Purpura, Thrombocytopaenic, IdiopathicPhase 2NCT00424177

Repeated Exposure to Eltrombopag in Adults With Idiopathic Thrombocytopenic Purpura (REPEAT)

A Phase 2 study testing eltrombopag in people with Purpura, Thrombocytopaenic, Idiopathic, run by GlaxoSmithKline at 3 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This open-label, repeat dosing study, TRA108057, will evaluate the efficacy, safety and tolerability of eltrombopag, when administered in a repeat, cyclic dosing schedule. The study will describe the effect of repeated (3 cycles), intermittent dosing of eltrombopag on the pharmacodynamics and durability of eltrombopag… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Purpura, Thrombocytopaenic, Idiopathic
What is being tested?: eltrombopag
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 66 participants
Where: 3 sitesin 2 countries
Study dates: Mar 2007to Nov 2008
Phase: Phase 2
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

Subjects eligible for enrolment in the study must meet all of the following criteria:
Subject has signed and dated a written inform consent.
Adults (≥18 years) diagnosed with chronic ITP according to the American Society of Hematology/British Committee for Standards in Hematology guidelines, and a platelet count between ≥20 Gi/L and ≤50 Gi/L on Day 1 (or within 24 hours prior to dosing on Day 1). In addition, a peripheral blood smear should support the diagnosis of ITP with no evidence of other causes of thrombocytopenia (e.g. pseudothrombocytopenia, myelodysplasia). The physical examination should not suggest any disease which may cause thrombocytopenia other than ITP.
Subjects who have previously received one or more prior ITP therapies. Previous treatments for ITP include but are not limited to corticosteroids, immunoglobulins, azathioprine, danazol, cyclophosphamide and/or rituximab.
Subjects must have either initially responded (platelet count >100 Gi/L) to a previous ITP therapy or have had a bone marrow biopsy consistent with ITP within 3 years to rule out myelodysplastic syndromes or other causes of thrombocytopenia.
It is important to clearly differentiate the effect of eltrombopag on platelet count from the treatment effects of prior and concomitant ITP therapies. Therefore:

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Previous therapy for ITP with immunoglobulins (IVIg and anti-D) must have been completed at least 1 week prior to randomization and the platelet count must show a clear downward trend after the last treatment with immunoglobulins. Previous treatment for ITP with splenectomy, rituximab and cyclophosphamide must have been completed at least 4 weeks prior to randomization, or clearly be ineffective.
Subjects treated with concomitant ITP medication (e.g. corticosteroids or azathioprine) must be receiving a dose that has been stable for a least 4 weeks prior to randomization. Subjects treated with cyclosporine A, mycophenolate mofetil or danazol must be receiving a dose that has been stable for at least 3 months prior to randomization.
Prothrombin time and activated partial thromboplastin time must be within 80 to 120% of normal range with no history of hypercoagulable state.
A complete blood count (CBC), within the reference range (including differential not indicative of a disorder other than ITP), with the following exceptions:
Platelet count between ≥20 Gi/L and ≤50 Gi/L on Day 1 (or within 24 hours of Day 1) is required for inclusion.
Hemoglobin: Subjects with hemoglobin levels between 10g/dL (100g/L) and the lower limit of normal are eligible for inclusion, if anemia is clearly attributable to ITP (excessive blood loss).
Absolute Neutrophil Count (ANC ) >1500/mL (1.5 x 10^9/L) is required for inclusion (elevated White Blood Cells/ANC above the reference range due to steroid treatment is acceptable).
The following clinical chemistries MUST NOT exceed the normal reference range by more than 20%: creatinine, Alanine aminotransferase, Aspartate aminotransferase, total bilirubin, total albumin and alkaline phosphatase.
Subject is practicing an acceptable method of contraception (documented in chart). Female subjects (or female partners of male subjects) must either be of non-childbearing potential (hysterectomy, bilateral oophorectomy, bilateral tubal ligation or post-menopausal >1 year), or of childbearing potential and use of one of the following acceptable methods of contraception from two weeks prior to administration of study medication, throughout the study, and 28 days after completion or premature discontinuation from the study:
Complete abstinence from intercourse; Intrauterine device (IUD); Two forms of barrier contraception (diaphragm plus spermicide, and for males condom plus spermicide); Male partner is sterile prior to entry into the study and is the only partner of the female; Systemic contraceptives (combined or progesterone only).
Subject is able to understand and comply with protocol requirements and instructions and intends to complete the study as planned.

Who cannot join

A subject will NOT be eligible for inclusion in this study if any of the following criteria apply:
Any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another primary diagnosis (e.g., thrombocytopenia is secondary to another disease).
Concurrent malignant disease and/or history of cancer treatment with cytotoxic chemotherapy and/or radiotherapy.
Any prior history of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), AND ≥ two of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (e.g., Factor V Leiden, Antithrombin III deficiency, etc), or any other family history of arterial or venous thrombosis.
Pre-existing cardiovascular disease (congestive heart failure, New York Heart Association Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation), or subjects with a QTc >450 msec.
Female subjects who are nursing or pregnant (positive serum or urine b-human chorionic gonadotrophin pregnancy test) at screening or pre-dose on Day 1.

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History of alcohol/drug abuse.
Treatment with an investigational drug within 30 days or five half-lives (whichever is longer) preceding the first dose of study medication.
Subject treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or Non Steroidal Anti Inflammatory Drugs) or anti-coagulants for > 3 consecutive days within 2 weeks of the study start and until the end of the study.
History of platelet agglutination abnormality that prevents reliable measurement of platelet counts.
All subjects with secondary immune thrombocytopenia, including those with laboratory or clinical evidence of human immunodeficiency virus (HIV) infection, anti-phospholipid antibody syndrome, chronic hepatitis B infection, hepatitis C virus infection, or any evidence for active hepatitis at the time of subject screening. If a potential subject has no clinical history that would support HIV infection or hepatitis infection, no further laboratory screening is necessary; however, standard medical practice would suggest further evaluation of patients who have risk factors for these infections.
Previous participation in a clinical study with eltrombopag.
Subjects planning to have cataract surgery.
In France, a subject is neither affiliated with nor a beneficiary of a social security category.

Trial locations and contacts

3 sites in 2 countries.

Country

City, postcode or hospital

3 sites in 2 countries

Germany2 sites

GSK Investigational Site
Berlin, State of Berlin, 13353
GSK Investigational Site
Hanover, Lower Saxony, 30625

Other trials in Germany

Russia1 site

GSK Investigational Site
Moscow, 125167

Other trials in Russia

About this study

Official title: An Open-label Repeat Dosing Study of Eltrombopag Olamine (SB-497115-GR) in Adult Subjects, With Chronic Idiopathic Thrombocytopenic Purpura (ITP)

For more information or to see if you qualify, please visit: http://www.itpstudy.com/gov

Conditions: Purpura, Thrombocytopaenic, Idiopathic
Being tested: eltrombopag
Keywords: idiopathic thrombocytopenic purpura, ITP, thrombocytopenia, platelets
Medical subject headings: Purpura, Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia

What participants receive

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

eltrombopagDrug
experimental

Study groups in NCT00424177
Study group	Role	What they receive
eltrombopag	Experimental	Not described Receives: Drug: eltrombopag

What is being measured

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

Number of Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1
Complete blood count, platelet count by blood draw
Measured: Day 42 of each cycle

Secondary outcomes (6)

Number of Participants Who Responded (Platelet Count Greater Than or Equal to 50 Gi/L and at Least 2x Baseline) for at Least 80 Percent of Their On-therapy Assessments During Weeks 2-6.
CBC, platelet counts
Measured: Up to 42 days of dosing
Changes in Participants' Platelet Counts During 3 Cycles of Treatment
Changes from baseline, during on-therapy periods of a cycle, during off-therapy periods of a cycle, and within 4 weeks of permanent discontinuation of eltrombopag treatment.
Measured: Up to 1 year
Number of Participants Who Required Rescue Medication
New idiopathic thrombocytopenic purpura (ITP) medication, increase dose of a concomitant ITP medication from baseline, platelet transfusion, and/or splenectomy
Measured: Up to 3 cycles of treatment including follow-up visits following last dose of eltrombopag
Change in Participants Anti-platelet Antibody Levels From Baseline Through Follow-up
Change in participants' anti-platelet antibody levels was measured as the number of samples positive for at least 1 glycoprotein from baseline to follow-up. Serum glycoprotein-specific antigens: GPIIb/IIIa, Ib/IX, and Ia/IIa
Measured: Up to 1 year
Number of Participants With the Indicated Bleeding Signs and Symptoms Using the World Health Organization Bleeding Scale
World health Organization (WHO) Bleeding Scale Grade 0 = no bleeding, Grade 1 = petechiae, Grade 2 = mild blood loss, Grade 3 = gross blood loss, Grade 4 = debilitating blood loss.
Measured: Baseline, on-treatment visits (Weeks 1-6), off-treatment visits (Weeks 1-4)
Number of Participants With the Indicated Bleeding Signs and Symptoms Using the ITP Bleeding Score
ITP Bleeding Score: Grade 0 = no bleeding, Grade 1 = mild bleeding, Grade 2 = severe bleeding
Measured: Baseline, on-treatment visits (Weeks 1-6), off-treatment visits (Weeks 1-4)

Timeline

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

Mar 2007Nov 2008

First posted18 Jan 2007 (estimated)
Study startMar 2007
Primary completionAug 2008
Study completionNov 2008

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.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Purpura, Thrombocytopaenic, IdiopathicStudies of the same condition, open and under way.
More trials run by GlaxoSmithKlineThe 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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 66 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00424177
Sponsor's protocol ID: TRA108057
Status last verified: May 2012
First submitted: 17 Jan 2007
Last updated: 30 Jul 2013
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Bussel JB, Saleh MN, Vasey SY, Mayer B, Arning M, Stone NL. Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP). Br J Haematol. 2013 Feb;160(4):538-46. doi: 10.1111/bjh.12169. Epub 2012 Dec 24.
PubMed 23278590
This study has not been published in the scientific literature.
Tarantino MD, Fogarty P, Mayer B, Vasey SY, Brainsky A. Efficacy of eltrombopag in management of bleeding symptoms associated with chronic immune thrombocytopenia. Blood Coagul Fibrinolysis. 2013 Apr;24(3):284-96. doi: 10.1097/MBC.0b013e32835fac99.
PubMed 23492914

Study at a glance

From the registry record, last updated by the sponsor on 30 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label Repeat Dosing Study of Eltrombopag Olamine (SB-497115-GR) in Adult Subjects, With Chronic Idiopathic Thrombocytopenic Purpura (ITP)
ClinicalTrials.gov ID: NCT00424177
Acronym / sponsor ID: TRA108057
Conditions: Purpura, Thrombocytopaenic, Idiopathic
Keywords: idiopathic thrombocytopenic purpura, ITP, thrombocytopenia, platelets
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 66 participants (actual)
Lead sponsor: GlaxoSmithKlineIndustry (pharma, biotech, devices)
Study start: Mar 2007
Primary completion: Aug 2008 (actual)
Study completion: Nov 2008 (actual)
First posted: 18 Jan 2007 (estimated)
Results: Posted 11 Aug 2009

Clinical summary

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

For more information or to see if you qualify, please visit: http://www.itpstudy.com/gov

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
eltrombopag	Experimental	Drug: eltrombopag

Who may qualify

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

Key inclusion criteria (17 listed)

Subject has signed and dated a written inform consent.
Adults (≥18 years) diagnosed with chronic ITP according to the American Society of Hematology/British Committee for Standards in Hematology guidelines, and a platelet count between ≥20 Gi/L and ≤50 Gi/L on Day 1 (or within 24 hours prior to dosing on Day 1). In addition, a peripheral blood smear should support the diagnosis of ITP with no evidence of other causes of thrombocytopenia (e.g. pseudothrombocytopenia, myelodysplasia). The physical examination should not suggest any disease which may cause thrombocytopenia other than ITP.
Subjects who have previously received one or more prior ITP therapies. Previous treatments for ITP include but are not limited to corticosteroids, immunoglobulins, azathioprine, danazol, cyclophosphamide and/or rituximab.
Subjects must have either initially responded (platelet count >100 Gi/L) to a previous ITP therapy or have had a bone marrow biopsy consistent with ITP within 3 years to rule out myelodysplastic syndromes or other causes of thrombocytopenia.
It is important to clearly differentiate the effect of eltrombopag on platelet count from the treatment effects of prior and concomitant ITP therapies. Therefore:

Key exclusion criteria (14 listed)

A subject will NOT be eligible for inclusion in this study if any of the following criteria apply:
Any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another primary diagnosis (e.g., thrombocytopenia is secondary to another disease).
Concurrent malignant disease and/or history of cancer treatment with cytotoxic chemotherapy and/or radiotherapy.
Any prior history of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), AND ≥ two of the following risk factors: hormone replacement therapy, systemic contraception (containing estrogen), smoking, diabetes, hypercholesterolemia, medication for hypertension, cancer, hereditary thrombophilic disorders (e.g., Factor V Leiden, Antithrombin III deficiency, etc), or any other family history of arterial or venous thrombosis.
Pre-existing cardiovascular disease (congestive heart failure, New York Heart Association Grade III/IV), or arrhythmia known to increase the risk of thromboembolic events (e.g. atrial fibrillation), or subjects with a QTc >450 msec.

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
eltrombopagexperimental	Drug	eltrombopag

Primary outcome

Number of Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1
Time frame: Day 42 of each cycle
Complete blood count, platelet count by blood draw

6 secondary outcomes

Number of Participants Who Responded (Platelet Count Greater Than or Equal to 50 Gi/L and at Least 2x Baseline) for at Least 80 Percent of Their On-therapy Assessments During Weeks 2-6.
Time frame: Up to 42 days of dosing
Changes in Participants' Platelet Counts During 3 Cycles of Treatment
Time frame: Up to 1 year
Number of Participants Who Required Rescue Medication
Time frame: Up to 3 cycles of treatment including follow-up visits following last dose of eltrombopag
Change in Participants Anti-platelet Antibody Levels From Baseline Through Follow-up
Time frame: Up to 1 year
Number of Participants With the Indicated Bleeding Signs and Symptoms Using the World Health Organization Bleeding Scale
Time frame: Baseline, on-treatment visits (Weeks 1-6), off-treatment visits (Weeks 1-4)
Number of Participants With the Indicated Bleeding Signs and Symptoms Using the ITP Bleeding Score
Time frame: Baseline, on-treatment visits (Weeks 1-6), off-treatment visits (Weeks 1-4)

Results and safety

Results posted to the registry on 11 Aug 2009, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treatment Period	66	55

Primary outcome: Number of Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1

Time frame: Day 42 of each cycle. Number. Unit: participants

Measure	Cycle 1 n = 52	Cycle 2 n = 51	Cycle 3 n = 49
Responders	52	41	38
Non-responders	0	10	11

Cycle 2 vs Cycle 3: Proportion of subjects in Cycle 2 or 3: 0.87 (95% CI 0.74 to 0.94)

Population analysed: Primary Analysis Population: All participants who entered the study, received at least 1 dose of eltrombopag, and responded in Cycle 1

6 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cycle 1	0 of 66 (0%)	39 of 66 (59.1%)	Not reported
Cycle 2	1 of 55 (1.8%)	30 of 55 (54.5%)	Not reported
Cycle 3	0 of 51 (0%)	38 of 51 (74.5%)	Not reported
More Than 1 to 30 Days After Last Dose	1 of 66 (1.5%)	17 of 66 (25.8%)	Not reported
More Than 30 Days After Last Dose	1 of 66 (1.5%)	7 of 66 (10.6%)	Not reported
All Cycles	4 of 66 (6.1%)	55 of 66 (83.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2013.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 23278590Bussel JB, Saleh MN, Vasey SY, Mayer B, Arning M, Stone NL. Repeated short-term use of eltrombopag in patients with chronic immune thrombocytopenia (ITP). Br J Haematol. 2013 Feb;160(4):538-46. doi: 10.1111/bjh.12169. Epub 2012 Dec 24.
BackgroundThis study has not been published in the scientific literature.
Background: PubMed 23492914Tarantino MD, Fogarty P, Mayer B, Vasey SY, Brainsky A. Efficacy of eltrombopag in management of bleeding symptoms associated with chronic immune thrombocytopenia. Blood Coagul Fibrinolysis. 2013 Apr;24(3):284-96. doi: 10.1097/MBC.0b013e32835fac99.

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.

GSK Clinical Trials study director

GlaxoSmithKline

3 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing eltrombopag in people with Purpura, Thrombocytopaenic, Idiopathic, run by GlaxoSmithKline at 3 sites in 2 countries.

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 17 inclusion and 14 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: finished, as of the sponsor's last update on 30 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Germany, Russia. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 11 Aug 2009). 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 Participants Who Responded (Platelet Count >=50 Gi/L and >=2x Baseline) to Eltrombopag Treatment in Cycle 2 or Cycle 3 Given Participants Responded in Cycle 1 (time frame: Day 42 of each cycle). 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 NCT00424177, which also lists 3 linked publications.

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 30 Jul 2013; AzTrials last refreshed its copy on 8 Nov 2026.