NCT03762265

A Study of PRN1008 in Patients With Pemphigus

A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor Rilzabrutinib (PRN1008) in Moderate to Severe Pemphigus

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 131 (actual)
• Sponsor: Principia Biopharma, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This was a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (blinded treatment \[BT\] period) followed by an open-label extension \[OLE\] period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the open-label extension period, eligible participants might continue in a long term extension (LTE) Period of 48 weeks.

Detailed description

A total of 131 male or female participants with newly diagnosed or relapsing moderate to severe pemphigus (pemphigus vulgaris \[PV\] or pemphigus foliaceus \[PF\]) were enrolled in the trial worldwide. The trial would last 68 weeks (approximately 17 months) for each participant. For participants eligible to enroll in the LTE, the trial might last up to 116 weeks. Participants were randomized at Day 1, using a 1:1 ratio to receive PRN1008 or placebo twice per day, by relapsing/newly diagnosed disease history (newly diagnosed defined as within 6 months of screening).

Conditions

• Pemphigus

Interventions

Type	Name	Description
DRUG	Rilzabrutinib	Pharmaceutical form: Tablet Route of administration: Oral
DRUG	Placebo	Pharmaceutical form: Tablet Route of administration: Oral

Timeline

• Start date: 2019-01-08
• Primary completion: 2021-07-30
• Completion: 2021-12-17
• First posted: 2018-12-03
• Last updated: 2023-08-02
• Results posted: 2022-09-21

Locations

88 sites across 19 countries: United States, Argentina, Australia, Brazil, Bulgaria, Canada, Croatia, France, Germany, Greece, Israel, Italy, Poland, Serbia, Spain, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study