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

April 15, 2019 updated by: Novartis Pharmaceuticals

An Extension Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenia Purpura (ITP)

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.

Study Overview

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Krasnodar, Russian Federation, 350007
        • Novartis Investigative Site
      • Moscow, Russian Federation, 117997
        • Novartis Investigative Site
      • Saint Petersburg, Russian Federation, 198205
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 months to 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eltrombopag
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.
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 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events
Time Frame: 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

Up to week 4 follow up period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 18, 2013

Primary Completion (Actual)

July 4, 2017

Study Completion (Actual)

July 4, 2017

Study Registration Dates

First Submitted

June 6, 2013

First Submitted That Met QC Criteria

July 24, 2014

First Posted (Estimate)

July 28, 2014

Study Record Updates

Last Update Posted (Actual)

July 5, 2019

Last Update Submitted That Met QC Criteria

April 15, 2019

Last Verified

April 1, 2019

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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