Immune ThrombocytopeniaNCT07587723
A Real-world Study of Treatment Patterns and Outcomes in Primary Immune Thrombocytopenia Patients Treated With Eltrombopag
An observational study of Immune Thrombocytopenia, run by Novartis Pharmaceuticals at 1 site in Switzerland.
Trial summary
This study aims to describe the treatment patterns and clinical outcomes of treatment with eltrombopag in primary immune thrombocytopenia (ITP) patients treated with prior corticosteroid therapy. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Immune Thrombocytopenia
- How many people
- 150 participants
- Where
- 1 siteSwitzerland
- Study dates
- 25 Jun 2026to 29 Sep 2028
- Phase
- Observational
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients diagnosed with primary ITP who were treated with eltrombopag following corticosteroid treatment.
Who may be able to join
- HCP inclusion criteria:
- A licensed HCP (e.g., internal medicine specialist, hematologist, hematologist-oncologist or immunologist etc.) involved in the management of adult patients with primary ITP.
- Actively involved in treatment decision-making for primary ITP patients at the time of the study.
- Have a minimum of 3 years of experience managing patients with primary ITP.
- Currently, managing an average of at least 2 adult primary ITP patients per month, including those treated with eltrombopag.
- Able and willing to complete a web-based survey (approximately 60 to 70 minutes) and abstract data from 2 eligible patient charts.
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- Practicing in one of the study countries (US, UK, or Germany).
- Have access to a computer or mobile device with internet connectivity to complete the survey.
- Not listed as inactive or retired in the panel database.
- Patient-level inclusion criteria:
- Confirmed diagnosis of primary ITP.
- Aged ≥18 years at the time of diagnosis.
- Received first-line corticosteroid therapy (with or without intravenous immune globulin [IVIg]).
- Initiated eltrombopag treatment (with or without corticosteroids) as first non-steroidal ITP treatment between 01 January 2020 and 31 December 2024.
- Have at least 6 months of follow-up data available after the index date (unless deceased within that period).
Who cannot join
- HCP exclusion criteria:
- Currently employed by a pharmaceutical company or regulatory authority involved in the approval or marketing of treatments for primary ITP.
- Unable to provide data for patients who meet the study's inclusion criteria.
- Not currently managing patients with primary ITP or are not involved in treatment decisions for these patients.
- Patient-level exclusion criteria:
- 1. Diagnosis of Evans syndrome, secondary ITP, or non-immune thrombocytopenia.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
About this study
Official title: Real-world Treatment Patterns and Outcomes of Eltrombopag in Patients With Primary Immune Thrombocytopenia
This study aims to describe the treatment patterns and clinical outcomes of treatment with eltrombopag in primary immune thrombocytopenia (ITP) patients treated with prior corticosteroid therapy.
This study will use secondary data provided by health care professionals (HCPs) for a sample of primary ITP patients, and primary data from a structured HCP survey. Data will be collected from HCPs working in various clinical centers across the United States (US), United Kingdom (UK), and Germany.
- Conditions
- Immune Thrombocytopenia
- Keywords
- Primary Immune Thrombocytopenia, Eltrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Eltrombopag Cohort | Adult patients diagnosed with primary ITP who initiated eltrombopag treatment between 01 January 2020 and 31 December 2024 following corticosteroid treatment. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and Percentage of Patients Achieving an Initial Platelet Response Within 12 Weeks of Start of Treatment
Initial platelet response is defined as a platelet count ≥50 × 10^9/L.
Measured: 12 weeks
Secondary outcomes (29)
Number and Percentage of Patients Achieving an Initial Complete Platelet Response Within 12 Weeks of Start of Treatment
Complete platelet response is defined as a platelet count of ≥100 × 10^9/L.
Measured: 12 weeks
Number and Percentage of Patients Achieving a Partial Platelet Response Within 12 Weeks of Start of Treatment
Partial platelet response is defined as a platelet count of ≥30 × 10^9/L.
Measured: 12 weeks
Number and Percentage of Patients With No or Suboptimal Platelet Response Within 12 Weeks of Start of Treatment
No or suboptimal platelet response is defined as a platelet count of <30 × 10^9/L.
