Open-label Study of Tofacitinib for Moderate to Severe Skin Involvement in Young Adults With Lupus

Systemic Lupus Erythematosus Clinical Trial

Official title: A 3-part Open-label Study Assessing Safety, Tolerability, Pharmacokinetic and -Dynamic Profiles, and Efficacy of Tofacitinib in Young Adults From Age 18 to 45 With Moderate to Severe Skin Involvement Due to Lupus

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

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 Part C. ;

Related Conditions & MeSH terms

• Cutaneous Lupus
• Lupus Erythematosus, Systemic
• Systemic Lupus Erythematosus

• NCT number: NCT03288324
• Study type: Interventional
• Source: Children's Hospital Medical Center, Cincinnati
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: August 23, 2017
• Completion date: December 1, 2022