A Study of Patients' Preferences for Primary Immune Thrombocytopenia Treatment

NCT07786077 | Recruiting

• 1 other identifier: CVAY736Q12001
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to evaluate treatment preferences of patients with Primary Immune Thrombocytopenia (ITP) in the United States (US) by conducting a preference elicitation survey.

Conditions

Primary Immune Thrombocytopenia

Keywords

Primary Immune Thrombocytopenia; ITP

Outcome Measures

Primary Outcomes (3)

• Preference Weights for Treatment Attributes

  MDT will be used to elicit trade-offs that participants with ITP are willing to make between multiple treatment attributes. Preference weights will be estimated for each treatment attribute included in the MDT. Higher preference weights indicate treatment attributes that have a stronger influence on preferences, while lower weights indicate treatment attributes that have a weaker influence.

  1 Day

• Maximum Acceptable Risk (MAR) and Minimum Acceptable Benefit (MAB)

  To evaluate the benefit-risk trade-offs that participants are willing to make, marginal rates of substitutions (MRS) will be calculated based on the Dirichlet model. MRS will be represented as either MAR or MAB. * MAR measures the median maximum level of risk that participants are willing to tolerate in certain treatment attributes in return for a specified improvement in other treatment attributes. * MAB measures the median minimum level of benefit required in certain treatment attributes for participants to tolerate deteriorations in other treatment attributes.

  1 Day

• Differences in Preference Weights Associated With Personal Characteristics

  Preference weights associated with personal characteristics will be assessed using covariate-adjusted Dirichlet regression. Personal characteristics include sociodemographic, clinical characteristics such as race/ethnicity, living location, employment status, level of education, clinical setting usually treated in, ITP symptoms, and treatment experience, as well as other characteristics such as participants' risk tolerance level, health literacy and numeracy level.

  1 Day

Study Arms (1)

ITP Cohort

Adults with ITP in the US who are being treated in either 1L or 2L settings.

Eligibility Criteria

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

Study Population

Adults receiving 1L or 2L treatment for ITP who reside in the US.

You may qualify if:

• Currently living in the US.
• Aged 18 years and older on the day of providing informed consent.
• A diagnosis of primary ITP:
• a. For participants receiving first-line (1L) treatment for ITP: i. Diagnosed with primary ITP and started 1L corticosteroids within 3 months of diagnosis ii. Responded to 1L corticosteroid treatment \[with or without intravenous immunoglobulin (IVIg)\] b. For participants receiving second-line (2L) treatment for ITP: i. Assessed as needing or are taking thrombopoietin receptor agonist (TPO-RA) treatment for 2L treatment ii. Has previously been treated with a 1L corticosteroid (with or without IVIg) with insufficient response or relapse

You may not qualify if:

• For participants receiving 1L treatment for ITP only: Previous or current use of treatments different from corticosteroids and/or IVIg/anti-D immunoglobulin.
• For participants receiving 2L ITP treatment: Previous or current use of treatments different from corticosteroids, IVIg/anti-D immunoglobulin or one TPO-RA (i.e. patients who have used any treatment in a third-line setting are ineligible).

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2026
• First Posted: August 25, 2026
• Study Start: December 8, 2025
• Primary Completion (Estimated): December 15, 2026
• Study Completion (Estimated): December 15, 2026
• Last Updated: August 25, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)