Idiopathic Thrombocytopenic PurpuraPhase 3NCT00415532

Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura

A Phase 3 study testing Romiplostim in people with Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen.

Completed

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

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
What is being tested?: Romiplostim
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 234 participants
Where: No sites listed yet
Study dates: 1 Dec 2006to 11 May 2009
Phase: Phase 3
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

Subject is ≥ 18 years of age
Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines
If subject is > 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP
Subject has received at least 1 prior therapy for ITP
Subject has a platelet count < 50,000 or their platelet count falls to < 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy
Before any study-specific procedure, the appropriate written informed consent must be obtained

Who cannot join

Subject has had a splenectomy for any reason
Subject has an active malignancy
Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years
Subject has a known history of bone marrow stem cell disorder
Subject has participated in any study evaluating polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein
Subject is receiving other investigational agents or procedures

Show all 12Show fewer

Subject is currently enrolled in, or has completed within the last 30 days, another investigational device or drug study
Subject is pregnant or breast feeding
Subject is not using adequate contraceptive precautions
Subject has known sensitivity to any recombinant E. coli-derived product
Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative
Subject has any kind of disorder that compromises the ability of the subject to comply with study procedures

About this study

Official title: A Randomized, Controlled, Open-label Study Evaluating the Efficacy and Tolerability of AMG 531 Versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects With Immune (Idiopathic) Thrombocytopenic Purpura

This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety Follow-up. Patients randomized to romiplostim must complete the taper or discontinuation of medical SOC for ITP as soon as medically feasible after the initiation of romiplostim. After the completion or discontinuation of the study treatment period, any participant who does not transfer in to another romiplostim study will complete a 6-month Safety Follow-up period.

Conditions: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
Being tested: Medical Standard of Care for ITP, Romiplostim
Keywords: splenectomy, platelet, AMG 531, thrombopoietin, blood disorder, bleeding disorder, immune thrombocytopenic purpura, idiopathic thrombocytopenic purpura, immune (idiopathic) thrombocytopenic purpura, TPO, thrombopoietic protein
Medical subject headings: Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia, Purpura, Thrombocytopenic, Jacobs syndrome, Hematologic Diseases, Hemostatic Disorders

What participants receive

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

Medical Standard of Care for ITPDrug
RomiplostimBiological
Also called AMG 531

Study groups in NCT00415532
Study group	Role	What they receive
Romiplostim	Experimental	Romiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks. Receives: Biological: Romiplostim
Standard of Care	Other	Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks. Receives: Drug: Medical Standard of Care for ITP

What is being measured

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

Number of Participants With Splenectomy During 52-Week Treatment Period
Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.
Measured: 52 weeks
Number of Participants With Treatment Failure During 52-Week Treatment Period
Treatment failure was defined by platelet counts ≤ 20 x 10^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.
Measured: 52 weeks

Secondary outcomes (6)

Time to Splenectomy
Time to splenectomy in days calculated from date of randomization to date of splenectomy, or censored at date of end of treatment visit if no splenectomy was done during treatment period.
Measured: 52 weeks
Percentage of Participants With Platelet Response
Platelet response was defined as platelet counts > 50 x 10^9/L, measured at each study visit (excluding those within 8 weeks of prior rescue medication use) up to the time of splenectomy or the end of initial treatment period, whichever occurred first.
Measured: Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52
Change in ITP-PAQ Physical Health Domain of Symptoms
Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of symptoms. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life.
Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
Measured: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Fatigue
Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of fatigue. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
Measured: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Bother
Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of bother. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life. Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
Measured: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Activity
Change from Baseline in the Immune Thrombocytopenic Purpura (ITP) Patient Assessment Questionnaire (PAQ) physical health domain of activity. This domain has a range of 0 to 100, with higher scores indicating a better health-related quality of life.
Model included fixed effects of baseline assessment, geographic region, treatment group, assessment week, splenectomy status and treatment group-by-assessment week interaction. Treatment group and splenectomy status are time-varying covariates.
Measured: Baseline and 52 weeks

Timeline

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

1 Dec 200611 May 2009

First posted25 Dec 2006 (estimated)
Study start1 Dec 2006
Primary completion7 Nov 2008
Study completion11 May 2009

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 (2)Papers the sponsor has linked to this study.
Other trials for Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
More trials run by AmgenThe 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 234 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00415532
Sponsor's protocol ID: 20060131
Status last verified: Nov 2022
First submitted: 21 Dec 2006
Last updated: 8 Nov 2022
Results posted: Yes, on ClinicalTrials.gov

Publications

Kuter DJ, Mathias SD, Rummel M, Mandanas R, Giagounidis AA, Wang X, Deuson RR. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012 May;87(5):558-61. doi: 10.1002/ajh.23163. Epub 2012 Mar 28.
PubMed 22460421
Kuter DJ, Rummel M, Boccia R, Macik BG, Pabinger I, Selleslag D, Rodeghiero F, Chong BH, Wang X, Berger DP. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med. 2010 Nov 11;363(20):1889-99. doi: 10.1056/NEJMoa1002625.
PubMed 21067381

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?

Official record

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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)

All Idiopathic Thrombocytopenic Purpura trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Controlled, Open-label Study Evaluating the Efficacy and Tolerability of AMG 531 Versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
ClinicalTrials.gov ID: NCT00415532
Acronym / sponsor ID: 20060131
Conditions: Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
Keywords: splenectomy, platelet, AMG 531, thrombopoietin, blood disorder, bleeding disorder, immune thrombocytopenic purpura, idiopathic thrombocytopenic purpura
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 234 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: 1 Dec 2006 (actual)
Primary completion: 7 Nov 2008 (actual)
Study completion: 11 May 2009 (actual)
First posted: 25 Dec 2006 (estimated)
Results: Posted 8 Jul 2013

Clinical summary

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

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Romiplostim	Experimental	Biological: RomiplostimRomiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks.
Standard of Care	Other	Drug: Medical Standard of Care for ITPMedical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.

Who may qualify

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

Key inclusion criteria (6 listed)

Subject is ≥ 18 years of age
Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines
If subject is > 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP
Subject has received at least 1 prior therapy for ITP
Subject has a platelet count < 50,000 or their platelet count falls to < 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy

Key exclusion criteria (12 listed)

Subject has had a splenectomy for any reason
Subject has an active malignancy
Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years
Subject has a known history of bone marrow stem cell disorder
Subject has participated in any study evaluating polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein

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
Medical Standard of Care for ITP	Drug	Standard of Care
RomiplostimAlso: AMG 531	Biological	Romiplostim

Primary outcome

Number of Participants With Splenectomy During 52-Week Treatment Period
Time frame: 52 weeks
Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.
Number of Participants With Treatment Failure During 52-Week Treatment Period
Time frame: 52 weeks
Treatment failure was defined by platelet counts ≤ 20 x 10^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.

6 secondary outcomes

Time to Splenectomy
Time frame: 52 weeks
Percentage of Participants With Platelet Response
Time frame: Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52
Change in ITP-PAQ Physical Health Domain of Symptoms
Time frame: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Fatigue
Time frame: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Bother
Time frame: Baseline and 52 weeks
Change in ITP-PAQ Physical Health Domain of Activity
Time frame: Baseline and 52 weeks

Results and safety

Results posted to the registry on 8 Jul 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Standard of Care	77	60
Romiplostim	157	142

Primary outcome: Number of Participants With Splenectomy During 52-Week Treatment Period

Time frame: 52 weeks. Number. Unit: Participants

Measure	Standard of Care n = 77	Romiplostim n = 157
Had splenectomy	28	14
Did not have splenectomy	49	143

Standard of Care vs Romiplostim: Odds Ratio (OR): 0.172 (95% CI 0.084 to 0.352), p <0.0001, Cochran-Mantel-Haenszel

Population analysed: The Full Analysis set includes all randomized patients.

Primary outcome: Number of Participants With Treatment Failure During 52-Week Treatment Period

Time frame: 52 weeks. Number. Unit: Participants

Measure	Standard of Care n = 77	Romiplostim n = 157
Had treatment failure	23	18
Did not have treatment failure	54	139

Standard of Care vs Romiplostim: Odds Ratio (OR): 0.307 (95% CI 0.154 to 0.611), p = 0.0005, Cochran-Mantel-Haenszel

Population analysed: Full analysis set. Participants who discontinued study during treatment period prior to experiencing treatment failure were considered as having had treatment failure.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: 52 week treatment period. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Standard of Care	28 of 73 (38.4%)	60 of 73 (82.2%)	Not reported
AMG 531	36 of 156 (23.1%)	136 of 156 (87.2%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 22460421Kuter DJ, Mathias SD, Rummel M, Mandanas R, Giagounidis AA, Wang X, Deuson RR. Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care. Am J Hematol. 2012 May;87(5):558-61. doi: 10.1002/ajh.23163. Epub 2012 Mar 28.
Background: PubMed 21067381Kuter DJ, Rummel M, Boccia R, Macik BG, Pabinger I, Selleslag D, Rodeghiero F, Chong BH, Wang X, Berger DP. Romiplostim or standard of care in patients with immune thrombocytopenia. N Engl J Med. 2010 Nov 11;363(20):1889-99. doi: 10.1056/NEJMoa1002625.
AmgenTrials clinical trials websiteLink provided by the sponsor.

Related trials for Idiopathic Thrombocytopenic Purpura

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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
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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.

MD study director

Amgen

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Romiplostim in people with Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen.

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 6 inclusion and 12 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 8 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.

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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 8 Jul 2013). 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 With Splenectomy During 52-Week Treatment Period (time frame: 52 weeks). The study lists 2 primary outcomes in all. 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 NCT00415532, which also lists 2 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 8 Nov 2022.