Multiple Rising Does Study (Subcutaneous Doses) of BI 655064 in Male and Female Patients With Chronic Primary Immune Thrombocytopenic Purpura (ITP).

Phase 1

Terminated

• Conditions: Purpura, Thrombocytopenic, Idiopathic
• Interventions: Drug: BI 655064

• Registration Number: NCT02009761
• Lead Sponsor: Boehringer Ingelheim

Brief Summary

BI 655064 will be administered subcutaneously once weekly in patients with immune thrombocytopenic purpura (ITP) for up to 12 weeks.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-12-09
• Study First Submitted QC: 2013-12-09
• Study First Posted: 2013-12-12
• Study Start: 2013-12-18
• Primary Completion: 2016-04-26
• Study Completion: 2016-04-26
• Results First Submitted: 2022-09-26
• Status Verified: 2023-08
• Results First Submitted QC: 2023-08-29
• Last Update Submitted: 2023-08-29
• Results First Posted: 2024-03-15
• Last Update Posted: 2024-03-15

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BI 655064 120 mg / 180 mg	BI 655064	The patients were administered 120 mg BI 655064 solution for subcutaneous injection q1w (once a week) subcutaneously for 4 weeks. Patients who showed an increase in platelet count above or equal to 100 x 10\^9/L continued treatment with 120 mg BI 655064 solution for subcutaneous injection q1w for additional 8 weeks, followed by 12 weeks of follow-up. Patients whose platelet count stayed below 100 x 10\^9/L continued treatment for 2 weeks with 180 mg BI 655064 solution for subcutaneous injection q1w followed by 120 mg BI 655064 solution for subcutaneous injection q1w for additional 6 weeks, followed by 12 weeks of follow-up.

Primary Outcome Measures

Name	Time	Method
Number of Patients With Response in Platelet Count	Up to 12 weeks.	This outcome measure presents number of patients with a response in platelet count. Response was defined as: \[A\] An increase in platelet count by greater than 20 x 10\^9/L from baseline at any time-point between Week 1 and Week 12, and \[B\] Platelet count above 50 x 10\^9/L at any time point between Week 1 and Week 12 with no rescue therapy.; Baseline was defined as the platelet count at Visit 2 before administration of BI 655064. In case the patient fulfilled only one of the conditions, he/she was considered a non-responder.

Secondary Outcome Measures

Name	Time	Method
Number of Subjects With Drug-related Adverse Events	From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.	Number of participants with investigator defined drug-related adverse events (AEs) is reported.
Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP]	Up to 12 weeks.	This outcome measure presents the number of patients reaching the cut-off point for ITP, which was defined as: \[A\] Platelet count ≥ 100 x 10\^9/L at any time point between Week 1 and Week 12.

Trial Locations

Locations (2)

University of Southern California; 🇺🇸 Los Angeles, California, United States

New York Hospital, Cornell Medical Center; 🇺🇸 New York, New York, United States