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Immune ThrombocytopeniaNCT07805408

A Real World Study of Treatment Patterns and Effectiveness of Standard of Care in Primary Immune Thrombocytopenia Patients

An observational study of Immune Thrombocytopenia, run by Novartis Pharmaceuticals at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The aim of this study was to describe real-world treatment patterns and standard of care treatment effectiveness and utilization outcomes in adult primary ITP patients indicated for second-line treatment with any prior corticosteroid treatment in real-world treatment settings in the United States (US) identified in an… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Immune Thrombocytopenia
How many people
3,714 participants
Where
1 siteUnited States
Study dates
16 May 2025to 11 Nov 2025
Phase
Observational
Are there results?
None posted in the registry

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

Study population: Adult patients diagnosed with primary ITP from a US medical claim and EHR data source during the study period.

Who may be able to join

  • Evidence of a diagnosis of primary ITP during the study identification period.
  • Evidence of treatment with any allowed second-line treatment during the study identification period after the first primary ITP diagnosis.
  • Evidence of treatment with any allowed first-line treatment prior to index.
  • Aged ≥18 years at index.

Who cannot join

  • Fewer than 12 months continuous enrollment or activity prior to index.
  • Fewer than one inpatient or fewer than two outpatient records during baseline.
  • Evidence of any allowed second-line therapies or splenectomy prior to index.
  • Evidence of diagnosis of Evans syndrome, systemic lupus erythematosus, autoimmune lymphoproliferative syndrome, human immunodeficiency virus (HIV), hepatitis C virus (HCV), Helicobacter pylori, Sjogren's antiphospholipid syndrome prior to index, or active b-cell malignancies (not in remission) in the baseline period.
  • Continuous enrollment end date or death date prior to index.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Novartis

    East Hanover, New Jersey, 07936

Other trials in United States

About this study

Official title: Treatment Patterns and Real World Effectiveness of Standard of Care in Primary Immune Thrombocytopenia (ITP) Patients: A Real-world Retrospective Cohort Study Using the Optum® Market Clarity Database

The aim of this study was to describe real-world treatment patterns and standard of care treatment effectiveness and utilization outcomes in adult primary ITP patients indicated for second-line treatment with any prior corticosteroid treatment in real-world treatment settings in the United States (US) identified in an electronic health record (EHR) and claims database.

Keywords
Primary Immune Thrombocytopenia, Retrospective Cohort, Treatment Patterns, Effectiveness, Healthcare Resource Utilization
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 groups in NCT07805408
Study groupRoleWhat they receive
ITP CohortPrimary ITP patients with prior corticosteroid treatment being treated with second-line treatment.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number and Percentage of Patients With Prescribed Second-line Treatments of Interest

    Second-line treatments of interest, either as monotherapy or in a combination therapy, included:

    • Thrombopoietin Receptor Agonists (TPO-RAs): romiplostim, eltrombopag, avatrombopag.
    • Clusters of differentiation 20 (CD20) inhibitors: rituximab
    • Other therapies: fostamatinib
    • Immunosuppressants: azathioprine, mycophenolate mofetil
    • Splenectomy

    Measured: Up to approximately 8 years

Secondary outcomes (38)

  1. Duration of Patient Follow-up

    Measured: Up to approximately 8 years

  2. Time to Treatment Discontinuation (TTD)

    Measured: Up to approximately 8 years

  3. Time to Next Treatment (TTNT)

    Measured: Up to approximately 8 years

  4. Time to Next Treatment or Initiation of Rescue Treatment

    Measured: Up to approximately 8 years

  5. Number and Percentage of Patients Who Discontinued Treatment Within 1, 3, and 6 Months of Second-line Treatment Initiation

    Measured: 1, 3, and 6 months

  6. Number and Percentage of Patients by First Type of Activity Observed Following Treatment Discontinuation

    Activities included:

    • Switch to new treatment
    • Restart the same second-line treatment
    • Have rescue treatment without second-line treatment
    • First-line treatment without second-line treatment
    • Enter a treatment-free interval
    • End of EHR/claims disenrollment
    • Death

    Measured: 1, 3, and 6 months

  7. Number of Patients Who Entered a Treatment-free Interval

    Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment.

    Measured: Up to approximately 8 years

  8. Duration of Treatment-free Interval

    Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment.

    Measured: Up to approximately 8 years

  9. Number and Percentage of Combination Second-line Treatment Treated Patients Who Underwent Reductions

    Reduction was defined as a discontinuation of at least one therapeutic agent with maintenance of at least one therapeutic agent.

    Measured: Up to approximately 8 years

  10. Number and Percentage of Patients Who Used Add-on, Rescue, or Supportive Treatment During Second-line Treatment

    An add-on was the inclusion of a new, additional second-line treatment ≥28 days after initiation of the initial second-line treatment.

    Measured: Up to approximately 8 years

  11. Time to Initiation of Add-on or Rescue Treatment During Second-line Treatment

    Measured: Up to approximately 8 years

  12. Time to Initiation of Supportive Treatment During Second-line Treatment

    Measured: Up to approximately 8 years

  13. Platelet Counts

    Measured: 1, 3, and 6 months

  14. Number and Percentage of Patients Who Achieved Pre-defined Platelet Counts Within 3 Months of Treatment Initiation

    Measured among patients with at least one platelet count within 3 months of treatment initiation.

    Pre-defined platelet counts were categorized as follows:

    • ≥30 × 10^9/L,
    • ≥ 50 × 10^9/L, and
    • ≥100 × 10^9/L

    Measured: 3 months

  15. Number and Percentage of Patients With no Initial Response to Second-line Treatment

    Measured among patients with at least one platelet count within 3 months of treatment initiation. No initial response was defined as platelet counts <30 × 10^9/L within 3 months of treatment initiation.

    Measured: 3 months

  16. Number and Percentage of Patients Who Achieved Pre-defined Response Levels Within 6 Months of Treatment Initiation

    Measured among patients with at least one platelet count within 6 months of treatment initiation. Response levels were categorized as follows:

    • response (≥ 50 × 10^9/L), and
    • complete response (≥100 × 10^9/L)

    Measured: 6 months

  17. Among Patients With At Least Two Platelet Counts From 3 to 6 Months, Number and Percentage of Patients Who Reached Real-world Durable Response

    Real-world durable response was defined as ≥75% of platelet counts ≥50 × 10^9/L with a minimum of 2 platelet measurements and without other therapies.

    Measured: From 3 to 6 months

  18. Among Patients With At Least Two Platelet Counts From 6 to 12 months, Number and Percentage of Patients Who Reached Real-world Durable Response

    Real-world durable response was defined as ≥75% of platelet counts ≥50 × 10^9/L with a minimum of 2 platelet measurements and without other therapies.

    Measured: From 6 to 12 months

  19. Number and Percentage of Patients With Emergency Department (ED) or Inpatient (IP) Thrombotic Events Within 12 Months After Second-line Treatment Initiation

    Thrombotic events may have included pulmonary embolism, deep vein thrombosis, cerebral venous thrombosis, transient ischemic attack.

    Measured: 12 months

  20. Number of Thrombotic Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

    Measured: 12 months

  21. Number and Percentage of Patients With Infection Events Within 12 Months After Second-line Treatment Initiation

    Measured: 12 months

  22. Number of Infection Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

    Measured: 12 months

  23. Number and Percentage of Patients With Bleeding Events Within 12 Months After Second-line Treatment Initiation

    Measured: 12 months

  24. Number of Bleeding Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

    Measured: 12 months

  25. Overall Survival (OS)

    Measured: 12 months

  26. Number and Percentage of Patients With At Least One Healthcare Visit After Second-line Treatment Initiation

    Healthcare visits included outpatient visits, IP admissions, intensive care unit (ICU) admissions, and ED visits.

    Measured: 12 months

  27. Number of Healthcare Visits After Second-line Treatment Initiation, per Patient per Year (PPPY)

    Healthcare visits included outpatient visits, IP admissions, ICU admissions, and ED visits.

    Measured: 12 months

  28. Number of Oral Corticosteroid Medication Fills Within 12 Months After Second-line Treatment Initiation, PPPY

    Measured: 12 months

  29. Number of Days With Active Oral Corticosteroid Prescriptions Within 12 Months After Second-line Treatment Initiation, PPPY

    Measured: 12 months

  30. Duration of IP and ICU Admissions Among Patients Who Were Hospitalized After Second-line Treatment Initiation

    Measured: 12 months

  31. Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Failed Treatment

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months. Treatment failure was defined as platelet count <30 × 10^9/L.

    Measured: Up to approximately 8 years

  32. Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Received Rescue Treatment

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to approximately 8 years

  33. Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Initiated a New Second-line Treatment or Added Oral Corticosteroids

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to approximately 8 years

  34. Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Died

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to approximately 8 years

  35. Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Reached the End of the Study Period

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to approximately 8 years

  36. Among Patient Responders, Time From Second-line Treatment Initiation to Response

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to 6 months

  37. Among Patient Responders, Duration of Platelet Response

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.

    Measured: Up to approximately 8 years

  38. Number and Percentage of Patient Responders With Treatment-free Response

    Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months. Treatment-free response was defined as platelet count ≥30 × 10^9/L at 3 months after discontinuing second-line treatment.

    Measured: Up to 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Sep 2026
  2. Study start16 May 2025
  3. Primary completion11 Nov 2025
  4. Study completion11 Nov 2025

Resources

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
3,714 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07805408
Sponsor's protocol ID
CVAY736I12001
Status last verified
Jul 2026
First submitted
1 Sep 2026
Last updated
4 Sep 2026
Results posted
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Immune Thrombocytopenia, run by Novartis Pharmaceuticals at 1 site in United States.

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 4 inclusion and 5 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 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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 and Percentage of Patients With Prescribed Second-line Treatments of Interest (time frame: Up to approximately 8 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07805408.

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 4 Sep 2026.

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