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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03395210




Registration number
NCT03395210
Ethics application status
Date submitted
22/12/2017
Date registered
10/01/2018
Date last updated
29/05/2024

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
N/A
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
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03395210
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03395210