A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune (Idiopathic) Thrombocytopenia Purpura
Verified April 2015 by Amgen
Information provided by (Responsible Party):
First received: December 17, 2009
Last updated: April 29, 2015
Last verified: April 2015
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.
Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
||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)
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:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||December 2016 (Final data collection date for primary outcome measure)
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
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
|Ages Eligible for Study:
||1 Year and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
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
|Contact: Amgen Call Center
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||December 17, 2009
||April 29, 2015
||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
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on October 02, 2015
Blood Coagulation Disorders
Blood Platelet Disorders
Immune System Diseases
Signs and Symptoms