Specified Drug-use Surveillance of Fabhalta Capsules

May 19, 2026 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Active, not recruiting

Conditions

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 Type

Observational

Enrollment (Actual)

132

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
      • Fukushima, Japan, 9601295
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Novartis Investigative Site
      • Kagoshima, Japan, 892-0853
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Novartis Investigative Site
      • Kyoto, Japan, 604-8845
        • Novartis Investigative Site
      • Niigata, Japan, 9518520
        • Novartis Investigative Site
      • Okayama, Japan, 7008558
        • Novartis Investigative Site
      • Osaka, Japan, 542-0081
        • Novartis Investigative Site
      • Shizuoka, Japan, 422-8527
        • Novartis Investigative Site
    • Aichi-ken
      • Handa, Aichi-ken, Japan, 475-0817
        • Novartis Investigative Site
      • Nagoya, Aichi-ken, Japan, 453-8511
        • Novartis Investigative Site
      • Nagoya, Aichi-ken, Japan, 466-8650
        • Novartis Investigative Site
      • Okazaki, Aichi-ken, Japan, 444-8553
        • Novartis Investigative Site
      • Toyohashi, Aichi-ken, Japan, 441-8570
        • Novartis Investigative Site
      • Toyota, Aichi-ken, Japan, 470-0343
        • Novartis Investigative Site
      • Toyota, Aichi-ken, Japan, 471-8513
        • Novartis Investigative Site
    • Aomori
      • Hachinohe, Aomori, Japan, 0391104
        • Novartis Investigative Site
    • Chiba
      • Chiba, Chiba, Japan, 2608677
        • Novartis Investigative Site
      • Funabashi, Chiba, Japan, 273-8556
        • Novartis Investigative Site
      • Kashiwa, Chiba, Japan, 277-8567
        • Novartis Investigative Site
      • Narita, Chiba, Japan, 286-8523
        • Novartis Investigative Site
    • Ehime
      • Matsuyama, Ehime, Japan, 790-8524
        • Novartis Investigative Site
    • Fukuoka
      • Fukuoka, Fukuoka, Japan, 810-8563
        • Novartis Investigative Site
      • Kurume, Fukuoka, Japan, 830-8543
        • Novartis Investigative Site
    • Fukushima
      • Aizu-Wakamatsu, Fukushima, Japan, 9693482
        • Novartis Investigative Site
      • Kohriyama, Fukushima, Japan, 963-8022
        • Novartis Investigative Site
    • Gunma
      • Maebashi, Gunma, Japan, 371-0811
        • Novartis Investigative Site
    • Hiroshima
      • Fukuyama, Hiroshima, Japan, 720-0001
        • Novartis Investigative Site
    • Hokkaido
      • Hakodate, Hokkaido, Japan, 0410821
        • Novartis Investigative Site
      • Sapporo, Hokkaido, Japan, 0030006
        • Novartis Investigative Site
      • Sapporo, Hokkaido, Japan, 0640804
        • Novartis Investigative Site
    • Hyōgo
      • Amagasaki, Hyōgo, Japan, 660-8511
        • Novartis Investigative Site
      • Ashiya, Hyōgo, Japan, 659-0012
        • Novartis Investigative Site
      • Himeji, Hyōgo, Japan, 670-8540
        • Novartis Investigative Site
      • Kobe, Hyōgo, Japan, 6500047
        • Novartis Investigative Site
      • Kobe, Hyōgo, Japan, 651-0072
        • Novartis Investigative Site
      • Nishinomiya, Hyōgo, Japan, 662-0918
        • Novartis Investigative Site
      • Nishinomiya, Hyōgo, Japan, 6638501
        • Novartis Investigative Site
    • Ibaraki
      • Tsukuba, Ibaraki, Japan, 300-2622
        • Novartis Investigative Site
      • Tsukuba, Ibaraki, Japan, 3058576
        • Novartis Investigative Site
      • Tsukuba, Ibaraki, Japan, Ibaraki
        • Novartis Investigative Site
    • Iwate
      • Morioka, Iwate, Japan, 0200066
        • Novartis Investigative Site
    • Kagawa-ken
      • Sakaidechō, Kagawa-ken, Japan, 762-8550
        • Novartis Investigative Site
    • Kagoshima-ken
      • Kanoya, Kagoshima-ken, Japan, 893-0024
        • Novartis Investigative Site
    • Kanagawa
      • Isehara, Kanagawa, Japan, 259-1193
        • Novartis Investigative Site
      • Yokohama, Kanagawa, Japan, 232-0024
        • Novartis Investigative Site
    • Kumamoto
      • Kumamoto, Kumamoto, Japan, 860-8556
        • Novartis Investigative Site
    • Kyoto
      • Kyoto, Kyoto, Japan, 603-8151
        • Novartis Investigative Site
      • Uji, Kyoto, Japan, 611-0042
        • Novartis Investigative Site
    • Miyagi
      • Ōsaki, Miyagi, Japan, 989-6183
        • Novartis Investigative Site
    • Miyazaki
      • Miyazaki, Miyazaki, Japan, 880-8510
        • Novartis Investigative Site
      • Nobeoka, Miyazaki, Japan, 882-0835
