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Single Arm, Open-label, Long-term Study of Romiplostim in Thrombocytopenic Pediatric Subjects With ITP.

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ClinicalTrials.gov Identifier: NCT02279173
Recruitment Status : Active, not recruiting
First Posted : October 30, 2014
Last Update Posted : October 29, 2018
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief 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.

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia Drug: Romiplostim Phase 3

Detailed 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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 204 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
Actual Study Start Date : December 10, 2014
Actual Primary Completion Date : March 9, 2017
Estimated Study Completion Date : September 23, 2019


Arm Intervention/treatment
Experimental: Romiplostim
Romiplostim subcutaneous weekly injection
Drug: Romiplostim
Romiplostim subcutaneous weekly injection




Primary Outcome Measures :
  1. 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 ]
    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


Secondary Outcome Measures :
  1. 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 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

  2. 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 ]
    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.

  3. Subject incidence of rescue ITP medications used [ Time Frame: 36 months ]
    Subject incidence of rescue ITP medications used

  4. 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 anti-romiplostim neutralizing antibodies and cross-reactive antibodies to thrombopoietin (TPO) at any time during the study

  5. The incidence of adverse events, including clinically significant changes in laboratory values [ Time Frame: 38 months ]
    The incidence of adverse events, including clinically significant changes in laboratory values


Other Outcome Measures:
  1. The subject incidence with a sustained platelet count of ≥ 50 x 10^9/L for 6 months or greater without the use of any ITP medications (concomitant, rescue, or romiplostim). [ Time Frame: 36 Months ]
    • The subject incidence with a sustained platelet count of ≥50 x 10^9/L for 6 months or greater without the use of any ITP medications (concomitant, rescue, or romiplostim).

  2. The incidence of splenectomy during the treatment period for subjects entering the study pre-splenectomy. [ Time Frame: 36 months ]
    The incidence of splenectomy during the treatment period for subjects entering the study pre-splenectomy.

  3. The subject incidence of romiplostim self-administration. [ Time Frame: 36 months ]
    The subject incidence of romiplostim self-administration.



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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02279173


  Show 78 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen

Additional Information:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02279173     History of Changes
Other Study ID Numbers: 20101221
2011-005019-96 ( EudraCT Number )
First Posted: October 30, 2014    Key Record Dates
Last Update Posted: October 29, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication (or other new use) have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
URL: https://www.amgen.com/datasharing

Keywords provided by Amgen:
Pediatric
Immune Thrombocytopenia
Amgen

Additional relevant MeSH terms:
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms