NCT06606314

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P50-P75 for all trials

Timeline
29mo left

Started Sep 2024

Longer than P75 for all trials

Geographic Reach
1 country

92 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress41%
Sep 2024Sep 2028

First Submitted

Initial submission to the registry

September 18, 2024

Completed
Same day until next milestone

Study Start

First participant enrolled

September 18, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 23, 2024

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2028

Last Updated

April 13, 2026

Status Verified

April 1, 2026

Enrollment Period

4 years

First QC Date

September 18, 2024

Last Update Submit

April 7, 2026

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

paroxysmal nocturnal hemoglobinuriaPNHFabhalta

Outcome Measures

Primary Outcomes (1)

  • Occurrence status of infections.

    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 Outcomes (3)

  • Occurrence status of serious hemolysis after discontinuation of Fabhalta.

    48weeks

  • Occurrence status of adverse events and adverse drug reactions.

    48weeks

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

    48weeks

Eligibility Criteria

Age0 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

All patients who received Fabhalta.

You may qualify if:

  • All patients who received Fabhalta.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (92)

Novartis Investigative Site

Handa, Aichi-ken, 475-0817, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 453-8511, Japan

RECRUITING

Novartis Investigative Site

Nagoya, Aichi-ken, 466-8650, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Okazaki, Aichi-ken, 444-8553, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Toyohashi, Aichi-ken, 441-8570, Japan

RECRUITING

Novartis Investigative Site

Toyota, Aichi-ken, 470-0343, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Toyota, Aichi-ken, 471-8513, Japan

RECRUITING

Novartis Investigative Site

Hachinohe, Aomori, 039-1104, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Chiba, Chiba, 2608677, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Funabashi, Chiba, 273-8556, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kashiwa, Chiba, 277-8567, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Narita, Chiba, 286-8523, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Matsuyama, Ehime, 790-8524, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Fukuoka, Fukuoka, 810-8563, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kurume, Fukuoka, 830-8543, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Aizu-Wakamatsu, Fukushima, 9693482, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kohriyama, Fukushima, 963-8022, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Maebashi, Gunma, 371-0811, Japan

COMPLETED

Novartis Investigative Site

Fukuyama, Hiroshima, 720-0001, Japan

RECRUITING

Novartis Investigative Site

Hakodate, Hokkaido, 041-0821, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 003-0006, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 064 0804, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Amagasaki, Hyōgo, 660-8511, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ashiya, Hyōgo, 659-0012, Japan

COMPLETED

Novartis Investigative Site

Himeji, Hyōgo, 670-8540, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, 6500047, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, 651-0072, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Nishinomiya, Hyōgo, 662-0918, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Nishinomiya, Hyōgo, 663 8501, Japan

COMPLETED

Novartis Investigative Site

Tsukuba, Ibaraki, 300-2622, Japan

COMPLETED

Novartis Investigative Site

Tsukuba, Ibaraki, 3058576, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Tsukuba, Ibaraki, Ibaraki, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Morioka, Iwate, 020 0066, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Sakaidechō, Kagawa-ken, 762-8550, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kanoya, Kagoshima-ken, 893-0024, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Isehara, Kanagawa, 259-1193, Japan

COMPLETED

Novartis Investigative Site

Isehara, Kanagawa, 259-1193, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Yokohama, Kanagawa, 232-0024, Japan

COMPLETED

Novartis Investigative Site

Kyoto, Kyoto, 603-8151, Japan

COMPLETED

Novartis Investigative Site

Uji, Kyoto, 611-0042, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ōsaki, Miyagi, 989-6183, Japan

COMPLETED

Novartis Investigative Site

Miyazaki, Miyazaki, 880-8510, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Nobeoka, Miyazaki, 882-0835, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Matsumoto, Nagano, 3908621, Japan

RECRUITING

Novartis Investigative Site

Nagano, Nagano, 3808582, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Suwa, Nagano, 392-8510, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ōmura, Nagasaki, 856-8562, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ikoma, Nara, 630-0293, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Nagaoka, Niigata, 940-2085, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Beppu, Oita Prefecture, 874-0840, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Uruma, Okinawa, 904-2293, Japan

RECRUITING

Novartis Investigative Site

Habikino, Osaka, 583-0872, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Izumi, Osaka, 594-0071, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Moriguchi, Osaka, 570-8540, Japan

COMPLETED

Novartis Investigative Site

Suita, Osaka, 565-0871, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Saga, Saga-ken, 849-8501, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Fukaya, Saitama, 366-0052, Japan

COMPLETED

Novartis Investigative Site

Kawagoe, Saitama, 3508550, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Tokorozawa, Saitama, 359-0037, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Hikone, Shiga, 522-0057, Japan

RECRUITING

Novartis Investigative Site

Ōtsu, Shiga, 520-0804, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Rittō, Shiga, 520-3046, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Iwata, Shizuoka, 438-8550, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shimotsuga Gun, Tochigi, 321-0293, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shimotsuke, Tochigi, 329-0498, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Utsunomiya, Tochigi, 321-0974, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 1138431, Japan

COMPLETED

Novartis Investigative Site

Higashimurayama, Tokyo, 189-8511, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Itabashi Ku, Tokyo, 173 8606, Japan

COMPLETED

Novartis Investigative Site

Itabashi-ku, Tokyo, 1738610, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Meguro City, Tokyo, 153-8581, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Minato-ku, Tokyo, 105-8471, Japan

COMPLETED

Novartis Investigative Site

Nerima City, Tokyo, 113-0033, Japan

COMPLETED

Novartis Investigative Site

Nerima City, Tokyo, 179-0072, Japan

COMPLETED

Novartis Investigative Site

Ōta-ku, Tokyo, 143 8541, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shibuya City, Tokyo, 150-8935, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shinagawa Ku, Tokyo, 1418625, Japan

COMPLETED

Novartis Investigative Site

Shinagawa-ku, Tokyo, 1428666, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, 160-0023, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Toyama, Toyama, 930-0859, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Tanabe, Wakayama, 646-8588, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kofu, Yamanashi, 400-8506, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Fukushima, 960-1295, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ishikawa, 9208641, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kagoshima, 892-0853, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kumamoto, 860-8556, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kyoto, 604-8845, Japan

RECRUITING

Novartis Investigative Site

Kyoto, 6068507, Japan

RECRUITING

Novartis Investigative Site

Niigata, 9518520, Japan

COMPLETED

Novartis Investigative Site

Okayama, 7008558, Japan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Osaka, 542-0081, Japan

COMPLETED

Novartis Investigative Site

Shizuoka, 422-8527, Japan

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+81337978748novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
OTHER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 18, 2024

First Posted

September 23, 2024

Study Start

September 18, 2024

Primary Completion (Estimated)

September 30, 2028

Study Completion (Estimated)

September 30, 2028

Last Updated

April 13, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

JP(92)