Home Reported Outcomes in PNH

Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US

The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Other: PNH-relevant therapies

Detailed Description

The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid recruitment method of clinic referrals and community referrals will be employed to identify participants, who will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs, with the possibility of integrating electronic health record (EHR) and/or claims data.

• Study Type: Observational
• Enrollment (Estimated): 128

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 Investigative Site

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Individuals aged 18+ who have been diagnosed with PNH, regardless of symptom or treatment history, and are US-based with a proficient understanding of and ability to read the English language.

Description

Inclusion Criteria:

Study participants eligible for inclusion in this study must meet all of the following criteria:

• Aged 18 or older
• US-based with a proficient understanding of and ability to read the English language
• Any patient with a diagnosis of PNH, regardless of symptom or treatment history

Exclusion Criteria:

Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Participants with PNH	Other: PNH-relevant therapies; This is an observational study. There is no treatment allocation. The decision to initiate PNH-relevant therapies (such as eculizumab, ravulizumab, pegcetacoplan, iptacopan, and others) will be based solely on clinical judgement.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PNH symptom burden	Primary endpoint will be measured through longitudinal Home Reported Outcomes (HRO) tracking of self-selected symptoms at enrollment and associated changes in severity against self-reported baselines.	Baseline, month 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants by treatment utilization and management of potential flare events	Treatment utilization and management of potential flare events tracked during the study period	Up to 6 months
Number of participants by reported treatment use and treatment switching	HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration	Up to 6 months
Health-related quality of life (HRQoL)	HRQoL HRO tracking data: Mood & behavioral health; Productivity; Reasons for discontinuing or changing a treatment	up to 6 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): June 11, 2024
• Primary Completion (Estimated): June 15, 2026
• Study Completion (Estimated): June 15, 2026

Study Registration Dates

• First Submitted: May 8, 2024
• First Submitted That Met QC Criteria: May 8, 2024
• First Posted (Actual): May 13, 2024

Study Record Updates

• Last Update Posted (Estimated): August 27, 2025
• Last Update Submitted That Met QC Criteria: August 26, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: PRO; PNH; Paroxysmal nocturnal hemoglobinuria; patient-reported outcome; real-world data; iptacopan; home reported outcomes; HRO
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Hemic and Lymphatic Diseases; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: CLNP023C1US01

Plan for Individual participant data (IPD)

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