Specified Drug-use Surveillance of Fabhalta Capsules

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria

• Registration Number: NCT06606314
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.

Detailed Description

The observation period will be 48 weeks after the start of treatment with Fabhalta.

For patients in whom treatment with Fabhalta is discontinued within 48 weeks after the start of the treatment, adverse events occurring by the last day of the treatment + 30 days and concomitant drugs will be monitored and recorded in CRFs.

Study Timeline

• Study First Submitted: 2024-09-18
• Study First Submitted QC: 2024-09-18
• Study Start: 2024-09-19
• Study First Posted: 2024-09-23
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-12
• Last Update Posted: 2025-01-14
• Primary Completion: 2028-09-30
• Study Completion: 2028-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

All patients who received Fabhalta.

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Exclusion Criteria

Patients receiving Fabhalta for an unapproved indication under the Clinical Trials Act or GCP.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Occurrence status of infections.	48weeks	Type, incidence, seriousness, severity, clinical course, factors affecting onset (status of vaccination, name of vaccine administered, complications, baseline white blood cell count/neutrophil count, etc.), and causative organism (including serotype) of adverse events/adverse drug reactions of infections during the treatment period.

Secondary Outcome Measures

Name	Time	Method
Occurrence status of serious hemolysis after discontinuation of Fabhalta.	48weeks	Number of patients with serious hemolysis after discontinuation of Fabhalta.
Occurrence status of adverse events and adverse drug reactions.	48weeks	Incidence of adverse events and adverse drug reactions during the treatment period.
Incidence of breakthrough hemolysis and change of hemolysis-related markers and status of red blood cell transfusion-free.	48weeks	Incidence of breakthrough hemolysis per year and change over time in hemolysis-related markers (LDH, Hb, etc.) and status of red blood cell transfusion-free up to Week 48.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇯🇵 Osaka, Japan