ThrombocytopeniaPhase 3NCT00102323
AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy
A Phase 3 study testing AMG 531 in people with Thrombocytopenia and Idiopathic Thrombocytopenic Purpura, run by Amgen.
Trial summary
The purpose of this study is to evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by the platelet response. This study will also evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia, Idiopathic Thrombocytopenic Purpura
- What is being tested?
- AMG 531
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- No sites listed yet
- Study dates
- 29 Mar 2005to 1 Mar 2007
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Diagnosis of ITP according to American Society of Hematology (ASH) guidelines (Appendix F)
- Have had a splenectomy for the treatment of ITP greater than or equal to 24 weeks prior to study entry
- Subjects greater than 60 years of age must have a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
- The platelet count (calculated from the mean of the 2 counts taken during the screening and pre-treatment periods) must be:
- * less than 30 x 10^9/L for those subjects not receiving any ITP therapy, with no count greater than 35 x 10^9/L,
- * less than 50 x 10^9/L for those subjects receiving a constant dose schedule of corticosteroids, azathioprine or danazol with no count greater than 55 x 10^9/L
Show all 10Show fewer
- A serum creatinine concentration less than or equal to 2 mg/dl(less than or equal to 176.8 µmol/L)
- Adequate liver function, as evidenced by a serum bilirubin less than or equal to 1.5 times the laboratory normal range
- Hemoglobin greater than 11.0 g/dL
- Written informed consent (see Section 12.1)
Who cannot join
- Any known history of bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study)
- Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
- Documented diagnosis of arterial thrombosis (i.e., stroke, transient ischemic attack or myocardial infarction) in the past year
- History of venous thrombosis (i.e., deep vein thrombosis, pulmonary embolism) including those subjects who are on ant-coagulation therapy
- Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure [NYHA greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or cardiac arrhythmia)
- Have 3 or more of the following predisposing factors for thromboembolic events: diabetes; smoker; using oral contraceptives; on estrogen therapy; known positive for anti-phospholipid antibodies; hypertriglyceridemia; hypercholesteremia (greater than 240 mg/dL); treatment for hypertension
Show all 19Show fewer
- Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C virus
- Currently receiving any treatment for ITP except corticosteroids, azathioprine or danazol administered at a constant dose and schedule
- IV Ig or anti-D Ig within 2 weeks before the screening visit
- Rituximab (for any indication) within 14 weeks before the screening visit or anticipated use during the time of the proposed study
- Received hematopoietic growth factors, including IL-11 (oprelvekin) within 4 weeks before the screening visit
- Past or present participation in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531 or related platelet product
- Received any aklylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
- Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication before the screening period
- Less than 8 weeks since major surgery
- Pregnant or breast feeding
- Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator
- Known hypersensitivity to any recombinant E coli-derived product
- Concerns for subject's compliance with the protocol
About this study
Official title: A Randomized, Placebo Controlled Study Evaluating the Efficacy and Safety of AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy
The purpose of this study is to evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by the platelet response. This study will also evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531.
- Being tested
- Placebo, AMG 531
- Keywords
- immune (idiopathic) thrombocytopenic purpura, post-splenectomy, ITP, Thrombopenia
- 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.
PlaceboDrug
Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. Placebo is supplied as a lyophilized power in a 5 mL single use glass vial.
AMG 531Biological
Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. AMG 531 is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| AMG 531 | Experimental | Active Investigational Product Receives: Biological: AMG 531 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by durable platelet response during the last 8 weeks of treatment and other platelet response parameters
Measured: Last 8 weeks of treatment
Secondary outcomes (3)
To evaluate the overall safety of AMG 531
Measured: Entire study duration plus follow up period
To evaluate the possible reductions in concurrent ITP therapies while receiving AMG 531
Measured: Entire study duration
To evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531
Measured: Entire study duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2005 (estimated)
- Study start29 Mar 2005
- Primary completion1 Sep 2006
- Study completion1 Mar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)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
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 63 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
- NCT00102323
- Sponsor's protocol ID
- 20030105
- Status last verified
- Nov 2022
- First submitted
- 27 Jan 2005
- Last updated
- 7 Nov 2022
- Results posted
- No
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 28411254Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M. Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies. Br J Haematol. 2019 May;185(3):503-513. doi: 10.1111/bjh.15803. Epub 2019 Feb 21.
