A Study of PRN1008 in Patients With Pemphigus
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03762265|
Recruitment Status : Active, not recruiting
First Posted : December 3, 2018
Last Update Posted : April 26, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pemphigus||Drug: PRN1008 Oral Tablet Drug: Placebo Oral Tablet||Phase 3|
Approximately 120 male or female patients with newly diagnosed or relapsing moderate to severe pemphigus (pemphigus vulgaris [PV] or pemphigus foliaceus [PF]) will be enrolled in the trial worldwide.
The trial will last 68 weeks (approximately 17 months) for each participant. For patients eligible to enroll in the Long Term Extension, the trial may last up to 116 weeks.
Patients will be 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).
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||131 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor PRN1008 in Moderate to Severe Pemphigus|
|Actual Study Start Date :||January 8, 2019|
|Estimated Primary Completion Date :||August 2021|
|Estimated Study Completion Date :||February 2023|
Drug: PRN1008 Oral Tablet
Bruton's agammaglobulinemia tyrosine kinase (BTK) inhibitor
|Placebo Comparator: Placebo||
Drug: Placebo Oral Tablet
- The proportion of patients who are in Complete Remission (CR) from Week ≤ 29 to Week 37 with prednisone dose of ≤5 mg/day [ Time Frame: 37 Weeks ]Complete Remission (CR) defined as the absence of new and established lesions
- Cumulative Corticosteroids (CS) dose over first 36 weeks (to Week 37) [ Time Frame: 37 Weeks ]Cumulative CS dose over first 36 weeks (to Week 37)
- Change in EuroQOL-5 Dimension 5 Level (EQ-5D-5L) score from baseline to Weeks 5, 13, 25, and 61 [ Time Frame: 61 Weeks ]EQ-5D-5L questionnaire has 5 dimensions: "Mobility", "Self-Care," "Usual Activities", "Pain/Discomfort", "Anxiety/Depression", and all dimensions are described by 5 problem levels.
- Change in Autoimmune Bullous Disease Quality of Life (ABQOL) from baseline to Weeks 5, 13, 25, and 61 [ Time Frame: 61 Weeks ]The ABQOL questionnaire is a 17 item questionnaire assessing quality of life in patients with autoimmune blistering diseases
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03762265