        • Novartis Investigative Site
    • Nagano
      • Matsumoto, Nagano, Japan, 3908621
        • Novartis Investigative Site
      • Nagano, Nagano, Japan, 3808582
        • Novartis Investigative Site
      • Suwa, Nagano, Japan, 392-8510
        • Novartis Investigative Site
    • Nagasaki
      • Ōmura, Nagasaki, Japan, 856-8562
        • Novartis Investigative Site
    • Nara
      • Ikoma, Nara, Japan, 630-0293
        • Novartis Investigative Site
    • Niigata
      • Nagaoka, Niigata, Japan, 940-2085
        • Novartis Investigative Site
    • Oita Prefecture
      • Beppu, Oita Prefecture, Japan, 874-0840
        • Novartis Investigative Site
    • Okinawa
      • Uruma, Okinawa, Japan, 904-2293
        • Novartis Investigative Site
    • Osaka
      • Habikino, Osaka, Japan, 583-0872
        • Novartis Investigative Site
      • Izumi, Osaka, Japan, 594-0071
        • Novartis Investigative Site
      • Moriguchi, Osaka, Japan, 570-8540
        • Novartis Investigative Site
      • Suita, Osaka, Japan, 565-0871
        • Novartis Investigative Site
    • Saga-ken
      • Saga, Saga-ken, Japan, 849-8501
        • Novartis Investigative Site
    • Saitama
      • Fukaya, Saitama, Japan, 366-0052
        • Novartis Investigative Site
      • Kawagoe, Saitama, Japan, 3508550
        • Novartis Investigative Site
      • Tokorozawa, Saitama, Japan, 359-0037
        • Novartis Investigative Site
    • Shiga
      • Hikone, Shiga, Japan, 522-0057
        • Novartis Investigative Site
      • Rittō, Shiga, Japan, 520-3046
        • Novartis Investigative Site
      • Ōtsu, Shiga, Japan, 520-0804
        • Novartis Investigative Site
    • Shizuoka
      • Iwata, Shizuoka, Japan, 438-8550
        • Novartis Investigative Site
    • Tochigi
      • Shimotsuga Gun, Tochigi, Japan, 3210293
        • Novartis Investigative Site
      • Shimotsuke, Tochigi, Japan, 329-0498
        • Novartis Investigative Site
      • Utsunomiya, Tochigi, Japan, 321-0974
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo Ku, Tokyo, Japan, 1138431
        • Novartis Investigative Site
      • Higashimurayama, Tokyo, Japan, 189-8511
        • Novartis Investigative Site
      • Itabashi Ku, Tokyo, Japan, 1738606
        • Novartis Investigative Site
      • Itabashi-ku, Tokyo, Japan, 1738610
        • Novartis Investigative Site
      • Meguro City, Tokyo, Japan, 153-8581
        • Novartis Investigative Site
      • Minato-ku, Tokyo, Japan, 105-8471
        • Novartis Investigative Site
      • Nerima City, Tokyo, Japan, 113-0033
        • Novartis Investigative Site
      • Nerima City, Tokyo, Japan, 179-0072
        • Novartis Investigative Site
      • Shibuya City, Tokyo, Japan, 150-8935
        • Novartis Investigative Site
      • Shinagawa Ku, Tokyo, Japan, 1418625
        • Novartis Investigative Site
      • Shinagawa-ku, Tokyo, Japan, 1428666
        • Novartis Investigative Site
      • Shinjuku Ku, Tokyo, Japan, 160-0023
        • Novartis Investigative Site
      • Ōta-ku, Tokyo, Japan, 1438541
        • Novartis Investigative Site
    • Toyama
      • Toyama, Toyama, Japan, 930-0859
        • Novartis Investigative Site
    • Wakayama
      • Tanabe, Wakayama, Japan, 646-8588
        • Novartis Investigative Site
    • Yamanashi
      • Kofu, Yamanashi, Japan, 400-8506
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All patients who received Fabhalta.

Description

Inclusion Criteria:

All patients who received Fabhalta.

·

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
48weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurrence status of serious hemolysis after discontinuation of Fabhalta.
Time Frame: 48weeks
Number of patients with serious hemolysis after discontinuation of Fabhalta.
48weeks
Occurrence status of adverse events and adverse drug reactions.
Time Frame: 48weeks
Incidence of adverse events and adverse drug reactions during the treatment period.
48weeks
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.
48weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 18, 2024

Primary Completion (Estimated)

September 30, 2028

Study Completion (Estimated)

September 30, 2028

Study Registration Dates

First Submitted

September 18, 2024

First Submitted That Met QC Criteria

September 18, 2024

First Posted (Actual)

September 23, 2024

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 19, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLNP023C11401

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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