Clinical trial NCT06411626: Home Reported Outcomes in PNH
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: Paroxysmal nocturnal hemoglobinuria
PNH
home reported outcomes
HRO
patient-reported outcome
PRO
real-world data
iptacopan
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-05-13
Enrollment: 128
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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.

Primary outcomes:
measure: PNH symptom burden
timeFrame: Baseline, month 6
Secondary outcomes:
measure: Number of participants by treatment utilization and management of potential flare events
timeFrame: Up to 6 months
measure: Number of participants by reported treatment use and treatment switching
timeFrame: Up to 6 months
measure: Health-related quality of life (HRQoL)
timeFrame: up to 6 months
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis Investigative Site
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: