Thrombocytopenic PurpuraPhase 1/2NCT00117143

Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

A Phase 1/2 study testing Romiplostim in people with Thrombocytopenic Purpura, run by Amgen.

Completed

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

Trial summary

The purpose of this study is to assess the safety and tolerability of AMG 531 (romiplostim), a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Thrombocytopenic Purpura
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: 16 participants
Where: No sites listed yet
Study dates: 2 Dec 2002to 19 Jul 2004
Phase: Phase 1/2
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

Greater than or equal to 3 months history of ITP, regardless of splenectomy status, and completion of at least 1 prior treatment for ITP
2 of 3 pretreatment platelet counts that were less than 30 x 10^9/L (if not currently on ITP therapy) or less than 50 x 10^9/L (if currently receiving corticosteroids for ITP therapy)
Ability to give informed consent

Who cannot join

Known history of arterial thrombosis, active malignancy, or bone marrow stem cell disorder

About this study

Official title: An Open-label, Unit Dose-finding Study Evaluating the Safety and Efficacy of Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

Conditions: Thrombocytopenic Purpura
Being tested: Romiplostim
Keywords: Immune Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura, ITP, Thrombocytopenia
Medical subject headings: Purpura, Thrombocytopenic, Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia

What participants receive

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

RomiplostimDrug
Also called AMG 531, NPLATE
Administered subcutaneously on day 1 and on day 15 or 22 if the platelet count was ≤ 50 x 10⁹/L and not rising, peak platelet count was ≤ 450 x 10⁹/L and no serious adverse events related to treatment were observed.

Study groups in NCT00117143
Study group	Role	What they receive
Romiplostim	Experimental	Participants will receive a maximum of 2 administrations of romiplostim by subcutaneous injection, the first on day 1 of the study and the second on day 15 or 22 depending on the participant's platelet count. Romiplostim doses to be tested were 30, 100, 300, and 500 μg. Receives: Drug: Romiplostim

What is being measured

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

Number of Participants With Adverse Events
Measured: From first dose through 8 weeks after last dose of study drug (11 weeks)
Number of Participants With Positive Anti-Romiplostim Antibodies
The presence or development of antibodies to romiplostim and endogenous thrombopoietin was assessed using a neutralizing bioassay. Antibody analyses were conducted on study days 29 and at the end-of-study visit (day 78). The number of participants with positive antibody binding at any time during the study is reported.
Measured: Days 29 and 78

Secondary outcomes (7)

Number of Participants Who Achieved a Targeted Therapeutic Platelet Response
Targeted therapeutic platelet response was defined as a (single) platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L.
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
Measured: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L From Baseline
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
Measured: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Number of Participants With Peak Platelet Counts of ≥ 100 x 10⁹ Cells/L
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
Measured: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
Number of Participants With Peak Platelet Counts of ≥ 450 x 10⁹ Cells/L
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
Measured: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
Change From Baseline to Peak Platelet Level
Platelet count data after the use of rescue medication were not included.
Measured: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Time to Peak Platelet Count
Time from the date of study drug administration (day 1, 15 or 22) to the day of peak platelet count after each dose. Platelet count data after the use of rescue medication were not included.
Measured: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78
Duration Within the Targeted Therapeutic Range
Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L.
Platelet count data after the use of rescue medication were not included.
Measured: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78

Timeline

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

2 Dec 200219 Jul 2004

First posted4 Jul 2005 (estimated)
Study start2 Dec 2002
Primary completion19 Jul 2004
Study completion19 Jul 2004

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 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Enrolment: 16 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00117143
Sponsor's protocol ID: 20010218
Status last verified: Nov 2019
First submitted: 30 Jun 2005
Last updated: 26 Jun 2023
Results posted: Yes, on ClinicalTrials.gov

Publications

Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14.
PubMed 28411254
Newland A, Caulier MT, Kappers-Klunne M, Schipperus MR, Lefrere F, Zwaginga JJ, Christal J, Chen CF, Nichol JL. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol. 2006 Nov;135(4):547-53. doi: 10.1111/j.1365-2141.2006.06339.x.
PubMed 17061981

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

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
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 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

All Thrombocytopenic Purpura trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-label, Unit Dose-finding Study Evaluating the Safety and Efficacy of Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
ClinicalTrials.gov ID: NCT00117143
Acronym / sponsor ID: 20010218
Conditions: Thrombocytopenic Purpura
Keywords: Immune Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura, ITP, Thrombocytopenia
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 16 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: 2 Dec 2002 (actual)
Primary completion: 19 Jul 2004 (actual)
Study completion: 19 Jul 2004 (actual)
First posted: 4 Jul 2005 (estimated)
Results: Posted 20 Dec 2019

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: Not applicable
Intervention model: Sequential
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Romiplostim	Experimental	Drug: RomiplostimParticipants will receive a maximum of 2 administrations of romiplostim by subcutaneous injection, the first on day 1 of the study and the second on day 15 or 22 depending on the participant's platelet count. Romiplostim doses to be tested were 30, 100, 300, and 500 μg.