PubMed 30793285Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R. Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials. Am J Hematol. 2020 Jun;95(6):643-651. doi: 10.1002/ajh.25776. Epub 2020 Mar 21.
PubMed 32129511Weitz I, Sanz MA, Henry D, Schipperus M, Godeau B, Northridge K, Gleeson M, Danese M, Deuson R. A novel approach to the evaluation of bleeding-related episodes in patients with chronic immune thrombocytopenia. Curr Med Res Opin. 2012 May;28(5):789-96. doi: 10.1185/03007995.2012.684046. Epub 2012 Apr 25.
PubMed 22494019Kuter 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 19671919Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrere F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. doi: 10.1016/S0140-6736(08)60203-2.
PubMed 18242413
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo Controlled Study Evaluating the Efficacy and Safety of AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Refractory to Splenectomy
- ClinicalTrials.gov ID
- NCT00102323
- Acronym / sponsor ID
- 20030105
- Keywords
- immune (idiopathic) thrombocytopenic purpura, post-splenectomy, ITP, Thrombopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 29 Mar 2005 (actual)
- Primary completion
- 1 Sep 2006 (actual)
- Study completion
- 1 Mar 2007 (actual)
- First posted
- 28 Jan 2005 (estimated)
- Results
- Not posted in the source record
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 evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by the platelet response. This study will also evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| AMG 531 | Experimental | Biological: AMG 531Active Investigational Product |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of ITP according to American Society of Hematology (ASH) guidelines (Appendix F)
- Have had a splenectomy for the treatment of ITP greater than or equal to 24 weeks prior to study entry
- Subjects greater than 60 years of age must have a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
- The platelet count (calculated from the mean of the 2 counts taken during the screening and pre-treatment periods) must be:
- * less than 30 x 10^9/L for those subjects not receiving any ITP therapy, with no count greater than 35 x 10^9/L,
Key exclusion criteria (19 listed)
- Any known history of bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study)
- Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
- Documented diagnosis of arterial thrombosis (i.e., stroke, transient ischemic attack or myocardial infarction) in the past year
- History of venous thrombosis (i.e., deep vein thrombosis, pulmonary embolism) including those subjects who are on ant-coagulation therapy
- Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure [NYHA greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or cardiac arrhythmia)
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 |
|---|---|---|
| PlaceboWeekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. Placebo is supplied as a lyophilized power in a 5 mL single use glass vial. | Drug | Placebo |
| AMG 531Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. AMG 531 is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder. | Biological | AMG 531 |
Primary outcome
To evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by durable platelet response during the last 8 weeks of treatment and other platelet response parameters
Time frame: Last 8 weeks of treatment
3 secondary outcomes
To evaluate the overall safety of AMG 531
Time frame: Entire study duration plus follow up period
To evaluate the possible reductions in concurrent ITP therapies while receiving AMG 531
Time frame: Entire study duration
To evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531
Time frame: Entire study duration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Nov 2022.
- 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 30793285Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M. Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies. Br J Haematol. 2019 May;185(3):503-513. doi: 10.1111/bjh.15803. Epub 2019 Feb 21.
- Background: PubMed 32129511Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R. Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials. Am J Hematol. 2020 Jun;95(6):643-651. doi: 10.1002/ajh.25776. Epub 2020 Mar 21.
- Derived from this study: PubMed 22494019Weitz I, Sanz MA, Henry D, Schipperus M, Godeau B, Northridge K, Gleeson M, Danese M, Deuson R. A novel approach to the evaluation of bleeding-related episodes in patients with chronic immune thrombocytopenia. Curr Med Res Opin. 2012 May;28(5):789-96. doi: 10.1185/03007995.2012.684046. Epub 2012 Apr 25.
- 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.
- Derived from this study: PubMed 18242413Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrere F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. doi: 10.1016/S0140-6736(08)60203-2.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
- FDA-approved Drug LabelingLink 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 AMG 531 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 10 inclusion and 19 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 7 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by durable platelet response during the last 8 weeks of treatment and other platelet response parameters (time frame: Last 8 weeks of treatment). 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 NCT00102323, which also lists 6 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 7 Nov 2022.