ThrombocytopeniaPhase 3NCT00116688
Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
A Phase 3 study testing Romiplostim in people with Thrombocytopenia and Idiopathic Thrombocytopenic Purpura, run by Amgen.
Trial summary
The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Thrombocytopenia, Idiopathic 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
- 313 participants
- Where
- No sites listed yet
- Study dates
- Aug 2004to Jan 2010
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must have previously completed a romiplostim ITP study
- Platelet count ≤ 50 x 10 ^9/L
- Written informed consent
Who cannot join
- Bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim ITP study
- Received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study
- Currently enrolled in or has not yet completed at least 4 weeks since ending device or drug trial(s) (other than the previous romiplostim ITP study), or subject is receiving other investigational agent(s) other than romiplostim
- Not using adequate contraceptive precautions
- Not available for follow-up assessments
- Has any kind of disorder that compromises the ability of the participant to give informed consent and does not have a legally-acceptable representative and/or is unable to comply with study procedures.
About this study
Official title: An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of AMG 531 in Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.
- Being tested
- Romiplostim
- Keywords
- Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic
- Medical subject headings
- Thrombocytopenia, Purpura, Thrombocytopenic, Idiopathic, Purpura, Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimBiological
Also called AMG 531, Nplate
Romiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Romiplostim weekly subcutaneous dosing based on screening weight and platelet count. Starting dose of 1 µg/kg up to a maximum dose of 10 µg/kg. Receives: Biological: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events
Participants with one or more occurrences of one or more adverse events up to 8 weeks after the end of treatment. Participants with more than one event were only counted once.
Measured: Duration of treatment plus 8 weeks (up to 285 weeks)
Secondary outcomes (7)
Number of Participants With a Platelet Response
Platelet response was defined as having a platelet count of ≥ 50 x 10^9/L at any time on study, excluding platelet counts within 8 weeks after receiving any rescue medications.
Measured: Duration of treatment (up to 277 weeks)
Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies
The number of participants with a reduction or discontinuation of concurrent immune (idiopathic) thrombocytopenic purpura (ITP) therapies (corticosteroids, danazol, azathioprine) during the study.
Measured: Duration of treatment (up to 277 weeks)
Change From Baseline in ITP Patient Assessment Questionnaire
The ITP Patient Assessment Questionnaire (ITP-PAQ) assesses ITP-specific health-related quality of life (HRQOL). This questionnaire assesses ITP specific health-related quality of life (HRQOL). The questionnaire consists of 44 items and has six domains: These domains assess the impact of ITP on Physical Health, Mental Health, Work, Social Activity, Women's Health and Overall QOL. The impact of ITP on Physical Health consists of four sub-scales, which evaluate ITP related Symptoms, Fatigue, Bother and Activity. The impact of ITP on Mental Health consists of two sub-scales, which evaluate Psychological distress and Fear in a population with ITP. Items are scored from 0-100 with higher scores indicating better HRQOL.
Measured: Baseline to Week 48
Change From Baseline in Short Form 36 (SF-36)
The SF-36 is a widely used generic health-related quality of life measure. It has 36 questions with 8 domains: Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional and Mental Health. Items are scored from 0 to 100 with higher scores indicating better health status.
Measured: Baseline to Week 48
Change From Baseline in Euroqol-5D (EQ-5D) Index Score
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The instrument is applicable to a wide range of health conditions and treatments and results in a single index score and a visual analog scale (VAS) score. The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension comprises three levels (no problems, some/moderate problems, extreme problems). A unique EQ-5D health state is defined by combining one level from each of the five dimensions. EQ-5D index values range from -0.59 to 1.00. Higher EQ-5D Index scores represent better health status.
Measured: Baseline to Week 48
Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)
The EQ-5D is a patient-completed, multidimensional measure of health related quality of life. The EQ-5D VAS records the respondent's self-rated health status on a vertical graduated (0-100) visual analogue scale. Higher EQ-5D VAS scores represent better health status.
Measured: Baseline to Week 48
Patient Global Assessment
The Patient Global Assessment is two questions which assess the overall health-related quality of life (HRQOL) and symptoms of the patient. Each item is answered on a 15-point Likert scale ranging from 'A very great deal worse' (1) to 'A very great deal better' (15). A higher score indicates that quality of life or symptoms have improved.
