Last Update: Jul 03, 2024
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
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023C1US01
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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. 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.

Paroxysmal Nocturnal Hemoglobinuria
Recruiting
128
Jun 11, 2024
May 15, 2025
All
18 Years - 99 Years (Adult, Older Adult)

Interventions

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.

Eligibility Criteria

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.

Novartis Investigative Site

Recruiting

East Hanover,New Jersey,07936,United States

Worldwide Contacts

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Novartis Pharmaceuticals