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

Primary Purpose

Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Romiplostim
Sponsored by
Amgen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP focused on measuring Immune thrombocytopenic Purpura, Pediatric, Thrombocytopenia

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Romiplostim

Arm Description

Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 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 between 50 x 10^9/L and 200 x 10^9/L.

Outcomes

Primary Outcome Measures

Number of Participants With Adverse Events
The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: fatal life threatening (places the subject at immediate risk of death) required in-patient hospitalization or prolongation of existing hospitalization resulted in persistent or significant disability/incapacity congenital anomaly/birth defect other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.
Duration Adjusted Rate of Treatment Emergent Adverse Events
Exposure adjusted rate was defined as the total number of events divided by the duration of time participants were under observation. The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: fatal life threatening (places the subject at immediate risk of death) required in-patient hospitalization or prolongation of existing hospitalization resulted in persistent or significant disability/incapacity congenital anomaly/birth defect other medically important serious event. The investigator assessed whether each adverse event was possibly related to the study drug.
Number of Participants Who Developed Antibodies to Romiplostim
Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.

Secondary Outcome Measures

Percentage of Participants With a Platelet Response
Platelet response was defined as at least one platelet count ≥ 50 x 10^9/L in the absence of rescue medication during the study.
Percentage of Participants Who Used Concomitant ITP Therapy

Full Information

First Posted
December 17, 2009
Last Updated
January 7, 2020
Sponsor
Amgen
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1. Study Identification

Unique Protocol Identification Number
NCT01071954
Brief Title
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
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)
Study Type
Interventional

2. Study Status

Record Verification Date
January 2020
Overall Recruitment Status
Completed
Study Start Date
December 30, 2009 (Actual)
Primary Completion Date
January 12, 2017 (Actual)
Study Completion Date
January 12, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amgen

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura. This study is available to pediatric patients who have completed a previous romiplostim ITP study and meet the eligibility criteria of this study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
Keywords
Immune thrombocytopenic Purpura, Pediatric, Thrombocytopenia

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
66 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Romiplostim
Arm Type
Experimental
Arm Description
Participants received romiplostim administered by subcutaneous injection once a week. The starting dose of romiplostim was 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 between 50 x 10^9/L and 200 x 10^9/L.
Intervention Type
Biological
Intervention Name(s)
Romiplostim
Other Intervention Name(s)
Nplate
Intervention Description
Administered by subcutaneous injection once a week.
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events
Description
The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: fatal life threatening (places the subject at immediate risk of death) required in-patient hospitalization or prolongation of existing hospitalization resulted in persistent or significant disability/incapacity congenital anomaly/birth defect other medically important serious event. The investigator assessed whether each adverse event was possibly related to the investigational product.
Time Frame
From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Title
Duration Adjusted Rate of Treatment Emergent Adverse Events
Description
Exposure adjusted rate was defined as the total number of events divided by the duration of time participants were under observation. The adverse event (AE) severity grading scale used was the Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 grading scale, where Grade 1 = Mild AE; Grade 2 = Moderate AE; Grade 3 = Severe AE; Grade 4 = Life-threatening or disabling AE; Grade 5 = Death related to AE. A serious adverse event was defined as an adverse event that met at least one of the following serious criteria: fatal life threatening (places the subject at immediate risk of death) required in-patient hospitalization or prolongation of existing hospitalization resulted in persistent or significant disability/incapacity congenital anomaly/birth defect other medically important serious event. The investigator assessed whether each adverse event was possibly related to the study drug.
Time Frame
From first dose of study drug until 1 week after last dose. The median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Title
Number of Participants Who Developed Antibodies to Romiplostim
Description
Two validated assays were used to test for antibodies to romiplostim / the thrombopoietin-mimetic peptide component of romiplostim (TMP). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
Time Frame
Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Title
Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
Description
Two validated assays were used to test for antibodies to endogenous thrombopoietin (TPO). The first was an immunoassay to confirm the presence of antibodies. The second was a cell-based bioassay to detect neutralizing or inhibitory effects in vitro. If a sample was positive in both assays, a participant was defined as positive for neutralizing antibodies. Transient antibodies are those positive post-baseline but negative at the last time point tested. Persistent antibodies were those positive at the last time point tested.
Time Frame
Once a year until the end of treatment and 1 week after the end of treatment; median (minimim, maximum) time on study was 34 (2, 91) months.
Secondary Outcome Measure Information:
Title
Percentage of Participants With a Platelet Response
Description
Platelet response was defined as at least one platelet count ≥ 50 x 10^9/L in the absence of rescue medication during the study.
Time Frame
Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Title
Percentage of Participants Who Used Concomitant ITP Therapy
Time Frame
From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MD
Organizational Affiliation
Amgen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Research Site
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Research Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Research Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Research Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Research Site
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61615
Country
United States
Facility Name
Research Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Research Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Research Site
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Research Site
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118
Country
United States
Facility Name
Research Site
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Research Site
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Research Site
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68114
Country
United States
Facility Name
Research Site
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89109
Country
United States
Facility Name
Research Site
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
Research Site
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Research Site
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Research Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Research Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Research Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Research Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Research Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Research Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Research Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Research Site
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Facility Name
Research Site
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Facility Name
Research Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Research Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Research Site
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
Research Site
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4A 3J1
Country
Canada
Facility Name
Research Site
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08035
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
30846500
Citation
Tarantino MD, Bussel JB, Blanchette VS, Beam D, Roy J, Despotovic J, Raj A, Carpenter N, Mehta B, Eisen M. Long-term treatment with romiplostim and treatment-free platelet responses in children with chronic immune thrombocytopenia. Haematologica. 2019 Nov;104(11):2283-2291. doi: 10.3324/haematol.2018.202283. Epub 2019 Mar 7.
Results Reference
background
Links:
URL
http://www.amgentrials.com
Description
AmgenTrials clinical trials website

Learn more about this trial

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

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