Measured: Week 1 and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2005 (estimated)
- Study startAug 2004
- Primary completionJan 2010
- Study completionJan 2010
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 (5)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 313 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00116688
- Sponsor's protocol ID
- 20030213
- Status last verified
- Nov 2013
- First submitted
- 30 Jun 2005
- Last updated
- 18 Dec 2013
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
PubMed 18981291Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, Viallard JF, Macik G, Rummel M, Nie K, Jun S. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. doi: 10.1111/bjh.12260. Epub 2013 Feb 22.
PubMed 23432528Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001500;
Terrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001752;
Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.
PubMed 19671919
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of AMG 531 in Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
- ClinicalTrials.gov ID
- NCT00116688
- Acronym / sponsor ID
- 20030213
- Keywords
- Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 313 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- Aug 2004
- Primary completion
- Jan 2010 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 1 Jul 2005 (estimated)
- Results
- Posted 6 Apr 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety of romiplostim as a long-term treatment in thrombocytopenic subjects with ITP, to evaluate the long-term platelet response to romiplostim, and to evaluate changes in patient reported outcomes due to the use of romiplostim. Participants must have previously completed a romiplostim ITP study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Biological: RomiplostimRomiplostim weekly subcutaneous dosing based on screening weight and platelet count. Starting dose of 1 µg/kg up to a maximum dose of 10 µg/kg. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Must have previously completed a romiplostim ITP study
- Platelet count ≤ 50 x 10 ^9/L
- Written informed consent
Key exclusion criteria (6 listed)
- Bone marrow stem cell disorder or new active malignancies diagnosed since enrollment in the previous romiplostim ITP study
- Received any alkylating agents within 4 weeks before screening visit or anticipated use during the time of the proposed study
- Currently enrolled in or has not yet completed at least 4 weeks since ending device or drug trial(s) (other than the previous romiplostim ITP study), or subject is receiving other investigational agent(s) other than romiplostim
- Not using adequate contraceptive precautions
- Not available for follow-up assessments
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, NplateRomiplostim is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder. | Biological | Romiplostim |
Primary outcome
Number of Participants With Adverse Events
Time frame: Duration of treatment plus 8 weeks (up to 285 weeks)
Participants with one or more occurrences of one or more adverse events up to 8 weeks after the end of treatment. Participants with more than one event were only counted once.
7 secondary outcomes
Number of Participants With a Platelet Response
Time frame: Duration of treatment (up to 277 weeks)
Number of Participants With a Reduction or Discontinuation of Concurrent ITP Therapies
Time frame: Duration of treatment (up to 277 weeks)
Change From Baseline in ITP Patient Assessment Questionnaire
Time frame: Baseline to Week 48
Change From Baseline in Short Form 36 (SF-36)
Time frame: Baseline to Week 48
Change From Baseline in Euroqol-5D (EQ-5D) Index Score
Time frame: Baseline to Week 48
Change From Baseline in Euroqol-5D (EQ-5D) Visual Analogue Scale (VAS)
Time frame: Baseline to Week 48
Patient Global Assessment
Time frame: Week 1 and Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 18981291Bussel JB, Kuter DJ, Pullarkat V, Lyons RM, Guo M, Nichol JL. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009 Mar 5;113(10):2161-71. doi: 10.1182/blood-2008-04-150078. Epub 2008 Nov 3.
- Background: PubMed 23432528Kuter DJ, Bussel JB, Newland A, Baker RI, Lyons RM, Wasser J, Viallard JF, Macik G, Rummel M, Nie K, Jun S. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013 May;161(3):411-23. doi: 10.1111/bjh.12260. Epub 2013 Feb 22.
- BackgroundTerrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001500;
- BackgroundTerrell D, George J, Bussel J, Lyons R, Pullarkat V, Redner R, Nie, Selleslag D, Nie K, Woodard P.Home administration of romiplostim by patients with chronic immune thrombocytopenia (ITP).Journal-001752;
- Derived from this study: PubMed 19671919Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
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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 Thrombocytopenia and Idiopathic 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 6 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 18 Dec 2013. 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 6 Apr 2011). 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: Duration of treatment plus 8 weeks (up to 285 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 NCT00116688, which also lists 5 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 18 Dec 2013.