HematologyNCT00472290

Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)

A study testing Romiplostim (formerly AMG 531) in people with Hematology and MDS, 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: Hematology, MDS, Myelodysplastic Syndromes
What is being tested?: Romiplostim (formerly AMG 531)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 72 participants
Where: No sites listed yet
Study dates: 1 Apr 2007to 26 Dec 2011
Phase: Interventional
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 completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS
Subject has an Eastern Cooperative Oncology (ECOG) performance status of 0 to 2
Subject had a platelet count ≤ 50 x 10^9/L since the final dose of investigational product in the parent study
Subject or his/her legally acceptable representative provided written informed consent before any study-specific procedures were initiated

Who cannot join

Subject has been diagnosed with AML or has a blast count ≥ 10% by peripheral blood or bone marrow biopsy
Subject has a prior history of leukemia
Subject has a prior history of bone marrow or stem cell transplantation
Subject has a prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before randomization
Subject has active or uncontrolled infections
Subject has unstable angina, congestive heart failure [New York Heart Association (NYHA) > class II], uncontrolled hypertension (diastolic > 100 mmHg), uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction

Show all 15Show fewer

Subject has a history of arterial thrombosis (eg, stroke or transient ischemic attack) in the past year
Subject has a history of venous thrombosis that currently requires anti-coagulation therapy
Subject received interleukin (IL)-11 within 4 weeks of screening
Subject previously received a thrombopoietic growth factor (other than romiplostim)
Subject has a known hypersensitivity to any recombinant E coli-derived product (eg, Infergen®, Neupogen®, Somatropin, Actimmune)
Subject is currently enrolled in investigational device or drug study(ies), has not yet completed at least 4 weeks since ending investigational device or drug study(ies) (other than parent romiplostim study), or subject is receiving other investigational agent(s)/device(s)
Subject is of child-bearing potential and is evidently pregnant (eg, positive human chorionic gonadotropin [HCG] test) or is breast feeding
Subject is not using adequate contraceptive precautions
Subject has any kind of disorder that compromises his/her ability to give written informed consent (and does not have a legally acceptable representative) or is unable to comply with study procedures

About this study

Official title: An Open Label Extension Study Evaluating the Safety of Long Term Dosing of Romiplostim in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)

This is an open label extension study of romiplostim for treatment of thrombocytopenia (platelet count ≤ 50 x 10^9/L) in MDS subjects. The study is designed to assess the long-term safety of treatment with romiplostim, as measured by incidence of overall adverse events, the incidence of bleeding events, the utilization of platelet transfusions, and the duration of platelet response. The study will further describe the time to disease progression to acute myeloid leukemia (AML) and survival.

Conditions: Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Being tested: Romiplostim (formerly AMG 531)
Keywords: Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Medical subject headings: Myelodysplastic Syndromes, Thrombocytopenia

What participants receive

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

Romiplostim (formerly AMG 531)Drug
Subjects will begin the study at an initial dose of 750 µg.
Except for:
- Subject whose doses were escalated to doses higher than 750 µg AMG 531 weekly, and maintained a response per IWG guidelines for platelet response.
- Subjects who were stable at a lower dose of AMG 531 on the previous study. Doses will be adjusted throughout the study based on individual subject's platelet count.

Study groups in NCT00472290
Study group	Role	What they receive
Open Label Romiplostim (formerly AMG 531)	Experimental	Not described Receives: Drug: Romiplostim (formerly AMG 531)

What is being measured

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

Overall Summary of Adverse Events
Measured: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .
Incidence of Antibody (AB) Formation
Measured: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.

Secondary outcomes (5)

Weekly Bleeding Events Per 100 Subject Years
During the time since the first dose of IP to the end of the treatment period. A single bleeding event was defined as each individual bleeding episode that originated from a specific organ system (eg, gastrointestinal system or central nervous system). A bleeding event that continued for more than 7 days was counted as separate events every eighth day.
Measured: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Platelet Transfusion Events Per 100 Subject Years
During the time since the first dose of IP to the end of the treatment period. A discrete platelet transfusion event was defined as any number of platelet transfusions administered within a 3-day period. Platelet transfusions administered more than 3 days apart were counted as separate platelet transfusion events.
Measured: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Weeks With Platelet Response Per Year
During the time since the first dose of IP to the end of the treatment period. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Measured: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Time to First Platelet Response
Time since first dose of IP to the first platelet response. Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Measured: During treatment period. The average duration of romiplostim exposure is 56 weeks.
Duration of Platelet Response
Platelet response was based on the modified IWG 2006 criteria (Cheson et al, 2006) and was defined as, in the absence of platelet transfusion: an absolute increase in platelet count of ≥ 30 x 10^9/L for a subject starting with a platelet count of ≥ 20 x 10^9/L; or an increase in platelet count from < 20 x 10^9/L to ≥ 20 x 10^9/L and by at least 100% in a subject that started with a platelet count < 20 x 10^9/L.
Measured: During treatment period. The average duration of romiplostim exposure is 56 weeks.

