squibb

ACEi ‐ angiotensin converting enzyme inhibitors; ARB ‐ angiotensin receptor blocker; BMI ‐ body mass index; CHF ‐ chronic heart failure; CNI ‐ calcineurin inhibitor; DM ‐ diabetes mellitus; FSGS ‐ focal segmental glomerulosclerosis; (e)GFR ‐ (estimated) glomerular filtration rate; MCD ‐ minimal change disease; MesPGN ‐ mesangioproliferative glomerulonephritis; MMF ‐ mycophenolate mofetil; SDNS ‐ steroid‐dependent nephrotic syndrome; SRNS ‐ steroid‐resistant nephrotic syndrome; TRNS ‐ treatment‐resistant nephrotic syndrome; Up/C ‐ urinary protein creatinine ratio