Specified Drug-use Surveillance of Fabhalta Capsules

Specified Drug-use Surveillance of Fabhalta Capsules (Paroxysmal Nocturnal Hemoglobinuria, CLNP023C11401)

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06606314

Enrollment

132

Registered

2024-09-23

Start date

2024-09-18

Completion date

2028-09-30

Last updated

2026-09-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

paroxysmal nocturnal hemoglobinuria, PNH, Fabhalta

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

OTHER

Eligibility

Sex/Gender

ALL

Age

0 Years to 100 Years

Healthy volunteers

Inclusion criteria

All patients who received Fabhalta. ·

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.

Countries

Japan

Contacts

STUDY_DIRECTORNovartis Pharmaceuticals

Novartis Pharmaceuticals

Outcome results