Who may qualify

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

Key inclusion criteria (3 listed)

Greater than or equal to 3 months history of ITP, regardless of splenectomy status, and completion of at least 1 prior treatment for ITP
2 of 3 pretreatment platelet counts that were less than 30 x 10^9/L (if not currently on ITP therapy) or less than 50 x 10^9/L (if currently receiving corticosteroids for ITP therapy)
Ability to give informed consent

Key exclusion criteria (1 listed)

Known history of arterial thrombosis, active malignancy, or bone marrow stem cell disorder

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
RomiplostimAlso: AMG 531, NPLATEAdministered subcutaneously on day 1 and on day 15 or 22 if the platelet count was ≤ 50 x 10⁹/L and not rising, peak platelet count was ≤ 450 x 10⁹/L and no serious adverse events related to treatment were observed.	Drug	Romiplostim

Primary outcome

Number of Participants With Adverse Events
Time frame: From first dose through 8 weeks after last dose of study drug (11 weeks)
Number of Participants With Positive Anti-Romiplostim Antibodies
Time frame: Days 29 and 78
The presence or development of antibodies to romiplostim and endogenous thrombopoietin was assessed using a neutralizing bioassay. Antibody analyses were conducted on study days 29 and at the end-of-study visit (day 78). The number of participants with positive antibody binding at any time during the study is reported.

7 secondary outcomes

Number of Participants Who Achieved a Targeted Therapeutic Platelet Response
Time frame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L From Baseline
Time frame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Number of Participants With Peak Platelet Counts of ≥ 100 x 10⁹ Cells/L
Time frame: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
Number of Participants With Peak Platelet Counts of ≥ 450 x 10⁹ Cells/L
Time frame: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
Change From Baseline to Peak Platelet Level
Time frame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
Time to Peak Platelet Count
Time frame: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78
Duration Within the Targeted Therapeutic Range
Time frame: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78

Results and safety

Results posted to the registry on 20 Dec 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Romiplostim 30 µg	4	4
Romiplostim 100 µg	4	4
Romiplostim 300 µg	7	7
Romiplostim 500 µg	1	1

Primary outcome: Number of Participants With Adverse Events

Time frame: From first dose through 8 weeks after last dose of study drug (11 weeks). Count of participants. Unit: Participants

Measure	Romiplostim 30 µg n = 4	Romiplostim 100 µg n = 4	Romiplostim 300 µg n = 7	Romiplostim 500 µg n = 1
All adverse events	4	4	7	1
Serious adverse events	0	0	3	1
Treatment-related adverse events	1	3	3	1
Serious treatment-related adverse events	0	0	1	1
Discontinuations due to adverse events	0	0	0	0
Deaths	0	0	0	0

Population analysed: Participants who received at least 1 dose of romiplostim

Primary outcome: Number of Participants With Positive Anti-Romiplostim Antibodies

Time frame: Days 29 and 78. Count of participants. Unit: Participants

Measure	Romiplostim 30 µg n = 4	Romiplostim 100 µg n = 4	Romiplostim 300 µg n = 7	Romiplostim 500 µg n = 1
Anti-romiplostim antibodies	0	0	0	0
Anti-thrombopoietin antibodies	0	0	0	0

Population analysed: Participants who received at least 1 dose of romiplostim

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: From first dose through 8 weeks after last dose of study drug (11 weeks).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Romiplostim 30 µg	0 of 4 (0%)	4 of 4 (100%)	Not reported
Romiplostim 100 µg	0 of 4 (0%)	4 of 4 (100%)	Not reported
Romiplostim 300 µg	3 of 7 (42.9%)	7 of 7 (100%)	Not reported
Romiplostim 500 µg	1 of 1 (100%)	1 of 1 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 28411254Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14.
Background: PubMed 17061981Newland A, Caulier MT, Kappers-Klunne M, Schipperus MR, Lefrere F, Zwaginga JJ, Christal J, Chen CF, Nichol JL. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol. 2006 Nov;135(4):547-53. doi: 10.1111/j.1365-2141.2006.06339.x.
AmgenTrials clinical trials websiteLink provided by the sponsor.
FDA-approved Drug LabelingLink provided by the sponsor.

Related trials for Thrombocytopenic Purpura

RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
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 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (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.

MD study director

Amgen

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Romiplostim in people with Thrombocytopenic Purpura, 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 3 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: finished, as of the sponsor's last update on 26 Jun 2023. 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 combined 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.

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 20 Dec 2019). 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 Adverse Events (time frame: From first dose through 8 weeks after last dose of study drug (11 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 NCT00117143, 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 26 Jun 2023.