Safety and Efficacy Study of Romiplostim to Treat ITP in Pediatric Subjects
Information source: Amgen
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: 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
Intervention: romiplostim (Drug); Placebo (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: Amgen Official(s) and/or principal investigator(s): MD, Study Director, Affiliation: Amgen
Summary
The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of
thrombocytopenia in pediatric subjects with Immune Thrombocytopenia Purpura (ITP) as
measured by durable platelet response.
Clinical Details
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)
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Primary outcome: incidence of durable platelet response defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10^9/L during weeks 18 through 25 of treatment
Secondary outcome: incidence of overall platelet response defined as subjects who achieve a platelet count ≥ 50 x 10^9/L at a minimum of 4 times during weeks 2 to 25 of the treatment periodnumber of weekly platelet counts ≥ 50 x 10^9/L during weeks 2 to 25 of the treatment period incidence of rescue ITP medications used number of composite bleeding episodes defined as clinically significant bleeding events or the use of a rescue medication to prevent a clinically significant bleeding event during weeks 2 to 25 of the treatment period incidence of adverse events, including thromboembolic events and hematologic malignancies, clinically significant changes in laboratory values and the incidence of antibody formation
Eligibility
Minimum age: 1 Year.
Maximum age: 17 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Diagnosis of primary ITP according to the 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;AST and 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 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 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, PEG-rHuMGDF, Eltrombopag, rHuTPO or any platelet
producing agent
- Rituximab (for any indication) or 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 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
Locations and Contacts
Research Site, Orange, California 92868, United States
Research Site, San Diego, California 92123, United States
Research Site, Washington, 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, Randwick, New South Wales 2031, Australia
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, Hamilton, Ontario L8S 4K1, Canada
Research Site, Toronto, Ontario M5G 1X8, Canada
Research Site, Philadelphia, Pennsylvania 19104, United States
Research Site, Pittsburgh, Pennsylvania 15224, United States
Research Site, Montreal, Quebec H3H 1P3, Canada
Research Site, Montreal, Quebec H3T 1C5, Canada
Research Site, Quebec City, Quebec G1V 4G2, Canada
Research Site, Herston, Queensland 4029, Australia
Research Site, Nashville, Tennessee 37232, United States
Research Site, Fort Worth, Texas 76104, United States
Research Site, Houston, Texas 77030, United States
Research Site, Parkville, Victoria 3052, Australia
Research Site, La Crosse, Wisconsin 54601, United States
Additional Information
AmgenTrials clinical trials website
Starting date: December 2011
Last updated: July 13, 2015
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