Recruiting

Observational Study

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06411626

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

PNH-relevant therapies

Study Details

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

Study ID

NCT06411626

Start date

Jun 11, 2024

Status verified date

Aug, 2025

Completion date

Jun 15, 2026

Anticipated

Primary completion date

Jun 15, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

128 participants

Anticipated

Interventions and Outcome Measures

Arms

Participants with PNH

Participants with PNH

Interventions

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.

Primary outcome measure

  • PNH symptom burden [ Time Frame: Baseline, month 6 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

+41613241111novartis.email@novartis.com

Locations

Novartis Investigative Site

Recruiting

East Hanover, New Jersey, United States, 07936

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Aug 27, 2025

Last verified

Aug, 2025

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2025-08-27.