Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITPPhase 3NCT01071954
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
A Phase 3 study testing Romiplostim in people with Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP, run by Amgen at 31 sites in 4 countries.
Trial summary
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
- 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
- 66 participants
- Where
- 31 sitesin 4 countries
- Study dates
- 30 Dec 2009to 12 Jan 2017
- 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
- Subject or subject's legally acceptable representative has provided informed consent.
- Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.
Who cannot join
- Subject has or previously had any 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).
- Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
- Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
- Other investigational medications are excluded.
- 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) (with the exception of romiplostim in a previous clinical study).
- Female subject of child bearing potential (defined as having first menses) is not willing to use highly effective contraception during treatment and for 4 weeks after the end of treatment.
Show all 11Show fewer
- Female subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment.
- Subject has known sensitivity to any of the products to be administered during dosing.
- Subject previously has entered this study (this will depend on the type of study).
- Subject will not be available for protocol required study visits, to the best of the subject 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
31 sites in 4 countries.
31 sites in 4 countries
United States24 sites
Research Site
Atlanta, Georgia, 30322
Research Site
Chicago, Illinois, 60611
Research Site
Cincinnati, Ohio, 45229
Research Site
Columbus, Ohio, 43205
Research Site
Dallas, Texas, 75390
Research Site
Detroit, Michigan, 48201
Research Site
Fort Worth, Texas, 76104
Research Site
Houston, Texas, 77030
Research Site
Indianapolis, Indiana, 46260
Research Site
Iowa City, Iowa, 52242
Research Site
Kansas City, Missouri, 64108
Research Site
Las Vegas, Nevada, 89109
Research Site
Louisville, Kentucky, 40202
Research Site
Nashville, Tennessee, 37232
Research Site
New Brunswick, New Jersey, 08901
Research Site
New Orleans, Louisiana, 70118
Research Site
New York, New York, 10016
Research Site
New York, New York, 10021
Research Site
Omaha, Nebraska, 68114
Research Site
Orange, California, 92868
Research Site
Peoria, Illinois, 61615
Research Site
Pittsburgh, Pennsylvania, 15224
Research Site
San Diego, California, 92123
Research Site
Washington D.C., District of Columbia, 20010
Australia3 sites
Research Site
Parkville, Victoria, 3052
Research Site
Randwick, New South Wales, 2031
Research Site
South Brisbane, Queensland, 4101
Canada3 sites
Research Site
Montreal, Quebec, H3T 1C5
Research Site
Montreal, Quebec, H4A 3J1
Research Site
Toronto, Ontario, M5G 1X8
About this study
Official title: An Open Label Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura. This study is available to pediatric patients who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.
- Conditions
- Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
- Being tested
- Romiplostim
- Keywords
- Immune thrombocytopenic Purpura, Pediatric, Thrombocytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimBiological
Also called Nplate
Administered by subcutaneous injection once a week.
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; 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 between 50 x 10^9/L and 200 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 Participants With Adverse Events
The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE.
A serious adverse event was defined as an adverse event that met at least one of the following serious criteria:
- fatal
- life threatening (places the subject at immediate risk of death)
- required in-patient hospitalization or prolongation of existing hospitalization
- resulted in persistent or significant disability/incapacity
- congenital anomaly/birth defect
- other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.
Measured: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Duration Adjusted Rate of Treatment Emergent Adverse Events
Exposure adjusted rate was defined as the total number of events divided by the duration of time participants were under observation.
The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE.
A serious adverse event was defined as an adverse event that met at least one of the following serious criteria:
- fatal
- life threatening (places the subject at immediate risk of death)
- required in-patient hospitalization or prolongation of existing hospitalization
- resulted in persistent or significant disability/incapacity
- congenital anomaly/birth defect
- other medically important serious event. The investigator assessed whether each adverse event was possibly related to the study drug.
Measured: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Number of Participants Who Developed Antibodies to Romiplostim
Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies.
Transient antibodies are those positive post-baseline but negative at the last time point tested.
