Idiopathic Thrombocytopenic PurpuraPhase 2NCT01143038
Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim
A Phase 2 study testing Romiplostim in people with Idiopathic Thrombocytopenic Purpura, run by Amgen at 48 sites in 9 countries.
Trial summary
The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- 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
- 75 participants
- Where
- 48 sitesin 9 countries
- Study dates
- 30 Nov 2010to 26 Dec 2013
- Phase
- Phase 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
- Subject has been diagnosed with primary ITP according to the American Society of Hematology (ASH) guidelines (George et al, 1996) and previously received only 1st line therapies.
- First line therapy is defined as corticosteroids, immunoglobulin G (IVIG), anti-D and vinca alkaloids (used for the treatment of ITP related thrombocytopenia only). A platelet transfusion at any time during the six month period since the original diagnosis would not exclude the subject from study participation
- Initial diagnosis of primary ITP within 6 months of enrollment
- Age ≥ 18 years at screening
- A single platelet count ≤ 30 x 10⁹/L at any time during the screening period
- Subject or subject's legally acceptable representative has provided informed consent
Who cannot join
- Known history of a bone marrow stem cell disorder
- Surgical resection of the spleen
- Subject has a history of cancer or current malignancy other than basal cell carcinoma or cervical cancer in-situ with active treatment or disease within 5 years of screening
- Known history of congenital thrombocytopenia
- Known history of hepatitis B, hepatitis C, or human immunodeficiency virus
- Positive H. pylori by urea breath test or stool antigen test at screening
Show all 22Show fewer
- Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
- Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
- Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
- Previous history of recurrent venous thromboembolism or thrombotic events or an occurrence within 5 years of enrollment.
- Previous use of romiplostim, pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), eltrombopag, recombinant human thrombopoietin (rHuTPO) or any platelet producing agent
- Rituximab (for any indication) or mercaptopurine (6-MP) or anticipated use during the time of the proposed study
- All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
- Alkylating agents use at any time before or during the screening visit or anticipated during the time of the proposed study
- Known hypersensitivity to any recombinant E. coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
- Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s)
- Subject will have any other investigational procedures performed while enrolled in this clinical study
- Subject is pregnant or breast feeding, or planning to become pregnant within 5 weeks after the end of treatment
- Female subject of child bearing potential is not willing to use, in combination with her partner, highly effective contraception during treatment and for 4 weeks after the end of treatment
- Subject has previously enrolled into a romiplostim study
- Subject will not be available for protocol required study visits, to the best of the subject's and investigator's knowledge
- Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures
Trial locations and contacts
48 sites in 9 countries.
48 sites in 9 countries
United States8 sites
Research Site
Anaheim, California, 92801
Research Site
Bethesda, Maryland, 20817
Research Site
Boynton Beach, Florida, 33435
Research Site
Charleston, South Carolina, 29414
Research Site
Hickory, North Carolina, 28602
Research Site
Orange, California, 92868
Research Site
Philadelphia, Pennsylvania, 19140
Research Site
Richlands, Virginia, 24641
Italy8 sites
Research Site
Bari, 70124
Research Site
Catania, 95124
Research Site
Monza (MB), 20900
Research Site
Naples, 80131
Research Site
Novara, 28100
Research Site
Roma, 00161
Research Site
San Giovanni Rotondo FG, 71013
Research Site
Vicenza, 36100
Czechia5 sites
Research Site
Brno, 625 00
Research Site
Ostrava-Poruba, 708 52
Research Site
Prague, 100 34
Research Site
Prague, 128 08
Research Site
Prague, 128 20
France5 sites
Research Site
Bondy, 93143
Research Site
Créteil, 94010
Research Site
Dijon, 21000
Research Site
Pessac, 33604
Research Site
Toulouse, 31059
Poland5 sites
Research Site
Gdansk, 80-952
Research Site
Katowice, 40-032
Research Site
Lublin, 20-080
Research Site
Poznan, 61-505
Research Site
Wroclaw, 50-367
Spain5 sites
Research Site
A Coruña, Galicia, 15006
Research Site
Alcorcón, Madrid, 28922
Research Site
Barcelona, Catalonia, 08035
Research Site
Majadahonda, Madrid, 28222
Research Site
Málaga, Andalusia, 29010
United Kingdom5 sites
Research Site
Coventry, CV2 2DX
Research Site
Leeds, LS9 7TF
Research Site
London, E1 2ES
Research Site
London, SW17 0QT
Research Site
Oxford, OX3 9DU
Germany4 sites
Research Site
Cologne, 50674
Research Site
Dresden, 01307
Research Site
Duisburg, 47166
Research Site
Düsseldorf, 40479
Australia3 sites
Research Site
Adelaide, South Australia, 5000
Research Site
Randwick, New South Wales, 2031
Research Site
Woolloongabba, Queensland, 4102
About this study
Official title: A Phase 2 Interventional Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects With Immune Thrombocytopenia Purpura Receiving Romiplostim
The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.
Read the detailed description
The study includes a 4-week screening period, a 12-month romiplostim treatment period, and a romiplostim dose-tapering period.
During the 12-month treatment period romiplostim doses could be increased or decreased to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L. Participants who dose reduce such that they no longer require treatment with romiplostim during the 12-month treatment period will continue with all required study procedures up to 12 months and will be monitored for ITP remission for at least 6 months.
At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10^9/L will enter the tapering period, during which the romiplostim dose will be decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. If a participant maintains a platelet count of ≥ 50 x 10^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue), the participant will be followed for at least 6 months to confirm the incidence of ITP remission. If a participant's platelet count falls below 50 x 10^9/L and the participant has tapered off treatment with romiplostim, the participant will enter the stabilization period and reinitiate romiplostim for up to 8 weeks.
