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NCT Number: NCT06606314

Specified Drug-use Surveillance of Fabhalta Capsules

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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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

0 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis Investigative Site, Handa, Aichi-ken, Japan

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

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

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.

Treatment and study plan

Primary outcomes

  1. Occurrence status of infections.

    Time frame: 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 outcomes

  1. Occurrence status of serious hemolysis after discontinuation of Fabhalta.

    Time frame: 48weeks

    Number of patients with serious hemolysis after discontinuation of Fabhalta.

  2. Occurrence status of adverse events and adverse drug reactions.

    Time frame: 48weeks

    Incidence of adverse events and adverse drug reactions during the treatment period.

  3. Incidence of breakthrough hemolysis and change of hemolysis-related markers and status of red blood cell transfusion-free.

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Sep 23, 2024
Registry last updated
Jun 16, 2026

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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