Phase 3
N=66
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
Bottom Line
View on ClinicalTrials.gov: NCT01071954 ↗Enrolled (actual)
66
Serious AEs
29.2%
Results posted
Jan 2019
Primary outcome: Primary: Number of Participants With Adverse Events — 64; 19; 2; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Romiplostim (Biological)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Jan 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events |
64; 19; 2; 1; 21; 5 | — |
| PRIMARY Duration Adjusted Rate of Treatment Emergent Adverse Events |
1397.2; 29.7; 2.8; 0.6; 38.5; 6.6 | — |
| PRIMARY Number of Participants Who Developed Antibodies to Romiplostim |
5; 4; 1; 1; 0; 1 | — |
| PRIMARY Number of Participants Who Developed Antibodies to Endogenous Thrombopoietin |
2; 2; 0; 0; 0; 0 | — |
| SECONDARY Percentage of Participants With a Platelet Response |
93.8 | — |
| SECONDARY Percentage of Participants Who Used Concomitant ITP Therapy |
47.7 | — |
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.
Data sourced from ClinicalTrials.gov (NCT01071954). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.