Home Reported Outcomes in PNH

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06411626
• Other study ID #: CLNP023C1US01
• Status: Not yet recruiting
• Start date: June 15, 2024
• Est. completion date: May 15, 2025

Study information

• Verified date: June 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: +41613241111
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 128
• Est. completion date: May 15, 2025
• Est. primary completion date: May 15, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

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.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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