Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
NCT ID: NCT01444417
Last Updated: 2017-02-09
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
62 participants
INTERVENTIONAL
2012-01-31
2015-02-28
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Romiplostim
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.
Romiplostim
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.
Placebo
Participants received weekly subcutaneous placebo for 24 weeks.
Placebo
Matching placebo administered by subcutaneous injection
Interventions
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Romiplostim
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.
Placebo
Matching placebo administered by subcutaneous injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
* Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
* Other criteria may apply
1 Year
17 Years
ALL
No
Sponsors
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Amgen
INDUSTRY
Responsible Party
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Principal Investigators
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MD
Role: STUDY_DIRECTOR
Amgen
Locations
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Research Site
Orange, California, United States
Research Site
San Diego, California, United States
Research Site
Washington D.C., District of Columbia, United States
Research Site
Atlanta, Georgia, United States
Research Site
Chicago, Illinois, United States
Research Site
Peoria, Illinois, United States
Research Site
Indianapolis, Indiana, United States
Research Site
Iowa City, Iowa, United States
Research Site
Louisville, Kentucky, United States
Research Site
New Orleans, Louisiana, United States
Research Site
Detroit, Michigan, United States
Research Site
Kansas City, Missouri, United States
Research Site
Omaha, Nebraska, United States
Research Site
Las Vegas, Nevada, United States
Research Site
New Brunswick, New Jersey, United States
Research Site
New York, New York, United States
Research Site
New York, New York, United States
Research Site
Cincinnati, Ohio, United States
Research Site
Columbus, Ohio, United States
Research Site
Philadelphia, Pennsylvania, United States
Research Site
Pittsburgh, Pennsylvania, United States
Research Site
Nashville, Tennessee, United States
Research Site
Fort Worth, Texas, United States
Research Site
Houston, Texas, United States
Research Site
La Crosse, Wisconsin, United States
Research Site
Randwick, New South Wales, Australia
Research Site
Herston, Queensland, Australia
Research Site
Parkville, Victoria, Australia
Research Site
Hamilton, Ontario, Canada
Research Site
Toronto, Ontario, Canada
Research Site
Montreal, Quebec, Canada
Research Site
Montreal, Quebec, Canada
Research Site
Québec, Quebec, Canada
Countries
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References
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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.
Related Links
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AmgenTrials clinical trials website
Other Identifiers
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2010-018426-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
20080279
Identifier Type: -
Identifier Source: org_study_id
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