Home Reported Outcomes in PNH

NCT06411626 | Recruiting

• 1 other identifier: CLNP023C1US01
• Study Type: observational
• Target: 128
• Locations: 1 country
• Sites: 1

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Paroxysmal nocturnal hemoglobinuria; PNH; home reported outcomes; HRO; patient-reported outcome; PRO; real-world data; iptacopan

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Number of participants by treatment utilization and management of potential flare events

  Up to 6 months

• Number of participants by reported treatment use and treatment switching

  Up to 6 months

• Health-related quality of life (HRQoL)

  up to 6 months

Study Arms (1)

Participants with PNH

Participants with PNH

Other: PNH-relevant therapies

Interventions

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.

Participants with PNH

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis Investigative Site

East Hanover, New Jersey, 07936, United States

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: May 13, 2024
• Study Start: June 11, 2024
• Primary Completion (Estimated): June 15, 2026
• Study Completion (Estimated): June 15, 2026
• Last Updated: August 27, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)