A Study of Patients' Preferences for Primary Immune Thrombocytopenia Treatment

Patients' Preferences for Primary Immune Thrombocytopenia Treatment

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.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia

Detailed Description

This is a non-interventional study to assess treatment preferences from patients with ITP. Data will be collected from participants using a preference elicitation survey.

The study consists of two stages. Stage 1 includes the development of the survey and identification of key treatment attributes. Stage 2 involves administration of the finalized survey, followed by comprehensive analysis of the data to assess treatment preferences.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Recruiting
      • Novartis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

Inclusion criteria:

1. Currently living in the US.
2. Aged 18 years and older on the day of providing informed consent.

3. 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

Exclusion criteria:

1. For participants receiving 1L treatment for ITP only: Previous or current use of treatments different from corticosteroids and/or IVIg/anti-D immunoglobulin.
2. 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).

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): December 8, 2025
• Primary Completion (Estimated): December 15, 2026
• Study Completion (Estimated): December 15, 2026

Study Registration Dates

• First Submitted: August 21, 2026
• First Submitted That Met QC Criteria: August 21, 2026
• First Posted (Actual): August 25, 2026

Study Record Updates

• Last Update Posted (Actual): August 25, 2026
• Last Update Submitted That Met QC Criteria: August 21, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: ITP; Primary Immune Thrombocytopenia
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: CVAY736Q12001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO