A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)

Novartis

Status and phase: Completed; Phase 3

Conditions: Purpura, Thrombocytopenic, Idiopathic

Treatments: Drug: Eltrombopag Tablets; Drug: Eltrombopag PfOS

Study type: Interventional

Funder types: Industry

Identifiers

NCT02201290

2017-004082-27 (EudraCT Number)

117366

CETB115BRU01 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 9 patients

Sex: All

Ages: 1 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

9 participants in 1 patient group

Eltrombopag

Experimental group

Description:

Eligible subject will be allocated to 1 of 3 age-defined cohorts. Cohort 1: between 12 and 17 years old, Cohort 2: between 6 and 11 years old, and Cohort 3: between 1 and 5 years old. For Cohorts 1 and 2, eltrombopag tablets will be administered, however, subjects in Cohort 2 may use eltrombopag powder for oral suspension (Eltrombopag PfOS) if they have difficulty swallowing tablets and are receiving a dose of eltrombopag of \< 40 mg. For Cohort 3, either eltrombopag tablets or PfOS will be administered.

Treatment:

Drug: Eltrombopag Tablets

Drug: Eltrombopag PfOS