Trial Outcomes & Findings for A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP) (NCT NCT02201290)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

9 participants

Primary outcome timeframe

Up to week 4 follow up period

Results posted on

2019-07-05

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Eltrombopag treated participants
Overall Study STARTED	9
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Eltrombopag treated participants
Overall Study Withdrawal by Subject	2
Overall Study Lack of Efficacy	2
Overall Study Adverse Event	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eltrombopag n=9 Participants all treated participants
Age, Continuous	9.7 years STANDARD_DEVIATION 3.81 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Race/Ethnicity, Customized White - Caucasian	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to week 4 follow up period

Population: All treated subjects

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

Outcome measures

Outcome measures
Measure	Eltrombopag n=9 Participants Adverse events by system organ class and maximum severity for all treated participants
Number of Participants With Adverse Events Number of subjects with at least one event	3 participants
Number of Participants With Adverse Events Scleral haemorrhage	1 participants
Number of Participants With Adverse Events Autoimmune hepatitis	1 participants
Number of Participants With Adverse Events Epistaxis	1 participants

Adverse Events

Eltrombopag

Serious events: 3 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Eltrombopag n=9 participants at risk Eltrombopag was provided to sites by GlaxoSmithKline as tablets or as dry powder for oral suspension (PfOS) sachets
Eye disorders Scleral haemorrhage	11.1% 1/9 • up to week 4
Hepatobiliary disorders Autoimmune hepatitis	11.1% 1/9 • up to week 4
Respiratory, thoracic and mediastinal disorders Epistaxis	11.1% 1/9 • up to week 4

Other adverse events

Other adverse events
Measure	Eltrombopag n=9 participants at risk Eltrombopag was provided to sites by GlaxoSmithKline as tablets or as dry powder for oral suspension (PfOS) sachets
Infections and infestations Nasopharyngitis	33.3% 3/9 • up to week 4
Infections and infestations Pharyngitis	11.1% 1/9 • up to week 4
Infections and infestations Tonsillitis	11.1% 1/9 • up to week 4
Investigations Blood bilirubin increased	11.1% 1/9 • up to week 4
Metabolism and nutrition disorders Iron deficiency	11.1% 1/9 • up to week 4
Nervous system disorders Headache	22.2% 2/9 • up to week 4
Respiratory, thoracic and mediastinal disorders Epistaxis	11.1% 1/9 • up to week 4

Additional Information

Clinical Disclosure Office

Novartis Pharmaceuticals

Phone: (862) 778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER