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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02279173
First Posted: October 30, 2014
Last Update Posted: November 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Amgen
  Purpose
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 Intervention Phase
Immune Thrombocytopenia Drug: Romiplostim Phase 3

Study Type: Interventional
Study Design: 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)

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • 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:
  • 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

  • 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.

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

  • 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

  • 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:
  • 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).

  • 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.

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


Enrollment: 204
Actual Study Start Date: December 10, 2014
Estimated Study Completion Date: September 23, 2019
Primary Completion Date: March 9, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Romiplostim
Romiplostim subcutaneous weekly injection
Drug: Romiplostim
Romiplostim subcutaneous weekly injection

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.
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

  Contacts and Locations
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
  More Information

Additional Information:
Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT02279173     History of Changes
Other Study ID Numbers: 20101221
2011-005019-96 ( EudraCT Number )
First Submitted: October 1, 2014
First Posted: October 30, 2014
Last Update Posted: November 17, 2017
Last Verified: November 2017

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