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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Principia Biopharma Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

20 Mar 2018 → 12 Dec 2025

Decision date (initial)

2024-03-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Principia Biopharma, a Sanofi Company

External identifiers

EU CT number

2023-509397-39-00

EudraCT number

2017-004012-19

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacodynamic, Safety, Pharmacokinetic, Efficacy, Therapy

Part A: To characterize the safety and tolerability of up to four dose levels of rilzabrutinib in patients with ITP
Part A: To explore the clinical activity of up to four dose levels of rilzabrutinib in relapsed/refractory patients with ITP
Part B: To characterize the safety and tolerability of 400 mg BID dose of rilzabrutinib in patients with ITP
Part B: To further explore the clinical activity and durability of response of the selected dose of 400 mg BID of rilzabrutinib in patients with ITP who have relapsed or have an insufficient response to prior therapies
Part B: To evaluate the platelet response to rilzabrutinib therapy in the first 8 weeks of active treatment for the achievement of the primary endpoint

Secondary objectives 1

1. Part A and B: To characterize the pharmacokinetics of rilzabrutinib in patients with ITP

Conditions and MedDRA coding

Immune Thrombocytopenia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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

Exclusion criteria 4

1. Pregnant or lactating women
2. Current drug or alcohol abuse
3. History of solid organ transplant
4. Positive screening for HIV, hepatitis B, or hepatitis C

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Part A and B: Incidence of treatment-emergent adverse events (TEAEs)
2. Part A: Consecutive Increased Platelet Counts
3. Part B: Sustained Increase in Platelet Counts

Secondary endpoints 15

1. Part A: Percent of weeks with platelet counts ≥ 50,000/μL by dose level and overall
2. Part A: Proportion of patients with 4 out of the final 8 platelet counts ≥ 50,000/μL across all dose levels
3. 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
4. Part A: Number of weeks with platelet counts ≥50,000/μL across all dose levels
5. Part A: Number of weeks with platelet counts ≥30,000/μL across all dose levels
6. Part A: Time to first platelet count ≥50,000/μL across all dose levels
7. 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)
8. Part B: Proportion of all treated patients able to achieve 2 or more consecutive platelet counts, separated by at least 5 days, of ≥50,000/μL AND an increase of platelet count of ≥20,000/μL from baseline without use of rescue medication in the 4 weeks prior to the latest elevated platelet count
9. 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)
10. Part B: Proportion of patients receiving rescue medication
11. Part B: Change from baseline in ITP Bleeding Assessment Tool (ITP-BAT)
12. Part A: Proportion of patients receiving rescue medication at each dosing level and overall
13. Part A: Proportion of patients with a Grade 2 or higher bleeding event at each dosing level and overall
14. Part A: Bleeding scale (ITP-BAT scale) at the end of treatment period for each dosing level
15. Part A and B: Plasma PK parameters of rilzabrutinib

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Principia Biopharma Inc.

Sponsor organisation

Principia Biopharma Inc.

Address

100 Morris Street

City

Morristown

Postcode

07960-4563

Country

United States

Scientific contact point

Organisation

Principia Biopharma Inc.

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Principia Biopharma Inc.

Contact name

Clinical Sciences and Operations

Third parties 7

Locations

3 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 42 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications