ClinicalTrials.gov
ClinicalTrials.gov Menu

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03762265
Recruitment Status : Recruiting
First Posted : December 3, 2018
Last Update Posted : December 3, 2018
Sponsor:
Information provided by (Responsible Party):
Principia Biopharma Inc.

Brief Summary:
This is a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (Blinded Treatment Period) followed by an Open-Label Extension Period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus.

Condition or disease Intervention/treatment Phase
Pemphigus Drug: PRN1008 Oral Tablet Drug: Placebo Oral Tablet Phase 3

Detailed Description:

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.

Patients will be randomized at Day 1, using a 1:1 ratio to receive PRN1008 or placebo twice per day, and by relapsing/newly diagnosed disease history (newly diagnosed defined as within 6 months of Screening).


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : December 2018
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : March 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus

Arm Intervention/treatment
Experimental: Experimental Drug: PRN1008 Oral Tablet
Bruton's agammaglobulinemia tyrosine kinase (BTK) inhibitor

Placebo Comparator: Placebo Drug: Placebo Oral Tablet
Placebo




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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)

  2. 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.

  3. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female aged 18-80 years old with moderate to severe pemphigus, newly diagnosed or relapsing pemphigus vulgaris (PD) or pemphigus foliaceus (PF); positive anti-dsg1 or 3 autoantibody titer
  • Laboratory clearance with adequate hematologic, hepatic, and renal function, body mass index (BMI) >17.5
  • Female patients who are of reproductive potential must agree for the duration of active treatment to use an effective means of contraception
  • Able to provide written informed consent

Exclusion Criteria:

  • Previous use of a Bruton's tyrosine kinase (BTK) inhibitor
  • Pregnant or lactating women
  • Clinically significant abnormalities, ECG findings or poorly controlled atrial fibrillation
  • A history of malignancy of any type, other than surgically excised non-melanoma skin cancers or in situ cervical cancer within 5 years before Day 1
  • Use of excluded medications within specified timeframes (proton pump inhibitors, immunologic response modifiers, investigational drugs, CYP3A inducers or inhibitors, CYP3A-sensitive substrates)
  • History of drug abuse or excessive alcohol use
  • Blood or blood product donation of a unit or more within 4 weeks
  • Live vaccine within 28 days prior to Day 1 or plan to receive one during the trial
  • Clinically significant diseases, positive at Screening of HIV and hepatitis A and B, history of serious infections and active latent tuberculosis (TB)

Information from the National Library of Medicine

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


Contacts
Contact: Ann Neale 1-833-477-6700 clinicaltrials@principiabio.com

Locations
United States, Florida
Central Recruiting (Principia Biopharma) Recruiting
Coral Gables, Florida, United States, 33134
Sponsors and Collaborators
Principia Biopharma Inc.
Investigators
Study Director: Dolca Thomas, M.D. Principia Biopharma Inc.

Responsible Party: Principia Biopharma Inc.
ClinicalTrials.gov Identifier: NCT03762265     History of Changes
Other Study ID Numbers: PRN1008-012
First Posted: December 3, 2018    Key Record Dates
Last Update Posted: December 3, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Principia Biopharma Inc.:
Pemphigus Vulgaris (PV)
Pemphigus Foliaceus (PF)

Additional relevant MeSH terms:
Pemphigus
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases