The study is a 1-year 2-part double-blinded placebo controlled 2-arm clinical trial. Treatment arms are (1) MMF dosed as per body-surface area (MMFBSA; 600mg/m2 body surface area per dose about every 12 hours) and (2) pharmacokinetically-guided precision-dosing of MMF (MMFPK; MMF dosed twice daily to achieve an area under the concentration-time curve (AUC0-12h) of MPA \>60-70 mg\*h/L. The study goal is to determine the safety and efficacy of MMFPK compared to MMFBSA for the treatment of proliferative LN in subjects 8 to \<21 years.
Subjects will be randomized 1:1 to receive blinded treatment with MMFPK or MMFBSA for up to 53 weeks. The primary endpoint, clinical remission of LN, is measured at the end of Part 1 at week 26. Subjects in the MMFBSA arm who have only partial renal response (PRR) at the end of Part 1 will newly receive MMFPK upon entering Part 2 of the study (week 26 - 53). Subjects with complete renal responses (CRR) at the end of Part 1 will continue the same dosing regimen of MMF (MMFBSA or MMFPK) in Part 2 as was given in Part 1 of the study. Subjects in the MMFPK arm with PRR at the end of Part 1 will enter Part 2 and continue in the MMFPK arm. Subjects who are LN non-responders by the end of Part 1 at week 26 will be considered treatment failures and discontinued from the study intervention. All subjects who are discontinued from the study intervention for reasons of efficacy or safety will receive LN treatment and monitoring as per the treating physician's decision. However, these subjects will be asked to participate in study visits at weeks 26 and 53/End of Study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
105
MMF dosed 600mg/m2 body surface area per dose about every 12 hours
MMF dosed twice daily to achieve an area under the concentration-time curve (AUC 0-12h) of MPA \>=60-70 mg\*h/L
University of California, San Francisco
San Francisco, California, United States
RECRUITINGChildren's Hospital Colorado
Aurora, Colorado, United States
RECRUITINGEmory Children's Center
Atlanta, Georgia, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
RECRUITINGUniversity of Chicago Medicine- Comer Children's
Chicago, Illinois, United States
RECRUITINGWashington University in St. Louis School of Medicine
St Louis, Missouri, United States
RECRUITINGHackensack University Medical Center
Hackensack, New Jersey, United States
RECRUITINGHospital for Special Surgery
New York, New York, United States
RECRUITINGChildren's Hospital at Montefiore
New York, New York, United States
RECRUITINGUniversity of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
RECRUITING...and 9 more locations
To compare the efficacy of MMFPK therapy to the efficacy of MMFBSA therapy
the percentage of subjects achieving at least partial remission of LN (PRR) as per the adapted ACR/EULAR Criteria at Week 26
Time frame: 26 week
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