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

• Sponsor: Principia Biopharma, a Sanofi Company (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT03395210
• Collaborator: (none)
• Enrollment: 85
• Locations: 30
• Arm: 1
• Anticipated Duration (Months): 71.9
• Patients Per Site: 2.8
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Study Design

• Study Type: Interventional
• Anticipated 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: Mar 22, 2018
• Anticipated Primary Completion Date: Mar 2, 2023
• Anticipated Study Completion Date: Mar 19, 2024

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Part A and B: Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure) [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) [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) [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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 80 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigational Site Number 1087	Peoria	Illinois	United States	61615
2	Investigational Site Number 1091	Bethesda	Maryland	United States	20817
3	Investigational Site Number 1092	Boston	Massachusetts	United States	02114
4	Investigational Site Number 1099	Boston	Massachusetts	United States	02215
5	Investigational Site Number 1086	Midland	Michigan	United States	48670
6	Investigational Site Number 1097	New York	New York	United States	10021
7	Investigational Site Number 1095	Greenville	North Carolina	United States	27834
8	Investigational Site Number 1098	Seattle	Washington	United States	98195
9	Investigational Site Number 0105	Garran	Australia Capital Territory	Australia	2605
10	Investigational Site Number 0104	Concord	New South Wales	Australia	2139
11	Investigational site number 0102	Brisbane	Queensland	Australia	4102
12	Investigational Site Number 0101	Clayton	Victoria	Australia	3168
13	Investigational Site Number 0106	Parkville	Victoria	Australia	3050
14	Investigational Site Number 0103	Nedlands	Western Australia	Australia	6005
15	Investigational Site Number 0213	Pleven		Bulgaria	5800
16	Investigational Site Number 0214	Sofia		Bulgaria	1233
17	Investigational Site Number 0211	Varna		Bulgaria	9010
18	Investigational Site Number 1161	Toronto	Ontario	Canada	M5B 1W8
19	Investigational Site Number 1162	Montréal	Quebec	Canada	H4A 3J1
20	Investigational Site Number 0431	Brno		Czechia	625 00
21	Investigational Site Number 0433	Hradec Králové		Czechia	500 05
22	Investigational Site Number 0434	Ostrava		Czechia	708 52
23	Investigational Site Number 0432	Prague		Czechia	128 08
24	Investigational Site Number 0728	Den Haag	South Holland	Netherlands	2545CH
25	Investigational Site Number 0727	Rotterdam		Netherlands	3015 GD
26	Investigational Site Number 0542	Bergen		Norway	5021
27	Investigational Site Number 0541	Grålum		Norway	1714
28	Investigational Site Number 0980	London	England	United Kingdom	W12 0HS
29	Investigational Site Number 0981	Leicester		United Kingdom	LE1 5WW
30	Investigational Site Number 0983	London		United Kingdom	E1 2ES

Sponsors and Collaborators

• Principia Biopharma, a Sanofi Company

Investigators

• Study Director: Olga Bandman, MD, Principia Biopharma

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Principia Biopharma, a Sanofi Company
• ClinicalTrials.gov Identifier: NCT03395210
• Other Study ID Numbers: DFI17124; PRN1008-010
• First Posted: Jan 10, 2018
• Last Update Posted: May 12, 2022
• Last Verified: Mar 8, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Principia Biopharma, a Sanofi Company: ITP
• Additional relevant MeSH terms: Thrombocytopenia; Purpura; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombocytopenic

Study Results

No Results Posted as of May 12, 2022