Persistent antibodies were those positive at the last time point tested.
Measured: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies.
Transient antibodies are those positive post-baseline but negative at the last time point tested.
Persistent antibodies were those positive at the last time point tested.
Measured: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Secondary outcomes (2)
Percentage of Participants With a Platelet Response
Platelet response was defined as at least one platelet count ≥ 50 x 10^9/L in the absence of rescue medication during the study.
Measured: Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Percentage of Participants Who Used Concomitant ITP Therapy
Measured: From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2010 (estimated)
- Study start30 Dec 2009
- Primary completion12 Jan 2017
- Study completion12 Jan 2017
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 Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITPStudies 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 66 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
- NCT01071954
- Sponsor's protocol ID
- 20090340
- Other IDs
- 2009-016203-32, 2009
- Status last verified
- Jan 2020
- First submitted
- 17 Dec 2009
- Last updated
- 18 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Tarantino MD, Bussel JB, Blanchette VS, Beam D, Roy J, Despotovic J, Raj A, Carpenter N, Mehta B, Eisen M. Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia. Haematologica. 2019 Nov;104(11):2283-2291. doi: 10.3324/haematol.2018.202283. Epub 2019 Mar 7.
PubMed 30846500
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2020. 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 Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)
- ClinicalTrials.gov ID
- NCT01071954
- Acronym / sponsor ID
- 20090340
- Conditions
- Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
- Keywords
- Immune thrombocytopenic Purpura, Pediatric, Thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- 30 Dec 2009 (actual)
- Primary completion
- 12 Jan 2017 (actual)
- Study completion
- 12 Jan 2017 (actual)
- First posted
- 19 Feb 2010 (estimated)
- Results
- Posted 29 Jan 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura. This study is available to pediatric patients who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.
Study design
- Primary purpose
- Supportive care
- 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 by subcutaneous injection once a week. The starting dose of romiplostim was 1 μg/kg; 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 between 50 x 10^9/L and 200 x 10^9/L. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Subject or subject's legally acceptable representative has provided informed consent.
- Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.
Key exclusion criteria (11 listed)
- Subject has or previously had any 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).
- Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
- Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
- Other investigational medications are excluded.
- 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) (with the exception of romiplostim in a previous clinical study).
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: NplateAdministered by subcutaneous injection once a week. | Biological | Romiplostim |
Primary outcome
Number of Participants With Adverse Events
Time frame: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: * fatal * life threatening (places the subject at immediate risk of death) * required in-patient hospitalization or prolongation of existing hospitalization * resulted in persistent or significant disability/incapacity * congenital anomaly/birth defect * other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.
Duration Adjusted Rate of Treatment Emergent Adverse Events
Time frame: From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Exposure adjusted rate was defined as the total number of events divided by the duration of time participants were under observation. The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: * fatal * life threatening (places the subject at immediate risk of death) * required in-patient hospitalization or prolongation of existing hospitalization * resulted in persistent or significant disability/incapacity * congenital anomaly/birth defect * other medically important serious event. The investigator assessed whether each adverse event was possibly related to the study drug.
Number of Participants Who Developed Antibodies to Romiplostim
Time frame: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
Time frame: Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
2 secondary outcomes
Percentage of Participants With a Platelet Response
Time frame: Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Percentage of Participants Who Used Concomitant ITP Therapy
Time frame: From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 30846500Tarantino MD, Bussel JB, Blanchette VS, Beam D, Roy J, Despotovic J, Raj A, Carpenter N, Mehta B, Eisen M. Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia. Haematologica. 2019 Nov;104(11):2283-2291. doi: 10.3324/haematol.2018.202283. Epub 2019 Mar 7.
- AmgenTrials clinical trials websiteLink provided by the sponsor.
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
31 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Romiplostim in people with Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP, run by Amgen at 31 sites in 4 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 2 inclusion and 11 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 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, Australia, Canada, Spain. 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 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 29 Jan 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 of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).). The study lists 4 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 NCT01071954, 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 18 Jan 2020.