Clinical trial NCT02201290: A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Official title: An Extension Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenia Purpura (ITP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Purpura, Thrombocytopenic, Idiopathic
Keywords: Chronic immune thrombocytopenic purpura, ITP, eltrombopag olamine, thrombopoietin receptor agonist, pediatrics
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-07-28
Enrollment: 9
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
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.
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: Up to week 4 follow up period
Secondary outcomes:
Other outcomes:
Locations:
city: Krasnodar
country: Russian Federation
facility: Novartis Investigative Site
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
city: Saint Petersburg
country: Russian Federation
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: