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Trial record 1 of 6 for:    PRN1008
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A Study of PRN1008 in Adult Patients With Immune Thrombocytopenia (ITP)

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ClinicalTrials.gov Identifier: NCT03395210
Recruitment Status : Recruiting
First Posted : January 10, 2018
Last Update Posted : March 2, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Principia Biopharma, a Sanofi Company )

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.

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia Immune Thrombocytopenic Purpura Drug: PRN1008 Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 85 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 22, 2018
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2023


Arm Intervention/treatment
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

Drug: PRN1008
BTK inhibitor




Primary Outcome Measures :
  1. 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.

  2. 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

  3. 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



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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 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

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): NCT03395210


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext option 6 Contact-US@sanofi.com

Locations
Show Show 31 study locations
Sponsors and Collaborators
Principia Biopharma, a Sanofi Company
Investigators
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Study Director: Olga Bandman, MD Principia Biopharma
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Responsible Party: Principia Biopharma, a Sanofi Company
ClinicalTrials.gov Identifier: NCT03395210    
Other Study ID Numbers: DFI17124
PRN1008-010 ( Other Identifier: Principia Biopharma )
First Posted: January 10, 2018    Key Record Dates
Last Update Posted: March 2, 2021
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sanofi ( Principia Biopharma, a Sanofi Company ):
ITP
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Skin Manifestations
Thrombotic Microangiopathies
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases