Idiopathic Thrombocytopenic PurpuraPhase 3NCT01444417
Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
A Phase 3 study testing Romiplostim in people with Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen at 33 sites in 3 countries.
Trial summary
The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, 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?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 33 sitesin 3 countries
- Study dates
- Jan 2012to Feb 2015
- 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 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of primary ITP according to the American Society of Hematology (ASH) guidelines at least 6 months prior to screening, regardless of splenectomy status
- Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
- Age ≥ 1 year and < 18 years at the time of providing informed consent
- The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
- A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
- Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range during the screening period; aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 times the laboratory normal range during the screening period
- Hemoglobin > 10.0 g/dL during the screening period
- Subject and/or subject's legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required
Who cannot join
- Known history of a 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
- Known active or prior malignancy except adequately treated basal cell carcinoma
- Known history of congenital thrombocytopenia
- Known history of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV)
- Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
- Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
Show all 17Show fewer
- 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 venous thromboembolism or thrombotic events
- 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 6-mercaptopurine (6-MP) within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
- Splenectomy within 4 weeks of the screening visit
- All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
- Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
- Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
- Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
- Other criteria may apply
Trial locations and contacts
33 sites in 3 countries.
33 sites in 3 countries
United States25 sites
Research Site
Atlanta, Georgia, 30322
Research Site
Chicago, Illinois, 60611
Research Site
Cincinnati, Ohio, 45229
Research Site
Columbus, Ohio, 43205
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
La Crosse, Wisconsin, 54601
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
Philadelphia, Pennsylvania, 19104
Research Site
Pittsburgh, Pennsylvania, 15224
Research Site
San Diego, California, 92123
Research Site
Washington D.C., District of Columbia, 20010
Canada5 sites
Research Site
Hamilton, Ontario, L8S 4K1
Research Site
Montreal, Quebec, H3H 1P3
Research Site
Montreal, Quebec, H3T 1C5
Research Site
Québec, Quebec, G1V 4G2
Research Site
Toronto, Ontario, M5G 1X8
Australia3 sites
Research Site
Herston, Queensland, 4029
Research Site
Parkville, Victoria, 3052
Research Site
Randwick, New South Wales, 2031
About this study
Official title: A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response.
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura, Immune Thrombocytopenia
- Being tested
- Romiplostim, Placebo
- Keywords
- Immune (Idiopathic) Thrombocytopenic Purpura, Pediatric Immune (Idiopathic) Thrombocytopenic Purpura, Immune Thrombocytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia, Purpura, Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called AMG 531, Nplate®
The starting dose of romiplostim is 1 µg/kg administered weekly by subcutaneous injection. Participants will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L.
PlaceboDrug
Matching placebo administered by subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Romiplostim | Experimental | Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 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 ≥ 50 x 10^9/L. Receives: Drug: Romiplostim |
| Placebo | Placebo comparator | Participants received weekly subcutaneous placebo for 24 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With a Durable Platelet Response
A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10^9/L during the last 8 weeks of treatment (platelet counts obtained from week 18 to week 25). If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication.
Measured: Week 18 to week 25
Secondary outcomes (5)
Percentage of Participants With an Overall Platelet Response
Overall platelet response is defined as either a durable platelet response or transient platelet response.
Durable platelet response was defined as weekly platelet count ≥ 50 x 10^9/L for 6 or more times during week 18 to week 25 measurements. Participants may not have had a weekly response within 4 weeks after receiving any rescue medication.
Transient platelet response was defined as weekly platelet count ≥ 50 x 10^9/L for 4 or more times during week 2 to week 25 measurements but without durable platelet response. Participants may not have had a weekly response within 4 weeks after receiving any rescue medications.
Measured: Week 2 to week 25
Number of Weeks With Platelet Response
Number of weeks with platelet counts ≥ 50 x 10^9/L during week 2 to week 25 measurements. Participants may not have had a weekly response within 4 weeks after receiving any rescue medications.
Measured: Week 2 to week 25
Percentage of Participants Who Received Rescue Medication During the Treatment Period
Rescue medication is any medication (other than excluded medications) that is intended to increase platelet counts or prevent bleeding.
Measured: 24 weeks
Total Number of Composite Bleeding Episodes
A composite bleeding episode was defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinical significant bleeding event during weeks 2 through 25 of the treatment period. A clinically significant bleeding event was defined as a Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grade ≥ 2 bleeding event.
