This 76-week, 3-part Phase 1b/2 study is intended to evaluate the pharmacological properties
(pharmacokinetics and pharmacodynamics), safety, tolerability and preliminary effectiveness
of TOFA administrated to young adults (18-45 years) with moderately to severely active
SLE-CL. Subjects will be studied at the Cincinnati Children's Hospital Medical Center (CCHMC)
and in Cleveland at MetroHealth Medical Center.
Cohort 1 (n=10, weight > 40kg and age > 18 years and ≤ 45 years ) will undergo intense
PK-sampling to determine exposures following TOFA dosed at 5 mg BID. TOFA dose escalation
will not be considered for inadequate response of SLE-CL.
Cohort 2 (n=10, weight > 40kg and age > 18 years and ≤ 45 years) will be treated with the
same dose as Cohort 1. No PK sampling will occur for Cohort 2. Enrollment of Cohort 2 will
only start once Cohort 1 has completed 8 weeks of TOFA and results of PK analyses from Cohort
1 are available.
Part A (up to week 8) requires stable background medications;
Part B (up to week 24) allows for tapering of corticosteroids (CS) in the setting of
significant clinical improvement of SLE-CL as defined by a decrease in Cutaneous Lupus
Erythematosus Disease Area and Severity Index (CLASI) activity score by >50% from
baseline , and
Part C (until week 76) permits tapering of other background medications in subjects with
clinical remission of SLE-CL (CLASI activity score=0). TOFA dosing is kept stable during
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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