Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
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)
2 other identifiers
interventional
62
3 countries
33
Brief 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.
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 Jan 2012
Typical duration for phase_3
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 29, 2011
CompletedFirst Posted
Study publicly available on registry
September 30, 2011
CompletedStudy Start
First participant enrolled
January 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2015
CompletedResults Posted
Study results publicly available
January 19, 2017
CompletedFebruary 9, 2017
February 1, 2017
3.1 years
September 29, 2011
November 17, 2016
February 7, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
Week 18 to week 25
Secondary Outcomes (5)
Percentage of Participants With an Overall Platelet Response
Week 2 to week 25
Number of Weeks With Platelet Response
Week 2 to week 25
Percentage of Participants Who Received Rescue Medication During the Treatment Period
24 weeks
Total Number of Composite Bleeding Episodes
Week 2 to week 25
Number of Participants With Adverse Events
From the first dose of study drug until 4 weeks after last dose; 28 weeks.
Study Arms (2)
Romiplostim
EXPERIMENTALParticipants 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 COMPARATORParticipants received weekly subcutaneous placebo for 24 weeks.
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
- 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
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Amgenlead
Study Sites (33)
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
Philadelphia, Pennsylvania, 19104, United States
Research Site
Pittsburgh, Pennsylvania, 15224, United States
Research Site
Nashville, Tennessee, 37232, United States
Research Site
Fort Worth, Texas, 76104, United States
Research Site
Houston, Texas, 77030, United States
Research Site
La Crosse, Wisconsin, 54601, United States
Research Site
Randwick, New South Wales, 2031, Australia
Research Site
Herston, Queensland, 4029, Australia
Research Site
Parkville, Victoria, 3052, Australia
Research Site
Hamilton, Ontario, L8S 4K1, Canada
Research Site
Toronto, Ontario, M5G 1X8, Canada
Research Site
Montreal, Quebec, H3H 1P3, Canada
Research Site
Montreal, Quebec, H3T 1C5, Canada
Research Site
Québec, Quebec, G1V 4G2, Canada
Related Publications (1)
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.
PMID: 27103127DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Amgen Inc.
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
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 29, 2011
First Posted
September 30, 2011
Study Start
January 1, 2012
Primary Completion
February 1, 2015
Study Completion
February 1, 2015
Last Updated
February 9, 2017
Results First Posted
January 19, 2017
Record last verified: 2017-02