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Being tested
- Romiplostim
- Keywords
- ITP
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
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 will be administered weekly by subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Receives: Biological: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Months With Platelet Response During the 12-Month Treatment Period
The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.
Measured: 12 months
Secondary outcomes (4)
Percentage of Participants With ITP Remission
ITP remission was defined as maintaining every platelet count ≥ 50 x 10^9/L for at least 6 months in the absence of romiplostim and any other therapies to treat ITP.
Measured: Up to 24 months
Percentage of Participants With Splenectomy During the 12-month Treatment Period
If treatment with romiplostim was deemed ineffective or intolerable by the investigator, a splenectomy may have been performed.
Measured: 12 months
Number of Participants With Adverse Events
An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment-related (TRAE) or not was determined by investigator.
Measured: From first dose date of romiplostim to end of study (up to 24 months).
Number of Participants Who Developed Antibodies to Romiplostim
The number of participants who developed antibody formation (defined as negative at baseline and positive at post-baseline, transient or persistent) to romiplostim, endogenous thrombopoietin (eTPO), and thrombopoietin mimetic peptide (TMP, the peptide component of romiplostim) was summarized.
Measured: Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2010 (estimated)
- Study start30 Nov 2010
- Primary completion20 Sep 2013
- Study completion26 Dec 2013
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 (4)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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 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
- NCT01143038
- Sponsor's protocol ID
- 20080435
- Other IDs
- 2010-019987-35
- Status last verified
- Sep 2022
- First submitted
- 10 Jun 2010
- Last updated
- 21 Sep 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Newland A, Godeau B, Priego V, Viallard JF, Lopez Fernandez MF, Orejudos A, Eisen M. Remission and platelet responses with romiplostim in primary immune thrombocytopenia: final results from a phase 2 study. Br J Haematol. 2016 Jan;172(2):262-73. doi: 10.1111/bjh.13827. Epub 2015 Nov 5.
PubMed 26537623Cines 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 32129511
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Interventional Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects With Immune Thrombocytopenia Purpura Receiving Romiplostim
- ClinicalTrials.gov ID
- NCT01143038
- Acronym / sponsor ID
- 20080435
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Keywords
- ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 75 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 30 Nov 2010 (actual)
- Primary completion
- 20 Sep 2013 (actual)
- Study completion
- 26 Dec 2013 (actual)
- First posted
- 14 Jun 2010 (estimated)
- Results
- Posted 24 Feb 2016
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 describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.
Detailed description
The study includes a 4-week screening period, a 12-month romiplostim treatment period, and a romiplostim dose-tapering period.
During the 12-month treatment period romiplostim doses could be increased or decreased to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L. Participants who dose reduce such that they no longer require treatment with romiplostim during the 12-month treatment period will continue with all required study procedures up to 12 months and will be monitored for ITP remission for at least 6 months.
At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10^9/L will enter the tapering period, during which the romiplostim dose will be decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. If a participant maintains a platelet count of ≥ 50 x 10^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue), the participant will be followed for at least 6 months to confirm the incidence of ITP remission. If a participant's platelet count falls below 50 x 10^9/L and the participant has tapered off treatment with romiplostim, the participant will enter the stabilization period and reinitiate romiplostim for up to 8 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Biological: RomiplostimParticipants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. |
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 has been diagnosed with primary ITP according to the American Society of Hematology (ASH) guidelines (George et al, 1996) and previously received only 1st line therapies.
- First line therapy is defined as corticosteroids, immunoglobulin G (IVIG), anti-D and vinca alkaloids (used for the treatment of ITP related thrombocytopenia only). A platelet transfusion at any time during the six month period since the original diagnosis would not exclude the subject from study participation
- Initial diagnosis of primary ITP within 6 months of enrollment
- Age ≥ 18 years at screening
- A single platelet count ≤ 30 x 10⁹/L at any time during the screening period
Key exclusion criteria (22 listed)
- Known history of a bone marrow stem cell disorder
- Surgical resection of the spleen
- Subject has a history of cancer or current malignancy other than basal cell carcinoma or cervical cancer in-situ with active treatment or disease within 5 years of screening
- Known history of congenital thrombocytopenia
- Known history of hepatitis B, hepatitis C, or human immunodeficiency virus
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 will be administered weekly by subcutaneous injection | Biological | Romiplostim |
Primary outcome
Number of Months With Platelet Response During the 12-Month Treatment Period
Time frame: 12 months
The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.
4 secondary outcomes
Percentage of Participants With ITP Remission
Time frame: Up to 24 months
Percentage of Participants With Splenectomy During the 12-month Treatment Period
Time frame: 12 months
Number of Participants With Adverse Events
Time frame: From first dose date of romiplostim to end of study (up to 24 months).
Number of Participants Who Developed Antibodies to Romiplostim
Time frame: Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 26537623Newland A, Godeau B, Priego V, Viallard JF, Lopez Fernandez MF, Orejudos A, Eisen M. Remission and platelet responses with romiplostim in primary immune thrombocytopenia: final results from a phase 2 study. Br J Haematol. 2016 Jan;172(2):262-73. doi: 10.1111/bjh.13827. Epub 2015 Nov 5.
- 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.
- 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
48 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim in people with Idiopathic Thrombocytopenic Purpura, run by Amgen at 48 sites in 9 countries.
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 22 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 21 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Italy, Czechia, France, Poland and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
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 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 24 Feb 2016). 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 Months With Platelet Response During the 12-Month Treatment Period (time frame: 12 months). 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 NCT01143038, which also lists 4 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 21 Sep 2022.