A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
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)
2 other identifiers
interventional
66
4 countries
31
Brief 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. This study is available to pediatric patients who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Dec 2009
Longer than P75 for phase_3
31 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 17, 2009
CompletedStudy Start
First participant enrolled
December 30, 2009
CompletedFirst Posted
Study publicly available on registry
February 19, 2010
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 12, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
January 12, 2017
CompletedResults Posted
Study results publicly available
January 29, 2019
CompletedJanuary 18, 2020
January 1, 2020
7 years
December 17, 2009
January 8, 2019
January 7, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
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.
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.
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.
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.
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
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
From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Study Arms (1)
Romiplostim
EXPERIMENTALParticipants 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.
Interventions
Administered by subcutaneous injection once a week.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Amgenlead
Study Sites (31)
Research Site
Orange, California, 92868, United States
Research Site
San Diego, California, 92123, United States
Research Site
Washington D.C., District of Columbia, 20010, United States
Research Site
Atlanta, Georgia, 30322, United States
Research Site
Chicago, Illinois, 60611, United States
Research Site
Peoria, Illinois, 61615, United States
Research Site
Indianapolis, Indiana, 46260, United States
Research Site
Iowa City, Iowa, 52242, United States
Research Site
Louisville, Kentucky, 40202, United States
Research Site
New Orleans, Louisiana, 70118, United States
Research Site
Detroit, Michigan, 48201, United States
Research Site
Kansas City, Missouri, 64108, United States
Research Site
Omaha, Nebraska, 68114, United States
Research Site
Las Vegas, Nevada, 89109, United States
Research Site
New Brunswick, New Jersey, 08901, United States
Research Site
New York, New York, 10016, United States
Research Site
New York, New York, 10021, United States
Research Site
Cincinnati, Ohio, 45229, United States
Research Site
Columbus, Ohio, 43205, United States
Research Site
Pittsburgh, Pennsylvania, 15224, United States
Research Site
Nashville, Tennessee, 37232, United States
Research Site
Dallas, Texas, 75390, United States
Research Site
Fort Worth, Texas, 76104, United States
Research Site
Houston, Texas, 77030, United States
Research Site
Randwick, New South Wales, 2031, Australia
Research Site
South Brisbane, Queensland, 4101, Australia
Research Site
Parkville, Victoria, 3052, Australia
Research Site
Toronto, Ontario, M5G 1X8, Canada
Research Site
Montreal, Quebec, H3T 1C5, Canada
Research Site
Montreal, Quebec, H4A 3J1, Canada
Research Site
Barcelona, Catalonia, 08035, Spain
Related Publications (1)
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.
PMID: 30846500BACKGROUND
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- IHQ Medical Info-Clinical Trials
- Organization
- Amgen (EUROPE) GmbH
Study Officials
- STUDY DIRECTOR
MD
Amgen
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- SUPPORTIVE CARE
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 17, 2009
First Posted
February 19, 2010
Study Start
December 30, 2009
Primary Completion
January 12, 2017
Study Completion
January 12, 2017
Last Updated
January 18, 2020
Results First Posted
January 29, 2019
Record last verified: 2020-01