NCT03288324

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

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
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Children's Hospital Medical Center, Cincinnati · Academic / Other
• Sex: All
• Age: 18 Years – 45 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Cutaneous Lupus
• Systemic Lupus Erythematosus

Interventions

Type	Name	Description
DRUG	Tofacitinib	Tofacitinib 5 mg twice daily

Timeline

• Start date: 2017-08-23
• Primary completion: 2022-11-01
• Completion: 2023-12-01
• First posted: 2017-09-20
• Last updated: 2026-03-03
• Results posted: 2024-07-30

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study