Purpura, Thrombocytopenic, Idiopathic
Conditions
Keywords
Chronic immune thrombocytopenic purpura, ITP, eltrombopag olamine, thrombopoietin receptor agonist, pediatrics
Brief summary
This was an open-label Phase III extension study to evaluate the long-term safety of eltrombopag in pediatric patients with chronic ITP who previously participated in study TRA115450. This study allowed dosing of eltrombopag at an individualized dose for each subject based upon platelet count. The starting dose was based on the subject's dose at the end of the TRA115450 study. The maximum dose was 75 mg daily.
Interventions
DRUGEltrombopag Tablets
Eltrombopag tablets will be white, round film coated tablets containing eltrombopag olamine equivalent to 12.5 mg, 25 mg, 50 mg and 75 mg of eltrombopag. The 12.5 mg tablet will be smaller than the 25 mg, 50 mg and 75 mg tablets. Subjects will receive maximum dose of 75 mg once daily (QD).
DRUGEltrombopag PfOS
Eltrombopag PfOS is a reddish-brown to yellow powder contained inside an elongated sachet. Each sachet will contain eltrombopag olamine equivalent to 20 mg of eltrombopag per gram of powder. Subjects will receive maximum dose of 75 mg once daily (QD)
Sponsors
Novartis Pharmaceuticals
Study design
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
AEs were graded using the common toxicity criteria for adverse events (CTCAE) Version 4.03. If a patient reported more than one AE with the same preferred term (PT), the AE with the greatest severity was presented. If a patient reported more than one AE within the same primary system organ class (SOC), the patient was counted only once with the greatest severity at the SOC level, where applicable. An AE with missing CTCAE grade was included in the 'All grades' column of the summary tables. In AE summaries, the primary SOC was presented alphabetically and the PTs were sorted within the primary SOC in descending frequency.
Eligibility
Sex/Gender
ALL
Age
1 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Written informed consent must be obtained from the subject's guardian and accompanying informed assent from the subject (for children over 6 years old). * Subjects must be between 1 year and \<18 years of age at Day 1. * Subjects must have enrolled in TRA115450/PETIT2 study. * Subjects must have completed Part 1 and Part 2 of TRA115450/PETIT2 study. * Female subjects of child-bearing potential (after menarche) must have a negative pregnancy test within 24 hours of first dose of study treatment; agree and be able to provide a blood or urine specimen for pregnancy testing during the study; agree to use effective contraception during the study and for 28 days following the last dose of study treatment, and not be lactating. * Male subjects with a female partner of childbearing potential must agree to use effective contraception from 2 weeks prior to administration of the first dose of study treatment until 3 months after the last dose of study treatment.
Exclusion criteria
* Subjects with any clinically relevant abnormality, other than ITP, identified on the screening examination or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another primary diagnosis (e.g. Thrombocytopenia is secondary to another disease). * Any subject considered to be a child in care, defined as one who has been placed under the control or protection of an agency, organization, institution or entity by the courts, the government or a government body, acting in accordance with powers conferred on them by law or regulation. This can include a child cared for by foster parents or living in a care home or institution, provided that the arrangement falls within the definition above. The definition of a child in care does not include a child who is adopted or who has an appointed legal guardian.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events | Up to week 4 follow up period | Frequency of all adverse events (including Ophthalmic events) categorized using CTCAE toxicity grades and clinical laboratory test \[ Time Frame: Up to Week 4 Follow-up period \] Clinical laboratory assessments and frequency of all adverse events, categorized using Common Terminology Criteria for Adverse Events (CTCAE) toxicity grades will present safety and tolerability endpoints Serious Adverse Events are below. See All Adverse Events in the following section for specifics No statistical analysis was planned for this primary outcome |
Countries
Russia
Participant flow
Recruitment details
A total of 9 patients received Eltrombapag treatment during the extension study. Four patients (44.4%) completed the study while 5 patients (55.6%) discontinued from treatment and withdrew from the study.
Participants by arm
| Arm | Count |
|---|---|
| Eltrombopag all treated participants | 9 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Lack of Efficacy | 2 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Eltrombopag |
|---|---|
| Age, Continuous | 9.7 years STANDARD_DEVIATION 3.81 |
| Race/Ethnicity, Customized White - Caucasian | 9 Participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 9 |
| other Total, other adverse events | 6 / 9 |
| serious Total, serious adverse events | 3 / 9 |
Outcome results
Primary
Number of Participants With Adverse Events
Frequency of all adverse events (including Ophthalmic events) categorized using CTCAE toxicity grades and clinical laboratory test \[ Time Frame: Up to Week 4 Follow-up period \] Clinical laboratory assessments and frequency of all adverse events, categorized using Common Terminology Criteria for Adverse Events (CTCAE) toxicity grades will present safety and tolerability endpoints Serious Adverse Events are below. See All Adverse Events in the following section for specifics No statistical analysis was planned for this primary outcome
Time frame: Up to week 4 follow up period
Population: All treated subjects
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Eltrombopag | Number of Participants With Adverse Events | Number of subjects with at least one event | 3 participants |
| Eltrombopag | Number of Participants With Adverse Events | Scleral haemorrhage | 1 participants |
| Eltrombopag | Number of Participants With Adverse Events | Autoimmune hepatitis | 1 participants |
| Eltrombopag | Number of Participants With Adverse Events | Epistaxis | 1 participants |