Ph3 Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

Ph3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study With Open Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)

Sponsors

Lead Sponsor: Principia Biopharma Inc.

Source Principia Biopharma Inc.
Brief Summary

This is a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL on two counts at least 5 days apart in the 14 days before treatment begins. Patients will receive rilzabrutinib or placebo 400mg twice daily.

Detailed Description

This a randomized, double-blind study of rilzabrutinib in approximately 194 patients, aged 12 and older, with persistent or chronic ITP. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28 week open label period. Patients who respond to rilzabrutinib may enter a long term-extension.

After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Overall Status Not yet recruiting
Start Date November 2020
Completion Date June 2025
Primary Completion Date October 2023
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Sustained Increase in Platelet Counts (Efficacy Outcome Measure) 24 weeks
Secondary Outcome
Measure Time Frame
Incidence of Treatment Emergent Adverse Events (Safety Outcome Measure) 52 weeks of treatment, long term extension and 4 weeks of follow up post last dose
Enrollment 194
Condition
Intervention

Intervention Type: Drug

Intervention Name: Rilzabrutinib

Description: 400mg Caplet

Arm Group Label: Rilzabrutinib

Other Name: PRN1008

Intervention Type: Drug

Intervention Name: Placebo

Description: 400mg Caplet

Arm Group Label: Placebo

Other Name: PRN1008 Placebo

Eligibility

Criteria:

Inclusion Criteria:

- Male or female patients, aged 12 and older

- Primary ITP of duration greater than 6 months in ages 12-17

- Primary ITP of duration greater than 3 months in ages 18 and older

Exclusion Criteria:

- Pregnant or lactating woman

- Current drug or alcohol abuse

- History of solid organ transplant

- Secondary ITP

Gender: All

Minimum Age: 12 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Olga Bandman, MD Study Director Principia Biopharma
Overall Contact

Last Name: Regan Burns

Phone: 1-833-477-6700

Email: [email protected]

Location
Facility: Central Recruiting (Principia Biopharma)
Location Countries

United States

Verification Date

September 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Rilzabrutinib

Type: Experimental

Description: Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label

Label: Placebo

Type: Placebo Comparator

Description: Patients receive matching placebo 400mg orally twice daily for up to 24 weeks.

Acronym LUNA3
Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Triple (Participant, Care Provider, Investigator)

Masking Description: Double-Blind

Source: ClinicalTrials.gov