Purpura, Thrombocytopenic, IdiopathicPhase 1NCT02009761

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

A Phase 1 study testing BI 655064 in people with Purpura, Thrombocytopenic, Idiopathic, run by Boehringer Ingelheim at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

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

Who is it for?: Ages 18 years and older
Conditions the study is for: Purpura, Thrombocytopenic, Idiopathic
What is being tested?: BI 655064
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 6 participants
Where: 2 sitesUnited States
Study dates: 18 Dec 2013to 26 Apr 2016
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male and female chronic immune ITP patients

Who cannot join

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

Trial locations and contacts

2 sites in 1 country.

United States2 sites

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

Other trials in United States

About this study

Official title: 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.

Conditions: Purpura, Thrombocytopenic, Idiopathic
Being tested: BI 655064
Medical subject headings: Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BI 655064Drug
subcutaneous injection

Study groups in NCT02009761
Study group	Role	What they receive
BI 655064 120 mg / 180 mg	Experimental	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. Receives: Drug: BI 655064

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Up to 12 weeks.

Secondary outcomes (2)

Number of Subjects With Drug-related Adverse Events
Number of participants with investigator defined drug-related adverse events (AEs) is reported.
Measured: 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.
Measured: Up to 12 weeks.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Dec 201326 Apr 2016

First posted12 Dec 2013 (estimated)
Study start18 Dec 2013
Primary completion26 Apr 2016
Study completion26 Apr 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Purpura, Thrombocytopenic, IdiopathicStudies of the same condition, open and under way.
More trials run by Boehringer IngelheimThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02009761
Sponsor's protocol ID: 1293.7
Status last verified: Aug 2023
First submitted: 9 Dec 2013
Last updated: 15 Mar 2024
Results posted: Yes, on ClinicalTrials.gov
Individual data sharing: No: 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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
Not yet recruitingPhase 1
Phase 1 Dose Escalation Study of RXIM002 in Autoimmune Cytopenias
Immune Thrombocytopenia (ITP)
Not yet recruitingObservational study
Study of Thyroid Dysfunction in Immune Thrombocytopenic Purpura Patients
Thyroid Dysfunction, Immune Thrombocytopenic Purpura
RecruitingPhase 3
A Study Investigating Intravenous Human Normal Immunoglobulin 10% in Adults With Chronic Immune Thrombocytopenia (ITP)
Chronic Primary Immune Thrombocytopenia (ITP)

All Purpura, Thrombocytopenic, Idiopathic trials

Study at a glance

From the registry record, last updated by the sponsor on 15 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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.
ClinicalTrials.gov ID: NCT02009761
Acronym / sponsor ID: 1293.7
Conditions: Purpura, Thrombocytopenic, Idiopathic
Phase: Phase 1
Study type: Interventional
Recruitment status: Terminated
Enrolment: 6 participants (actual)
Lead sponsor: Boehringer IngelheimIndustry (pharma, biotech, devices)
Study start: 18 Dec 2013 (actual)
Primary completion: 26 Apr 2016 (actual)
Study completion: 26 Apr 2016 (actual)
First posted: 12 Dec 2013 (estimated)
Results: Posted 15 Mar 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
BI 655064 120 mg / 180 mg	Experimental	Drug: BI 655064The 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.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

Male and female chronic immune ITP patients

Key exclusion criteria (1 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BI 655064subcutaneous injection	Drug	BI 655064 120 mg / 180 mg

Primary outcome

Number of Patients With Response in Platelet Count
Time frame: 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.

2 secondary outcomes

Number of Subjects With Drug-related Adverse Events
Time frame: 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]
Time frame: Up to 12 weeks.

Results and safety

Results posted to the registry on 15 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BI 655064 120 mg / 180 mg	6	2

Primary outcome: Number of Patients With Response in Platelet Count

Time frame: Up to 12 weeks.. Count of participants. Unit: Participants

Measure	BI 655064 120 mg / 180 mg n = 6
No	5
Yes	1

Population analysed: Treated Set (TS): Included all patients who were dispensed trial medication and were documented to have taken at least one dose of trial medication.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first drug administration until end of the Residual Effect Period (REP), up to 6 weeks.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
BI 655064 120 mg / 180 mg	0 of 6 (0%)	6 of 6 (100%)	0 of 6

Limitations and caveats (from the sponsor): Trial enrollment was permanently stopped due to low patient enrollment. At the time of termination of enrollment, only 6 patients were entered (only 2 of them completed treatment).

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related InfoLink provided by the sponsor.
Individual participant dataSharing planned: No. 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

Related trials for Purpura, Thrombocytopenic, Idiopathic

RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
Not yet recruitingPhase 1
Phase 1 Dose Escalation Study of RXIM002 in Autoimmune Cytopenias
Immune Thrombocytopenia (ITP)
Not yet recruitingObservational study
Study of Thyroid Dysfunction in Immune Thrombocytopenic Purpura Patients
Thyroid Dysfunction, Immune Thrombocytopenic Purpura
RecruitingPhase 3
A Study Investigating Intravenous Human Normal Immunoglobulin 10% in Adults With Chronic Immune Thrombocytopenia (ITP)
Chronic Primary Immune Thrombocytopenia (ITP)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Boehringer Ingelheim study chair

Boehringer Ingelheim

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BI 655064 in people with Purpura, Thrombocytopenic, Idiopathic, run by Boehringer Ingelheim at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 15 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Patients With Response in Platelet Count (time frame: Up to 12 weeks.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02009761.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Mar 2024.