ThrombocytopaeniaPhase 4NCT01957176
A Rollover Study to Provide Continued Treatment With Eltrombopag
A Phase 4 study testing Eltrombopag (ELT) in people with Thrombocytopaenia, run by Novartis Pharmaceuticals at 13 sites in 12 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopaenia
- What is being tested?
- Eltrombopag (ELT)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 13 sitesin 12 countries
- Study dates
- 15 Oct 2013to 23 Feb 2022
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- In France, a subject will be eligible for inclusion in this study only if either affiliated to, or a beneficiary of, a social security category.
Who cannot join
- 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.
Trial locations and contacts
13 sites in 12 countries.
13 sites in 12 countries
Greece2 sites
Novartis Investigative Site
Athens, 11527
Novartis Investigative Site
Heraklion, Crete, 71201
About this study
Official title: Study 200170: A Rollover Study to Provide Continued Treatment With Eltrombopag
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.
Read the detailed description
This Phase IV, multicenter, non-randomized, open-label, uncontrolled, rollover study was designed to provide continued access to eltrombopag to eligible subjects. As this study was a rollover study, the subjects were enrolled into this study from the parent studies.
There were 3 cohorts in this study:
Cohort A: Adult subjects who have completed study treatment with eltrombopag during their participation in a parent study for MDS/AML (i.e., 114968/ASPIRE and PMA112509).
Cohort B: Adult subjects who have completed study treatment with eltrombopag during their participation in a parent study for ITP (i.e., TRA105325/EXTEND).
Cohort C: Pediatric subjects who have completed study treatment with eltrombopag during their participation in a parent study for ITP. Once a subject turned 18 years of age, they might remain in the study and follow the Cohort B procedures. No pediatric subjects were enrolled in this cohort/study.
The study consisted of a transition visit, study treatment visits, and a follow-up visit. Subjects in Cohort A and Cohort B completed the Transition visit assessments and then returned for their next schedule visit as per the Visit schedule. In the study treatment visit, subjects received a starting dose of eltrombopag at the same dose and administration that they were receiving at the time of their last study treatment visit in the parent study.
Safety was evaluated through physical examinations, clinical laboratory tests, and monitoring of adverse events. Additional safety assessments were done as per standard of care and/or when medically indicated.
Assessment of clinical benefit was performed throughout the study using local standard of care as determined by the Investigator to determine continued study participation and treatment with eltrombopag. Only subjects considered by the Investigator to be receiving clinical benefit without unacceptable toxicity may continue on study treatment. Once treatment with eltrombopag was permanently discontinued, the subject would attend the follow-up visit.
- Conditions
- Thrombocytopaenia
- Being tested
- Eltrombopag (ELT)
- Keywords
- myelodysplastic syndromes, thrombocytopenia, acute myeloid leukemia, idiopathic thrombocytopenic purpura, Eltrombopag, pediatrics
- Medical subject headings
- Thrombocytopenia, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute, Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Eltrombopag (ELT)Drug
Also called SB-497115
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: Eltrombopag (ELT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2013 (estimated)
- Study start15 Oct 2013
- Primary completion23 Feb 2022
- Study completion23 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ThrombocytopaeniaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01957176
- Sponsor's protocol ID
- 200170
- Other IDs
- 2013-001371-20, CETB115A2X01B
- Status last verified
- Nov 2022
- First submitted
- 4 Oct 2013
- Last updated
- 30 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study 200170: A Rollover Study to Provide Continued Treatment With Eltrombopag
- ClinicalTrials.gov ID
- NCT01957176
- Acronym / sponsor ID
- 200170
- Conditions
- Thrombocytopaenia
- Keywords
- myelodysplastic syndromes, thrombocytopenia, acute myeloid leukemia, idiopathic thrombocytopenic purpura, Eltrombopag, pediatrics
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 15 Oct 2013 (actual)
- Primary completion
- 23 Feb 2022 (actual)
- Study completion
- 23 Feb 2022 (actual)
- First posted
- 8 Oct 2013 (estimated)
- Results
- Posted 30 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This Phase IV, multicenter, non-randomized, open-label, uncontrolled, rollover study was designed to provide continued access to eltrombopag to eligible subjects. As this study was a rollover study, the subjects were enrolled into this study from the parent studies.
There were 3 cohorts in this study:
Cohort A: Adult subjects who have completed study treatment with eltrombopag during their participation in a parent study for MDS/AML (i.e., 114968/ASPIRE and PMA112509).
Cohort B: Adult subjects who have completed study treatment with eltrombopag during their participation in a parent study for ITP (i.e., TRA105325/EXTEND).
Cohort C: Pediatric subjects who have completed study treatment with eltrombopag during their participation in a parent study for ITP. Once a subject turned 18 years of age, they might remain in the study and follow the Cohort B procedures. No pediatric subjects were enrolled in this cohort/study.
The study consisted of a transition visit, study treatment visits, and a follow-up visit. Subjects in Cohort A and Cohort B completed the Transition visit assessments and then returned for their next schedule visit as per the Visit schedule. In the study treatment visit, subjects received a starting dose of eltrombopag at the same dose and administration that they were receiving at the time of their last study treatment visit in the parent study.
Safety was evaluated through physical examinations, clinical laboratory tests, and monitoring of adverse events. Additional safety assessments were done as per standard of care and/or when medically indicated.
Assessment of clinical benefit was performed throughout the study using local standard of care as determined by the Investigator to determine continued study participation and treatment with eltrombopag. Only subjects considered by the Investigator to be receiving clinical benefit without unacceptable toxicity may continue on study treatment. Once treatment with eltrombopag was permanently discontinued, the subject would attend the follow-up visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort A (Myelodysplastic syndrome (MDS)/ Acute myeloid leukemia (AML) adult subjects) | Experimental | Drug: Eltrombopag (ELT)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. |
| Cohort B (Idiopathic thrombocytopenic purpura (ITP) adult subjects) | Experimental | Drug: Eltrombopag (ELT)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. |
| Cohort C (Idiopathic thrombocytopenic purpura (ITP) pediatric subjects) | Experimental | Drug: Eltrombopag (ELT)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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Eltrombopag (ELT)Also: SB-497115Subjects 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. | Drug | 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) |
Primary outcome
Number of Participants With Adverse Events (AEs)
Time frame: From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
13 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Eltrombopag (ELT) in people with Thrombocytopaenia, run by Novartis Pharmaceuticals at 13 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Greece, Belgium, China, France, Hong Kong and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) (time frame: From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Dec 2022; AzTrials last refreshed its copy on 1 Nov 2026.