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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03395210




Registration number
NCT03395210
Ethics application status
Date submitted
22/12/2017
Date registered
10/01/2018

Titles & IDs
Public title
A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
Scientific 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
Secondary ID [1] 0 0
PRN1008-010
Secondary ID [2] 0 0
DFI17124
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Thrombocytopenia 0 0
Immune Thrombocytopenic Purpura 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Rilzabrutinib

Experimental: Rilzabrutinib (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


Treatment: Drugs: Rilzabrutinib
BTK inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A and B: Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure)
Timepoint [1] 0 0
24 weeks of treatment, long term extension and 4 weeks of follow up post last dose]
Primary outcome [2] 0 0
Part A: Consecutive Increased Platelet Counts (Efficacy Outcome Measure)
Timepoint [2] 0 0
24 weeks
Primary outcome [3] 0 0
Part B: Sustained Increase in Platelet Counts (Efficacy Outcome Measure)
Timepoint [3] 0 0
24 weeks
Secondary outcome [1] 0 0
Part A: Percent of weeks with platelet counts = 50,000/µL by dose level and overall
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Part A: Proportion of patients with 4 out of the final 8 platelet counts = 50,000/µL across all dose levels
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Part A: Change from baseline to the average of the post Day 1 platelet counts by dose level and overall for patients who had >4 weeks of study drug on that given dose level
Timepoint [3] 0 0
24 weeks
Secondary outcome [4] 0 0
Part A: Number of weeks with platelet counts =50,000/µL across all dose levels.
Timepoint [4] 0 0
24 weeks
Secondary outcome [5] 0 0
Part A: Number of weeks with platelet counts =30,000/µL across all dose levels
Timepoint [5] 0 0
24 weeks
Secondary outcome [6] 0 0
Part A: Time to first platelet count =50,000/µL across all dose levels
Timepoint [6] 0 0
24 weeks
Secondary outcome [7] 0 0
Part B: Number of weeks with platelet count =50,000/µL OR =30,000/µL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given)
Timepoint [7] 0 0
24 weeks
Secondary outcome [8] 0 0
Part B: Proportion of all treated patients able to achieve=2 consecutive platelet counts, separated by=5days, of=50,000/µL AND increase of platelet count of=20,000/µL from baseline w/o rescue medication use in 4wks prior to latest elevated platelet count
Timepoint [8] 0 0
24 weeks
Secondary outcome [9] 0 0
Part B: Number of weeks with platelet counts =30,000/µL and doubling from baseline over the 24-week treatment period (platelet counts will be censored for 4 weeks after the use of rescue medication, if given)
Timepoint [9] 0 0
24 weeks
Secondary outcome [10] 0 0
Part B: Proportion of patients receiving rescue medication
Timepoint [10] 0 0
24 weeks
Secondary outcome [11] 0 0
Part B: Change from baseline in ITP Bleeding Assessment Tool (ITP-BAT)
Timepoint [11] 0 0
24 weeks
Secondary outcome [12] 0 0
Part A: Proportion of patients receiving rescue medication at each dosing level and overall
Timepoint [12] 0 0
24 weeks
Secondary outcome [13] 0 0
Part A: Proportion of patients with a Grade 2 or higher bleeding event at each dosing level and overall
Timepoint [13] 0 0
24 weeks
Secondary outcome [14] 0 0
Part A: Bleeding scale (ITP-BAT scale) at the end of treatment period for each dosing level
Timepoint [14] 0 0
24 weeks
Secondary outcome [15] 0 0
Part A and B: Plasma PK parameters of rilzabrutinib
Timepoint [15] 0 0
Up to long term extension and 4 weeks of follow up post last dose

Eligibility
Key inclusion criteria
* Male or female patients, aged 18 to 80 years old
* Immune-related ITP (both primary and secondary)
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/03/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2025
Actual
Sample size
Target
Accrual to date
Final
81
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 105 - Canberra
Recruitment hospital [2] 0 0
Investigational Site Number : 104 - Sydney
Recruitment hospital [3] 0 0
Investigational Site Number : 102 - Woolloongabba
Recruitment hospital [4] 0 0
Investigational Site Number : 101 - Clayton
Recruitment hospital [5] 0 0
Investigational Site Number : 106 - Parkville
Recruitment hospital [6] 0 0
Investigational Site Number : 103 - Perth
Recruitment postcode(s) [1] 0 0
2605 - Canberra
Recruitment postcode(s) [2] 0 0
2139 - Sydney
Recruitment postcode(s) [3] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [4] 0 0
3168 - Clayton
Recruitment postcode(s) [5] 0 0
3050 - Parkville
Recruitment postcode(s) [6] 0 0
6005 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Washington
Country [8] 0 0
Bulgaria
State/province [8] 0 0
Pleven
Country [9] 0 0
Bulgaria
State/province [9] 0 0
Sofia
Country [10] 0 0
Bulgaria
State/province [10] 0 0
Varna
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Canada
State/province [12] 0 0
Quebec
Country [13] 0 0
Czechia
State/province [13] 0 0
Brno
Country [14] 0 0
Czechia
State/province [14] 0 0
Hradec Kralove
Country [15] 0 0
Czechia
State/province [15] 0 0
Ostrava - Poruba
Country [16] 0 0
Czechia
State/province [16] 0 0
Praha 2
Country [17] 0 0
Netherlands
State/province [17] 0 0
Rotterdam
Country [18] 0 0
Netherlands
State/province [18] 0 0
s-Gravenhage
Country [19] 0 0
Norway
State/province [19] 0 0
Bergen
Country [20] 0 0
Norway
State/province [20] 0 0
Gralum
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Leicestershire
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London, City Of
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Birmingham

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Principia Biopharma, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Olga Bandman, MD
Address 0 0
Principia Biopharma
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03395210