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Trial record 2 of 2 for:    prn1008 | pemphigus

A Study of PRN1008 in Patients With Pemphigus

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ClinicalTrials.gov Identifier: NCT03762265
Recruitment Status : Recruiting
First Posted : December 3, 2018
Last Update Posted : April 2, 2020
Sponsor:
Information provided by (Responsible Party):
Principia Biopharma Inc.

Tracking Information
First Submitted Date  ICMJE November 29, 2018
First Posted Date  ICMJE December 3, 2018
Last Update Posted Date April 2, 2020
Actual Study Start Date  ICMJE January 8, 2019
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 30, 2018)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 30, 2018)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of PRN1008 in Patients With Pemphigus
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor PRN1008 in Moderate to Severe Pemphigus
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. After completing the Open-Label Extension Period, eligible patients may continue in a Long Term Extension Period of 48 weeks.
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. 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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Pemphigus
Intervention  ICMJE
  • Drug: PRN1008 Oral Tablet
    Bruton's agammaglobulinemia tyrosine kinase (BTK) inhibitor
  • Drug: Placebo Oral Tablet
    Placebo
Study Arms  ICMJE
  • Experimental: Experimental
    Intervention: Drug: PRN1008 Oral Tablet
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo Oral Tablet
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 30, 2018)
120
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2022
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
  • 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 HIV and hepatitis A and B, history of serious infections and active latent tuberculosis (TB)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ann Neale 1-833-477-6700 clinicaltrials@principiabio.com
Contact: Abby Kennedy 1-833-477-6700 clinicaltrials@principiabio.com
Listed Location Countries  ICMJE Argentina,   Australia,   Bulgaria,   Canada,   Croatia,   France,   Germany,   Greece,   Israel,   Italy,   Poland,   Serbia,   Spain,   Taiwan,   Turkey,   Ukraine,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03762265
Other Study ID Numbers  ICMJE PRN1008-012
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Principia Biopharma Inc.
Study Sponsor  ICMJE Principia Biopharma Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Dolca Thomas, M.D. Principia Biopharma Inc.
PRS Account Principia Biopharma Inc.
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP