A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Amgen
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT01071954
First received: December 17, 2009
Last updated: July 11, 2014
Last verified: July 2014
  Purpose

This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic subjects with Immune (Idiopathic) Thrombocytopenia Purpura. This study is available to pediatric subjects who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.


Condition Intervention Phase
Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Biological: Romiplostim
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
Official Title: An Open Label Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

Resource links provided by NLM:


Further study details as provided by Amgen:

Primary Outcome Measures:
  • the subject incidence and exposure adjusted incidence of adverse events, including clinically significant changes in laboratory values and incidence of antibody formation. [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Platelet response to romiplostim defined as platelet counts 50x109/L in the absence of rescue medication. [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • The prevalence of concurrent ITP therapy use over time (corticosteroids, danazol, azathioprine, etc.). [ Time Frame: 5 years ] [ Designated as safety issue: No ]

Estimated Enrollment: 100
Study Start Date: December 2009
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Single Arm
Starting Dose of 1µg/kg romiplostim or previous dose administered weekly Individual Subject Dose Adjustment to a Maximum Dose of 10 µg/kg based on platelet count
Biological: Romiplostim

Starting Dose of 1µg/kg romiplostim or previous dose administered weekly

Individual Subject Dose Adjustment to a Maximum Dose of 10 µg/kg based on platelet count

Rescue Medications Allowed at Discretion of Investigator

Reduction in Concurrent ITP Therapies May Occur when Platelet Count is >50 x 109/L


  Eligibility

Ages Eligible for Study:   1 Year and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject or subject's legally acceptable representative has provided informed consent.
  • Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.

Exclusion Criteria:

  • Subject has or previously had any 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).
  • Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
  • Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
  • Other investigational medications are excluded.
  • Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s) (with the exception of romiplostim in a previous clinical study).
  • Female subject of child bearing potential (defined as having first menses) is not willing to use highly effective contraception during treatment and for 4 weeks after the end of treatment.
  • Female subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment.
  • Subject has known sensitivity to any of the products to be administered during dosing.
  • Subject previously has entered this study (this will depend on the type of study).
  • Subject will not be available for protocol required study visits, to the best of the subject and investigator's knowledge.
  • Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01071954

Contacts
Contact: Amgen Call Center 866-572-6436

  Show 28 Study Locations
Sponsors and Collaborators
Amgen
Investigators
Study Director: MD Amgen
  More Information

Additional Information:
No publications provided

Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01071954     History of Changes
Other Study ID Numbers: 20090340
Study First Received: December 17, 2009
Last Updated: July 11, 2014
Health Authority: Australia: Therapeutic Goods Administration
European Union: European Medicines Agency
United States: Food and Drug Administration
Canada: Health Canada

Keywords provided by Amgen:
Immune thrombocytopenic Purpura
Pediatric
Thrombocytopenia

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

ClinicalTrials.gov processed this record on October 01, 2014