Ignite Creation Date:
2024-05-19 @ 5:34 PM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06411626
Status:
RECRUITING
Last Update Posted:
2024-07-03
First Post:
2024-05-08
Brief Title:
Home Reported Outcomes in PNH
Sponsor:
Novartis Pharmaceuticals
Organization:
Novartis
Study Overview
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
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 andor claims data
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None