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

NCT02201290 | Completed Phase 3 Results

• 3 other identifiers: 117366CETB115BRU012017-004082-27
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 3

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.

Conditions

Purpura, Thrombocytopenic, Idiopathic

Keywords

Chronic immune thrombocytopenic purpura, ITP, eltrombopag olamine, thrombopoietin receptor agonist, pediatrics

Outcome Measures

Primary Outcomes (1)

• 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

  Up to week 4 follow up period

Study Arms (1)

Eltrombopag

EXPERIMENTAL

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.

Drug: Eltrombopag Tablets; Drug: Eltrombopag PfOS

Interventions

Eltrombopag Tablets DRUG

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).

Eltrombopag

Eltrombopag PfOS DRUG

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)

Eltrombopag

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

Novartis Investigative Site

Krasnodar, 350007, Russia

Location

Novartis Investigative Site

Moscow, 117997, Russia

Location

Novartis Investigative Site

Saint Petersburg, 198205, Russia

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Clinical Disclosure Office
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

(862) 778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: June 6, 2013
• First Posted: July 28, 2014
• Study Start: June 18, 2013
• Primary Completion: July 4, 2017
• Study Completion: July 4, 2017
• Last Updated: July 5, 2019
• Results First Posted: July 5, 2019

Record last verified: 2019-04

Locations

RU (3)