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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01071954




Registration number
NCT01071954
Ethics application status
Date submitted
17/12/2009
Date registered
19/02/2010
Date last updated
18/01/2020

Titles & IDs
Public title
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
Scientific 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)
Secondary ID [1] 0 0
2009-016203-32
Secondary ID [2] 0 0
20090340
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Romiplostim

Experimental: Romiplostim - 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.


Treatment: Other: Romiplostim
Administered by subcutaneous injection once a week.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events
Timepoint [1] 0 0
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).
Primary outcome [2] 0 0
Duration Adjusted Rate of Treatment Emergent Adverse Events
Timepoint [2] 0 0
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).
Primary outcome [3] 0 0
Number of Participants Who Developed Antibodies to Romiplostim
Timepoint [3] 0 0
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.
Primary outcome [4] 0 0
Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin
Timepoint [4] 0 0
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 [1] 0 0
Percentage of Participants With a Platelet Response
Timepoint [1] 0 0
Assessed every 4 weeks for the duration of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).
Secondary outcome [2] 0 0
Percentage of Participants Who Used Concomitant ITP Therapy
Timepoint [2] 0 0
From baseline to the end of treatment; the median (minimum, maximum) duration of treatment was 135.0 weeks (5, 363 weeks).

Eligibility
Key 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.
Minimum age
1 Year
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Other
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/12/2009
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/01/2017
Sample size
Target
Accrual to date
Final
66
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Research Site - Randwick
Recruitment hospital [2] 0 0
Research Site - South Brisbane
Recruitment hospital [3] 0 0
Research Site - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Kentucky
Country [8] 0 0
United States of America
State/province [8] 0 0
Louisiana
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
Nebraska
Country [12] 0 0
United States of America
State/province [12] 0 0
Nevada
Country [13] 0 0
United States of America
State/province [13] 0 0
New Jersey
Country [14] 0 0
United States of America
State/province [14] 0 0
New York
Country [15] 0 0
United States of America
State/province [15] 0 0
Ohio
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
Tennessee
Country [18] 0 0
United States of America
State/province [18] 0 0
Texas
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario
Country [20] 0 0
Canada
State/province [20] 0 0
Quebec
Country [21] 0 0
Spain
State/province [21] 0 0
Cataluña

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT01071954
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01071954