Measured: Week 2 to week 25
Number of Participants With Adverse Events
A serious adverse event is defined as an adverse event that meets at least 1 of the following serious criteria:
- fatal,
- life threatening (places the subject at immediate risk of death),
- requires in-patient hospitalization or prolongation of existing hospitalization,
- results in persistent or significant disability/incapacity,
- congenital anomaly/birth defect, and/or
- other significant medical hazard. Adverse events were graded for severity according to the CTCAE version 3.0 grading scale, where Grade 3 = moderate, Grade 4 = life-threatening and Grade 5 = fatal.
Treatment-related adverse events (TRAEs) were those assessed by the investigator as possibly related to study drug. This relationship was determined by a "yes" or "no" response to the question: "Is there a reasonable possibility that the event may have been caused by study drug?"
Measured: From the first dose of study drug until 4 weeks after last dose; 28 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2011 (estimated)
- Study startJan 2012
- Primary completionFeb 2015
- Study completionFeb 2015
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 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 62 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
- NCT01444417
- Sponsor's protocol ID
- 20080279
- Other IDs
- 2010-018426-39
- Status last verified
- Feb 2017
- First submitted
- 29 Sep 2011
- Last updated
- 9 Feb 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. doi: 10.1016/S0140-6736(16)00279-8. Epub 2016 Apr 18.
PubMed 27103127
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
- ClinicalTrials.gov ID
- NCT01444417
- Acronym / sponsor ID
- 20080279
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura, Immune Thrombocytopenia
- Keywords
- Immune (Idiopathic) Thrombocytopenic Purpura, Pediatric Immune (Idiopathic) Thrombocytopenic Purpura, Immune Thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- AmgenIndustry (pharma, biotech, devices)
- Study start
- Jan 2012
- Primary completion
- Feb 2015 (actual)
- Study completion
- Feb 2015 (actual)
- First posted
- 30 Sep 2011 (estimated)
- Results
- Posted 19 Jan 2017
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 romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Romiplostim | Experimental | Drug: RomiplostimParticipants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 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 ≥ 50 x 10^9/L. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants received weekly subcutaneous placebo for 24 weeks. |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Diagnosis of primary ITP according to the American Society of Hematology (ASH) guidelines at least 6 months prior to screening, regardless of splenectomy status
- Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
- Age ≥ 1 year and < 18 years at the time of providing informed consent
- The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
- A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
Key exclusion criteria (17 listed)
- Known history of a 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
- Known active or prior malignancy except adequately treated basal cell carcinoma
- Known history of congenital thrombocytopenia
- Known history of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV)
- Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
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, Nplate®The starting dose of romiplostim is 1 µg/kg administered weekly by subcutaneous injection. Participants will return to the clinic weekly to provide platelet counts and undergo dose titrations under the supervision of the treating physician. Weekly dose increases will continue in increments of 1 µg/kg up to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustment will be allowed during the treatment period to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L. | Drug | Romiplostim |
| PlaceboMatching placebo administered by subcutaneous injection | Drug | Placebo |
Primary outcome
Percentage of Participants With a Durable Platelet Response
Time frame: Week 18 to week 25
A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10^9/L during the last 8 weeks of treatment (platelet counts obtained from week 18 to week 25). If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication.
5 secondary outcomes
Percentage of Participants With an Overall Platelet Response
Time frame: Week 2 to week 25
Number of Weeks With Platelet Response
Time frame: Week 2 to week 25
Percentage of Participants Who Received Rescue Medication During the Treatment Period
Time frame: 24 weeks
Total Number of Composite Bleeding Episodes
Time frame: Week 2 to week 25
Number of Participants With Adverse Events
Time frame: From the first dose of study drug until 4 weeks after last dose; 28 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 27103127Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. doi: 10.1016/S0140-6736(16)00279-8. Epub 2016 Apr 18.
- 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
33 sites in 3 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 Idiopathic Thrombocytopenic Purpura and Thrombocytopenia, run by Amgen at 33 sites in 3 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 8 inclusion and 17 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 9 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 33 sites in United States, Canada, Australia. 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. 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?
Yes. Results are posted in the registry (first posted 19 Jan 2017). 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?
Percentage of Participants With a Durable Platelet Response (time frame: Week 18 to week 25). 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 NCT01444417, 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 9 Feb 2017.