Timeline

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

1 Apr 200726 Dec 2011

First posted11 May 2007 (estimated)
Study start1 Apr 2007
Primary completion18 Jul 2011
Study completion26 Dec 2011

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 (1)Papers the sponsor has linked to this study.
Other trials for HematologyStudies 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 N/A
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 72 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00472290
Sponsor's protocol ID: 20060197
Status last verified: Dec 2017
First submitted: 10 May 2007
Last updated: 29 Dec 2017
Results posted: Yes, on ClinicalTrials.gov

Publications

Fenaux P, Muus P, Kantarjian H, Lyons RM, Larson RA, Sekeres MA, Becker PS, Orejudos A, Franklin J. Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy. Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14.
PubMed 28616874

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

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All Hematology trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open Label Extension Study Evaluating the Safety of Long Term Dosing of Romiplostim in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS)
ClinicalTrials.gov ID: NCT00472290
Acronym / sponsor ID: 20060197
Conditions: Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Keywords: Hematology, MDS, Myelodysplastic Syndromes, Thrombocytopenia
Study type: Interventional
Recruitment status: Completed
Enrolment: 72 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: 1 Apr 2007 (actual)
Primary completion: 18 Jul 2011 (actual)
Study completion: 26 Dec 2011 (actual)
First posted: 11 May 2007 (estimated)
Results: Posted 15 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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Open Label Romiplostim (formerly AMG 531)	Experimental	Drug: Romiplostim (formerly AMG 531)

Who may qualify

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

Key inclusion criteria (4 listed)

Subject completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS
Subject has an Eastern Cooperative Oncology (ECOG) performance status of 0 to 2
Subject had a platelet count ≤ 50 x 10^9/L since the final dose of investigational product in the parent study
Subject or his/her legally acceptable representative provided written informed consent before any study-specific procedures were initiated

Key exclusion criteria (15 listed)

Subject has been diagnosed with AML or has a blast count ≥ 10% by peripheral blood or bone marrow biopsy
Subject has a prior history of leukemia
Subject has a prior history of bone marrow or stem cell transplantation
Subject has a prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before randomization
Subject has active or uncontrolled infections

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
Romiplostim (formerly AMG 531)Subjects will begin the study at an initial dose of 750 µg. Except for: * Subject whose doses were escalated to doses higher than 750 µg AMG 531 weekly, and maintained a response per IWG guidelines for platelet response. * Subjects who were stable at a lower dose of AMG 531 on the previous study. Doses will be adjusted throughout the study based on individual subject's platelet count.	Drug	Open Label Romiplostim (formerly AMG 531)

Primary outcome

Overall Summary of Adverse Events
Time frame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .
Incidence of Antibody (AB) Formation
Time frame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.

5 secondary outcomes

Weekly Bleeding Events Per 100 Subject Years
Time frame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Platelet Transfusion Events Per 100 Subject Years
Time frame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Weeks With Platelet Response Per Year
Time frame: During the treatment period. The average duration of romiplostim exposure is 56 weeks.
Time to First Platelet Response
Time frame: During treatment period. The average duration of romiplostim exposure is 56 weeks.
Duration of Platelet Response
Time frame: During treatment period. The average duration of romiplostim exposure is 56 weeks.

Results and safety

Results posted to the registry on 15 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
Romiplostim	72	0

Primary outcome: Overall Summary of Adverse Events

Time frame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks .. Number. Unit: Participant

Measure	Romiplostim n = 72
Subjects Reporting Any AE	71
Subjects Reporting Any Treatment-related AE	19
Subjects Reporting Any Treat-related Serious AE	4
Subjects Reporting Any Serious AE	29
Subjects Withdrew Study or Stop IP Due to AE	8

Population analysed: Safety analysis includes subjects who took at least one dose of romiplostim.

Primary outcome: Incidence of Antibody (AB) Formation

Time frame: During treatment period from first dose of IP to End of Study visit, on Average 56 Weeks.. Number. Unit: Participant

Measure	Romiplostim n = 72
Positive binding AB to IP at or before baseline	5 (94.7)
Neutralizing AB to IP at or before baseline	0
Binding AB positive to IP post-baseline only	5
Persistent binding AB positive to IP	4
Transient binding AB positive to IP	1
Positive binding AB to TPO at or before baseline	4
Neutralizing AB to TPO at or before baseline	0
Binding AB positive to TPO post-baseline only	3

Population analysed: Safety analysis includes subjects who took at least one dose of romiplostim.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: The average duration is 56 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Romiplostim	29 of 72 (40.3%)	66 of 72 (91.7%)	Not reported

Limitations and caveats (from the sponsor): Early termination leading to small number of subjects analyzed;

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 28616874Fenaux P, Muus P, Kantarjian H, Lyons RM, Larson RA, Sekeres MA, Becker PS, Orejudos A, Franklin J. Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy. Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14.
AmgenTrials clinical trials websiteLink provided by the sponsor.

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RecruitingPhase 2
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Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
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Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
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RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
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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 study testing Romiplostim (formerly AMG 531) in people with Hematology and MDS, 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 4 inclusion and 15 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 29 Dec 2017. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 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?

Overall Summary of Adverse Events (time frame: During treatment period from first dose of IP to End of Study visit, on Average 56 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 NCT00472290, which also lists 1 linked publication.

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 29 Dec 2017.