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NCT Number: NCT06411626

Home Reported Outcomes in PNH

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.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site

East Hanover, New Jersey, 07936, United States

Location status: Recruiting

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

PNH-relevant therapies

Other

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.

Primary outcomes

  1. PNH symptom burden

    Time frame: 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 outcomes

  1. Number of participants by treatment utilization and management of potential flare events

    Time frame: Up to 6 months

    Treatment utilization and management of potential flare events tracked during the study period

  2. Number of participants by reported treatment use and treatment switching

    Time frame: Up to 6 months

    HRO-reported treatment use and treatment switching throughout the study duration, triangulated with symptoms reported throughout the study duration

  3. Health-related quality of life (HRQoL)

    Time frame: up to 6 months

    HRQoL HRO tracking data:

    • Mood & behavioral health
    • Productivity
    • Reasons for discontinuing or changing a treatment

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Novartis Pharmaceuticals

CONTACT

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 13, 2024
Registry last updated
Aug 27, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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