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

Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of Open Label Study With Multiple (and for Non Responders) Escalating Subcutaneous Doses of BI 655064 Once a Week in Patients With Chronic Primary Immune Thrombocytopenic Purpura.

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

Study Overview

• Status: Terminated
• Conditions: Purpura, Thrombocytopenic, Idiopathic
• Intervention / Treatment: Drug: BI 655064

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California
  • New York
    • New York, New York, United States, 10021
      • New York Hospital, Cornell Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

Male and female chronic immune ITP patients

Exclusion criteria:

Any treatment of ITP excluded except for a stable dose of corticosteroids (up to 20 mg)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: BI 655064 120 mg / 180 mg; 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.

Drug: BI 655064; subcutaneous injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients With Response in Platelet Count	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.	Up to 12 weeks.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Subjects With Drug-related Adverse Events	Number of participants with investigator defined drug-related adverse events (AEs) is reported.	From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP]	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.	Up to 12 weeks.

Collaborators and Investigators

• Sponsor: Boehringer Ingelheim
• Investigators: Study Chair: Boehringer Ingelheim, Boehringer Ingelheim

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): December 18, 2013
• Primary Completion (Actual): April 26, 2016
• Study Completion (Actual): April 26, 2016

Study Registration Dates

• First Submitted: December 9, 2013
• First Submitted That Met QC Criteria: December 9, 2013
• First Posted (Estimated): December 12, 2013

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: August 29, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Cytopenia; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: 1293.7

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD Plan Description

Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization).

For more details refer to:

https://www.mystudywindow.com/msw/datatransparency