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Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amgen
ClinicalTrials.gov Identifier:
NCT01444417
First received: September 29, 2011
Last updated: February 7, 2017
Last verified: February 2017
Results First Received: November 17, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Participant, Investigator, Outcomes Assessor;   Primary Purpose: Treatment
Conditions: Idiopathic Thrombocytopenic Purpura
Thrombocytopenia
Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Thrombocytopenic Purpura
Immune Thrombocytopenia
Interventions: Drug: Romiplostim
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
This study was conducted at 27 centers in the United States, Canada, and Australia. Participants were enrolled from 24 January 2012 to 03 September 2014.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Participants who met eligibility criteria were enrolled and stratified by the following 3 age categories: ≥ 1 to < 6 years; 6 to < 12 years; and 12 to < 18 years.

Reporting Groups
  Description
Placebo Participants received weekly subcutaneous placebo for 24 weeks.
Romiplostim Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L.

Participant Flow:   Overall Study
    Placebo   Romiplostim
STARTED   20   42 
Received Treatment   19   42 
COMPLETED   18   41 
NOT COMPLETED   2   1 
Ineligibility Determined                0                1 
Withdrawal by Subject                2                0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Placebo Participants received weekly subcutaneous placebo for 24 weeks.
Romiplostim Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L.
Total Total of all reporting groups

Baseline Measures
   Placebo   Romiplostim   Total 
Overall Participants Analyzed 
[Units: Participants]
 20   42   62 
Age 
[Units: Years]
Mean (Standard Deviation)
 9.4  (4.7)   9.7  (4.1)   9.6  (4.3) 
Age, Customized 
[Units: Participants]
     
≥ 1 - < 6 years   4   8   12 
≥ 6 - < 12 years   9   18   27 
≥ 12 - < 18 years   7   16   23 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      11  55.0%      24  57.1%      35  56.5% 
Male      9  45.0%      18  42.9%      27  43.5% 
Race/Ethnicity, Customized 
[Units: Participants]
     
American Indian or Alaska Native   0   0   0 
Asian   2   3   5 
Black or African American   2   6   8 
Multiple   0   1   1 
Native Hawaiian or Other Pacific Islander   0   1   1 
Other   1   5   6 
White   15   26   41 
Platelets 
[Units: 10^9/L]
Mean (Standard Deviation)
 19.9  (19.3)   17.5  (10.7)   18.3  (13.9) 


  Outcome Measures
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1.  Primary:   Percentage of Participants With a Durable Platelet Response   [ Time Frame: Week 18 to week 25 ]

2.  Secondary:   Percentage of Participants With an Overall Platelet Response   [ Time Frame: Week 2 to week 25 ]

3.  Secondary:   Number of Weeks With Platelet Response   [ Time Frame: Week 2 to week 25 ]

4.  Secondary:   Percentage of Participants Who Received Rescue Medication During the Treatment Period   [ Time Frame: 24 weeks ]

5.  Secondary:   Total Number of Composite Bleeding Episodes   [ Time Frame: Week 2 to week 25 ]

6.  Secondary:   Number of Participants With Adverse Events   [ Time Frame: From the first dose of study drug until 4 weeks after last dose; 28 weeks. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Amgen Inc.
phone: 866-572-6436


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01444417     History of Changes
Other Study ID Numbers: 20080279
2010-018426-39 ( EudraCT Number )
Study First Received: September 29, 2011
Results First Received: November 17, 2016
Last Updated: February 7, 2017