Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

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Brief Title

Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

Official Title

A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)

Brief Summary

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


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Percentage of Time With a Platelet Response During the First 6 Months of Treatment

Secondary Outcome

 Percentage of Time With a Platelet Response During the Overall Treatment Period

Condition

Immune Thrombocytopenia

Intervention

Romiplostim

Study Arms / Comparison Groups

 Romiplostim
Description:  Participants received romiplostim administered weekly by subcutaneous injection for up to 3 years. The starting dose was 1 µg/kg titrated in 1 µg/kg increments up to a maximum of 10 µg/kg to reach a target platelet count ≥ 50 x 10⁹/L.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

203

Start Date

December 10, 2014

Completion Date

August 8, 2019

Primary Completion Date

August 30, 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Diagnosis of primary ITP according to The American Society of Hematology (ASH)
             Guidelines 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, intravenous
             Immunoglobulin (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 upper limit of normal (ULN)

               -  Total serum bilirubin ≤ 1.5 x the ULN

               -  Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 x the
                  ULN

          -  For the EU, Switzerland and Turkey protocol supplement, subject must agree to a
             scheduled bone marrow biopsy and aspirate at Year 1 or Year 2 following romiplostim
             treatment and any unscheduled biopsies if clinically indicated

          -  For the EU, Switzerland and Turkey protocol supplement, a reticulin grade of 0, 1, 2,
             or 3 according to the modified Bauermeister grading scale, as assessed by central
             laboratory from a bone marrow biopsy performed within 1 year prior to planned first
             dose of romiplostim or consent to a pre-treatment bone marrow biopsy and aspirate
             prior to planned first dose of romiplostim

        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 Hepatitis B, Hepatitis C or human immunodeficiency virus (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 pegylated recombinant human megakaryocyte growth and development
             factor (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
      

Gender

All

Ages

1 Year - 18 Years

Accepts Healthy Volunteers

No

Contacts

MD, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02279173

Organization ID

20101221

Secondary IDs

2011-005019-96

Responsible Party

Sponsor

Study Sponsor

Amgen


Study Sponsor

MD, Study Director, Amgen


Verification Date

July 2020