Measured: 12 weeks
Number and Percentage of Patients Achieving Clinically Meaningful Platelet Response Thresholds Within 12 Weeks of Start of Treatment
Clinically meaningful platelet response thresholds as indicated by a platelet count increase from a pretreatment value <30 × 10^9/L, and/or documentation of improvement in clinical symptoms such as bleeding.
Measured: 12 weeks
Time to Platelet Response
Platelet response is defined as a platelet count ≥50 ×10^9/L.
Measured: Up to 4 years and 6 months after treatment initiation
Platelet Counts at Eltrombopag Treatment Initiation
Measured: Day 1
Platelet Counts at Eltrombopag Treatment Discontinuation
Measured: Up to 4 years and 6 months after treatment initiation
Number and Percentage of Patients With Bleeding Symptoms During Eltrombopag Treatment
Measured: Up to 4 years and 6 months after treatment initiation
Duration Between ITP Diagnosis and Eltrombopag Treatment Initiation
Measured: Up to 6 years prior to Day 1 of treatment
Number and Percentage of Patients With Concomitant Corticosteroid Treatment During Eltrombopag Treatment
Measured: Up to 4 years and 6 months after treatment initiation
Number and Percentage of Patients With Concomitant Corticosteroid Treatment Withdrawn During Eltrombopag Treatment
Measured: Up to 4 years and 6 months after treatment initiation
Time to Eltrombopag Treatment Discontinuation
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by Reason for Eltrombopag Initiation
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by Reason for Eltrombopag Discontinuation
Measured: Up to 4 years and 6 months after treatment initiation
Baseline Demographics
Measured: Up to 6 years prior to Day 1 of treatment
Number of Prior Corticosteroid Treatment Courses
Measured: Up to 6 years prior to Day 1 of treatment
Duration of Prior Corticosteroid Treatment Courses
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by Comorbidities
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by HCP-reported Reason for Eltrombopag Initiation
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by HCP-reported Factors Influencing Selection of Eltrombopag as Second-line Treatment
Factors influencing selection include platelet count, bleeding symptoms, comorbidities, prior treatment response, ease of administration, safety profile, guideline recommendation, and other.
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by Location of Eltrombopag Prescription
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by Type of Provider of Eltrombopag Treatment Initiation
Providers include general practitioner, immunologist, hematologist, internal medicine specialist, and other.
Measured: Day 1
Number of Patients Categorized by Typical Duration Between ITP Diagnosis and Eltrombopag Initiation as Reported by the HCP
Typical duration between diagnosis and treatment initiation categorized as <3 months, 3-6 months, 6-12 months, >12 months.
Measured: Up to 6 years prior to Day 1 of treatment
Number of Patients by Corticosteroid Use During Eltrombopag Treatment as Reported by the HCP
Corticosteroid use categorized as never, rarely, sometimes, often, or always.
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by HCP-reported Indicators for Treatment Escalation or Discontinuation
Indicators for treatment escalation or discontinuation include lack of platelet response, bleeding symptoms, adverse events, stable response, patient preference, and other.
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by Common Reasons for Eltrombopag Discontinuation as Reported by the HCP
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by the HCP-reported Treatment Success With Eltrombopag
Treatment success categorized as rapid platelet increase, sustained response, bleeding control, minimal side effects, improved quality of life, and other.
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by Patient Characteristics Associated With Better Eltrombopag Response as Reported by the HCP
Patient characteristics include younger age, no comorbidities, early initiation post-diagnosis, prior corticosteroid response, and other
Measured: Up to 4 years and 6 months after treatment initiation
Number of Patients by Patient Characteristics Associated With Poor Eltrombopag Response as Reported by the HCP
Patient characteristics include multiple comorbidities, delayed initiation, prior treatment failure, and other.
Measured: Up to 4 years and 6 months after treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start25 Jun 2026
- Primary completion29 Sep 2028 (estimated)
- Study completion29 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 150 participants (estimated)
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
- NCT07587723
- Sponsor's protocol ID
- CVAY736I12002
- Status last verified
- Oct 2026
- First submitted
- 8 May 2026
- Last updated
- 15 Oct 2026
- Results posted
- No
- Individual data sharing
- No