Home Reported Outcomes in PNH

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Other: PNH-relevant therapies

• Registration Number: NCT06411626
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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.

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 Timeline

• Study First Submitted: 2024-05-08
• Study First Submitted QC: 2024-05-08
• Study First Posted: 2024-05-13
• Study Start: 2024-06-11
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-11
• Last Update Posted: 2025-01-14
• Primary Completion: 2025-05-15
• Study Completion: 2025-05-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 128

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Participants with PNH	PNH-relevant therapies	Participants with PNH

Primary Outcome Measures

Name	Time	Method
PNH symptom burden	Baseline, month 6	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.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Novartis Investigative Site; 🇺🇸 East Hanover, New Jersey, United States