Clinical trial NCT01071954: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
Keywords: Immune thrombocytopenic Purpura
Pediatric
Thrombocytopenia
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2010-02-19
Enrollment: 66
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.


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.

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: 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).
measure: Duration Adjusted Rate of Treatment Emergent Adverse Events
timeFrame: 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).
measure: Number of Participants Who Developed Antibodies to Romiplostim
timeFrame: 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.
measure: Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
timeFrame: 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 outcomes:
measure: Percentage of Participants With a Platelet Response
timeFrame: Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
measure: Percentage of Participants Who Used Concomitant ITP Therapy
timeFrame: From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Other outcomes:
Locations:
city: Orange
country: United States
facility: Research Site
state: California
city: San Diego
country: United States
facility: Research Site
state: California
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Chicago
country: United States
facility: Research Site
state: Illinois
city: Peoria
country: United States
facility: Research Site
state: Illinois
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: Iowa City
country: United States
facility: Research Site
state: Iowa
city: Louisville
country: United States
facility: Research Site
state: Kentucky
city: New Orleans
country: United States
facility: Research Site
state: Louisiana
city: Detroit
country: United States
facility: Research Site
state: Michigan
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Omaha
country: United States
facility: Research Site
state: Nebraska
city: Las Vegas
country: United States
facility: Research Site
state: Nevada
city: New Brunswick
country: United States
facility: Research Site
state: New Jersey
city: New York
country: United States
facility: Research Site
state: New York
city: New York
country: United States
facility: Research Site
state: New York
city: Cincinnati
country: United States
facility: Research Site
state: Ohio
city: Columbus
country: United States
facility: Research Site
state: Ohio
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Nashville
country: United States
facility: Research Site
state: Tennessee
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Fort Worth
country: United States
facility: Research Site
state: Texas
city: Houston
country: United States
facility: Research Site
state: Texas
city: Randwick
country: Australia
facility: Research Site
state: New South Wales
city: South Brisbane
country: Australia
facility: Research Site
state: Queensland
city: Parkville
country: Australia
facility: Research Site
state: Victoria
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Barcelona
country: Spain
facility: Research Site
state: Cataluña
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
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.