A Rollover Study to Provide Continued Treatment With Eltrombopag

NCT01957176 | Completed Phase 4 Results FDA Drug

• 3 other identifiers: 2001702013-001371-20CETB115A2X01B
• Study Type: interventional
• Target: 22
• Locations: 12 countries
• Sites: 13

Brief Summary

The objective of this study was to provide continued treatment with eltrombopag for subjects who were participating in a Novartis-sponsored investigational study with eltrombopag (parent studies 114968/ASPIRE (NCT01440374), PMA112509 (NCT00903422), and TRA105325/EXTEND (NCT00351468), receiving clinical benefit without unacceptable toxicity and to collect long-term safety data.

Conditions

Thrombocytopaenia

Keywords

myelodysplastic syndromes; thrombocytopenia; acute myeloid leukemia; idiopathic thrombocytopenic purpura; Eltrombopag; pediatrics

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs)

  The distribution of adverse events was done via the analysis of frequencies for Adverse Events (AEs) and Serious Adverse Events (SAEs), through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.

  From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.

Study Arms (3)

Cohort A (Myelodysplastic syndrome (MDS)/ Acute myeloid leukemia (AML) adult subjects)

EXPERIMENTAL

All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.

Drug: Eltrombopag (ELT)

Cohort B (Idiopathic thrombocytopenic purpura (ITP) adult subjects)

EXPERIMENTAL

All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.

Drug: Eltrombopag (ELT)

Cohort C (Idiopathic thrombocytopenic purpura (ITP) pediatric subjects)

EXPERIMENTAL

All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.

Drug: Eltrombopag (ELT)

Interventions

Eltrombopag (ELT) DRUG

Subjects were dosed with ELT tablets or powder for oral suspension (PfOS) based on the dosage form used in the parent study. ELT tablets were white, round film coated tablets containing ELT olamine equivalent to 12.5 mg, 25 mg, 50 mg, 75 mg and 100 mg of ELT. ELT PfOS was a reddish-brown to yellow powder contained inside an elongated sachet. Each sachet contained ELT olamine equivalent to 20 mg of ELT per gram of powder.

Also known as: SB-497115

Cohort A (Myelodysplastic syndrome (MDS)/ Acute myeloid leukemia (AML) adult subjects); Cohort B (Idiopathic thrombocytopenic purpura (ITP) adult subjects); Cohort C (Idiopathic thrombocytopenic purpura (ITP) pediatric subjects)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Written informed consent has been obtained from the subject (or subject's legally acceptable representative) prior to performance of any study-specific procedure.
• The subject is participating in a GSK sponsored investigational study of eltrombopag (parent study) within the past 28 days and is receiving clinical benefit without unacceptable toxicity as determined by the investigator.
• Subjects with a QTc \<450 millisecond (msec) or \<480 msec for subjects with bundle branch block. The QTc is the QT interval corrected for heart rate according to either Bazett's formula (QTcB), Fridericia's formula (QTcF) or another method, machine or manual overread. For subject eligibility and withdrawal QTcF will be used. For purposes of data analysis, QTcF will be used. The QTc should be based on single or averaged QTc values of triplicate electrocardiograms (ECGs) obtained over a brief recording period.
• Women must be either of non-child bearing potential or women with child-bearing potential and men with reproductive potential must be willing to practice acceptable methods of birth control during the study.
• Women of childbearing potential must have a negative serum pregnancy test within 14 days of the first dose of study treatment and agree to use effective contraception, during the study and for 4 weeks following the last dose of study treatment.
• Men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from time of first dose until 16 weeks after the last dose of study treatment.

You may not qualify if:

• Permanent discontinuation of eltrombopag in the parent study based upon the study treatment discontinuation or study withdrawal criteria from the parent study. Subjects who permanently discontinued treatment because they completed all study related treatments remain eligible.
• The subject is pregnant or a lactating female.
• Any serious and/or unstable pre-existing medical, psychiatric disorder or other conditions at the time of transition to this study that could interfere with subject's safety, obtaining informed consent or compliance with the study procedures, in the opinion of the investigator or GSK Medical Monitor.
• French subjects: The French subject has participated in any study using an investigational drug during the previous 30 days, with the exception of eltrombopag, in the parent study.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (13)

Novartis Investigative Site

Leuven, 3000, Belgium

Location

Novartis Investigative Site

Shanghai, 200025, China

Location

Novartis Investigative Site

Paris, 75571, France

Location

Novartis Investigative Site

Athens, 11527, Greece

Location

Novartis Investigative Site

Heraklion, Crete, 71201, Greece

Location

Novartis Investigative Site

Shatin, Hong Kong

Location

Novartis Investigative Site

Tullamore, Ireland

Location

Novartis Investigative Site

Amsterdam, 1081 HV, Netherlands

Location

Novartis Investigative Site

San Isidro, Lima region, Lima 27, Peru

Location

Novartis Investigative Site

Chorzów, 41-500, Poland

Location

Novartis Investigative Site

Bucharest, 022328, Romania

Location

Novartis Investigative Site

Seoul, 02841, South Korea

Location

Novartis Investigative Site

Sousse, 4000, Tunisia

Location

MeSH Terms

Conditions

Thrombocytopenia; Myelodysplastic Syndromes; Leukemia, Myeloid, Acute; Purpura, Thrombocytopenic, Idiopathic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Bone Marrow Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; 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: Study Director
• 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 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 4, 2013
• First Posted: October 8, 2013
• Study Start: October 15, 2013
• Primary Completion: February 23, 2022
• Study Completion: February 23, 2022
• Last Updated: December 30, 2022
• Results First Posted: December 30, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will share

Locations

CN (1); NL (1); HK (1); GR (2); IE (1); PE (1); FR (1); BE (1); PL (1); RO (1); KR (1); TU (1)