Overview

This trial is active, not recruiting.

Condition immune thrombocytopenia (itp)
Treatment romiplostim
Phase phase 3
Sponsor Amgen
Start date December 2014
End date November 2019
Trial size 204 participants
Trial identifier NCT02279173, 20101221, 2011-005019-96

Summary

This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric subjects with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10^9/L and in the absence of ITP rescue medications in the past 4 weeks.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Romiplostim subcutaneous weekly injection
romiplostim
Romiplostim subcutaneous weekly injection

Primary Outcomes

Measure
The percentage of time with a platelet count of ≥ 50 x 10^9/L starting from week 2 in the first 6 months of the treatment period without rescue medication use in the past 4 weeks
time frame: 6 Months

Secondary Outcomes

Measure
The percentage of time with a platelet count of ≥ 50 x 10^9/L starting from week 2 until the end of the treatment period without rescue medication use in the past 4 weeks
time frame: 36 months
The percentage of time with an increase in platelet count ≥ 20 x10^9/L above baseline starting from week 2 until the end of the treatment period without rescue medication use within the past 4 weeks.
time frame: 36 months
Subject incidence of rescue ITP medications used
time frame: 36 months
The incidence of anti-romiplostim neutralizing antibodies and cross-reactive antibodies to thrombopoietin (TPO) at any time during the study
time frame: 38 months
The incidence of adverse events, including clinically significant changes in laboratory values
time frame: 38 months

Eligibility Criteria

Male or female participants from 1 year up to 18 years old.

Inclusion Criteria: Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines (Neunert et al, 2011) at least 6 months before screening, regardless of splenectomy status Age ≥ 1 year and < 18 years of age Refractory to prior ITP therapy, relapsed after at prior ITP therapy, or be ineligible for other therapies Examples of prior therapy include: corticosteroids, IVIG, anti-D immunoglobulin, platelet transfusions. Platelet count ≤ 30 x10^9/L or is experiencing uncontrolled bleeding Has provided informed consent before any study-specific procedure; Adequate hematologic, renal, and liver function during screening Hemoglobin > 10.0 g/dL Serum creatinine ≤ 1.5 x the ULN Total serum bilirubin ≤ 1.5 x the ULN AST and ALT ≤ 3.0 x the ULN Exclusion Criteria: 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) Prior bone marrow transplant or peripheral blood progenitor cell transplant Active or prior malignancy except non-melanoma skin cancers within the last 5 years History of myelodysplastic syndrome History of bleeding diathesis History of congenital thrombocytopenia History of HepB, HepC or HIV History of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia History of antiphospholipid antibody syndrome or known positive for lupus anticoagulant History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura History of venous thromboembolism or thrombotic events Previous use of romiplostim or previous use of eltrombopag within 4 weeks of enrollment Previous use of PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study Splenectomy within 4 weeks of 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 Currently enrolled in another investigational device or drug study, or less than 30 days since ending investigational study Will have investigational procedures while enrolled on study Female subject of child bearing potential (defined as having first menses) not willing to use, in combination with her partner highly effective methods of birth control during treatment and for 1 month after the end of treatment Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen®, Neupogen®, somatropin, and Actimmune®) Has previously enrolled into this study Will not be available for protocol-required study visits or procedures, to the best of the subject's and investigator's knowledge Any kind of disorder that, may compromise the subject to give written informed consent and/or to comply with all required study procedures

Additional Information

Official title A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
Description This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric subjects with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10^9/L and in the absence of ITP rescue medications in the past 4 weeks. This protocol will provide open label romiplostim to thrombocytopenic pediatric subjects with ITP diagnosed for at least 6 months and who have received at least 1 prior ITP therapy (excluding romiplostim) or are ineligible for other ITP therapies.
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Amgen.