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A Study of PRN1008 in Adult Patients With Immune Thrombocytopenia (ITP)

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: NCT03395210
Recruitment Status : Recruiting
First Posted : January 10, 2018
Last Update Posted : September 16, 2020
Sponsor:
Information provided by (Responsible Party):
Principia Biopharma Inc.

Tracking Information
First Submitted Date  ICMJE December 22, 2017
First Posted Date  ICMJE January 10, 2018
Last Update Posted Date September 16, 2020
Actual Study Start Date  ICMJE March 22, 2018
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 12, 2020)
  • Part A and B: Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure) [ Time Frame: 24 weeks of treatment, long term extension and 4 weeks of follow up post last dose] ]
    The incidence, severity and relationship of TEAEs during the treatment period and at the 4-week follow-up from last dose received.
  • Part A: Consecutive Increased Platelet Counts (Efficacy Outcome Measure) [ Time Frame: 24 weeks ]
    Proportion of subjects able to achieve two or more consecutive platelet counts of ≥ 50,000/μL AND an increase of platelet count of ≥20,000/μL from baseline, without the use of rescue medication
  • Part B: Sustained Increase in Platelet Counts (Efficacy Outcome Measure) [ Time Frame: 24 weeks ]
    Proportion of patients able to achieve platelet counts ≥50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication
Original Primary Outcome Measures  ICMJE
 (submitted: January 3, 2018)
  • Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure) [ Time Frame: 12 weeks of treatment and 12 weeks of follow up ]
    The incidence, severity and relationship of TEAEs during the treatment period and non-TEAEs in the 12-week follow-up period will also be assessed and examined for possible relationship to the PRN1008 treatment.
  • Consecutive Increased Platelet Counts (Efficacy Outcome Measure) [ Time Frame: 12 Weeks ]
    Proportion of subjects able to achieve two or more consecutive platelet counts of ≥ 50,000/μL AND an increase of platelet count of ≥20,000/μL from baseline, without the use of rescue medication
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
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 Adult Patients With Immune Thrombocytopenia (ITP)
Official Title  ICMJE An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients With Relapsed Immune Thrombocytopenia
Brief Summary This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study has been completed. Part B treatment dose is 400 mg twice daily.
Detailed Description

This is a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in approximately 60 patients in Part A and approximately 25 patients in Part B.

Part A enrolls patients with ITP who are refractory or relapsed with no available and approved therapeutic options. Eligible patients have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The active treatment period is 24 weeks and the post-treatment follow-up period is 4 weeks. In the dose-finding part of the study, each patient enrolled in the study is allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they do not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who respond to PRN1008 per protocol may enter a long term-extension.

Part B of the study will include approximately 25 patients with ITP who have relapsed or have an insufficient response to prior therapies. Eligible patients will have a platelet count <30,000/µL on two occasions no less than 7 days apart, within 15 days before treatment begins and a platelet count of ≤35,000/µL on Study Day 1 (SD1). The study consists of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there will be a 4-week safety follow-up period.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Immune Thrombocytopenia
  • Immune Thrombocytopenic Purpura
Intervention  ICMJE Drug: PRN1008
BTK inhibitor
Study Arms  ICMJE Experimental: PRN1008 Daily

Part A approximately 60 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension.

Part B approximately 25 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension

Intervention: Drug: PRN1008
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: August 12, 2020)
85
Original Estimated Enrollment  ICMJE
 (submitted: January 3, 2018)
24
Estimated Study Completion Date  ICMJE September 2023
Estimated Primary Completion Date September 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female patients, aged 18 to 80 years old
  • Immune-related ITP (both primary and secondary)

Exclusion Criteria:

  • Pregnant or lactating women
  • Current drug or alcohol abuse
  • History of solid organ transplant
  • Positive screening for HIV, hepatitis B, or hepatitis C
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: Quynh Tran 1-833-477-6700 clinicaltrials@principiabio.com
Listed Location Countries  ICMJE Australia,   Bulgaria,   Canada,   Czechia,   Netherlands,   Norway,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03395210
Other Study ID Numbers  ICMJE PRN1008-010
Has Data Monitoring Committee Yes
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 Not Provided
Responsible Party Principia Biopharma Inc.
Study Sponsor  ICMJE Principia Biopharma Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Olga Bandman, MD Principia Biopharma
PRS Account Principia Biopharma Inc.
Verification Date September 2020

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