Trial Outcomes & Findings for Multiple Rising Does Study (Subcutaneous Doses) of BI 655064 in Male and Female Patients With Chronic Primary Immune Thrombocytopenic Purpura (ITP). (NCT NCT02009761)
NCT ID: NCT02009761
Last Updated: 2024-03-15
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE1
Target enrollment
6 participants
Primary outcome timeframe
Up to 12 weeks.
Results posted on
2024-03-15
Participant Flow
This was an open label, multiple dose with dose escalation for non-responders, multiple injection study in patients with chronic primary immune thrombocytopenic purpura.
All subjects were screened for eligibility prior to participation in the trial. Subjects attended a specialist site which ensured that they (the subjects) strictly met all inclusion and none of the exclusion criteria. Subjects were not to be allocated to a treatment group if any of the entry criteria were violated.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
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.
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|---|---|
|
Overall Study
Adverse Event
|
4
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Baseline Characteristics
Multiple Rising Does Study (Subcutaneous Doses) of BI 655064 in Male and Female Patients With Chronic Primary Immune Thrombocytopenic Purpura (ITP).
Baseline characteristics by cohort
| Measure |
BI 655064 120 mg / 180 mg
n=6 Participants
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.
|
|---|---|
|
Age, Continuous
|
66.0 Years
STANDARD_DEVIATION 9.5 • n=5 Participants
|
|
Sex: Female, Male
Female
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3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 12 weeks.Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
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.
Outcome measures
| Measure |
BI 655064 120 mg / 180 mg
n=6 Participants
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.
|
|---|---|
|
Number of Patients With Response in Platelet Count
No
|
5 Participants
|
|
Number of Patients With Response in Platelet Count
Yes
|
1 Participants
|
SECONDARY outcome
Timeframe: From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
Number of participants with investigator defined drug-related adverse events (AEs) is reported.
Outcome measures
| Measure |
BI 655064 120 mg / 180 mg
n=6 Participants
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.
|
|---|---|
|
Number of Subjects With Drug-related Adverse Events
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 12 weeks.Population: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.
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.
Outcome measures
| Measure |
BI 655064 120 mg / 180 mg
n=6 Participants
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.
|
|---|---|
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Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP]
No
|
6 Participants
|
|
Number of Patients Reaching the Cut-off Point for Immune Thrombocytopenic Purpura [ITP]
Yes
|
0 Participants
|
Adverse Events
BI 655064 120 mg / 180 mg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
BI 655064 120 mg / 180 mg
n=6 participants at risk
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.
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|---|---|
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Eye disorders
Conjunctival haemorrhage
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Gastrointestinal disorders
Flatulence
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
General disorders
Hernia
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
2/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Injury, poisoning and procedural complications
Contusion
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Investigations
Platelet count decreased
|
50.0%
3/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Nervous system disorders
Headache
|
33.3%
2/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
33.3%
2/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
|
Vascular disorders
Haemorrhage
|
16.7%
1/6 • From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.
Treated Set (TS): Included all patients who received trial medication and were documented to have taken at least one dose of trial medication. The safety analysis was pre-specified to display overall, without differentiating treatments (BI 655064 180mg q1w in Weeks 5 and 6, and BI 655064 120mg q1w during the treatment period). The analysis presents the treatment emergent AEs. The on-treatment period: between drug intake until 6 weeks after the last study medication dose.
|
Additional Information
Boehringer Ingelheim, Call Center
Boehringer Ingelheim
Phone: 1800-243-0127
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER