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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01071954
• Date of registration: 17/12/2009
• Prospective Registration: Yes
• Primary sponsor: Amgen
• 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)
• Date of first enrolment: December 30, 2009
• Target sample size: 66
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01071954
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Supportive Care. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Spain, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion 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.



Age minimum: 1 Year
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP

Intervention(s)

Biological: Romiplostim

Primary Outcome(s)

Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin [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.]

Duration Adjusted Rate of Treatment Emergent Adverse Events [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).]

Number of Participants Who Developed Antibodies to Romiplostim [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.]

Number of Participants With Adverse Events [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).]

Secondary Outcome(s)

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).]

Percentage of Participants With a Platelet Response [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).]

Secondary ID(s)

2009-016203-32

20090340

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 29/01/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01071954