Specified Drug-use Surveillance of Fabhalta Capsules

NCT06606314 | Active Not Recruiting

• 1 other identifier: CLNP023C11401
• Study Type: observational
• Target: 132
• Locations: 1 country
• Sites: 91

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

paroxysmal nocturnal hemoglobinuria; PNH; Fabhalta

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

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (91)

Novartis Investigative Site

Handa, Aichi-ken, 475-0817, Japan

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Novartis Investigative Site

Nagoya, Aichi-ken, 453-8511, Japan

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Nagoya, Aichi-ken, 466-8650, Japan

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Okazaki, Aichi-ken, 444-8553, Japan

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Toyohashi, Aichi-ken, 441-8570, Japan

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Toyota, Aichi-ken, 470-0343, Japan

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Toyota, Aichi-ken, 471-8513, Japan

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Novartis Investigative Site

Hachinohe, Aomori, 0391104, Japan

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Chiba, Chiba, 2608677, Japan

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Funabashi, Chiba, 273-8556, Japan

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Kashiwa, Chiba, 277-8567, Japan

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Narita, Chiba, 286-8523, Japan

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Matsuyama, Ehime, 790-8524, Japan

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Fukuoka, Fukuoka, 810-8563, Japan

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Kurume, Fukuoka, 830-8543, Japan

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Aizu-Wakamatsu, Fukushima, 9693482, Japan

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Kohriyama, Fukushima, 963-8022, Japan

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Maebashi, Gunma, 371-0811, Japan

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Fukuyama, Hiroshima, 720-0001, Japan

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Hakodate, Hokkaido, 0410821, Japan

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Sapporo, Hokkaido, 0030006, Japan

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Sapporo, Hokkaido, 0640804, Japan

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Amagasaki, Hyōgo, 660-8511, Japan

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Ashiya, Hyōgo, 659-0012, Japan

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Himeji, Hyōgo, 670-8540, Japan

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Kobe, Hyōgo, 6500047, Japan

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Kobe, Hyōgo, 651-0072, Japan

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Nishinomiya, Hyōgo, 662-0918, Japan

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Nishinomiya, Hyōgo, 6638501, Japan

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Tsukuba, Ibaraki, 300-2622, Japan

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Tsukuba, Ibaraki, 3058576, Japan

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Tsukuba, Ibaraki, Ibaraki, Japan

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Morioka, Iwate, 0200066, Japan

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Sakaidechō, Kagawa-ken, 762-8550, Japan

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Kanoya, Kagoshima-ken, 893-0024, Japan

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Isehara, Kanagawa, 259-1193, Japan

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Yokohama, Kanagawa, 232-0024, Japan

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Kumamoto, Kumamoto, 860-8556, Japan

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Kyoto, Kyoto, 603-8151, Japan

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Uji, Kyoto, 611-0042, Japan

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Ōsaki, Miyagi, 989-6183, Japan

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Miyazaki, Miyazaki, 880-8510, Japan

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Nobeoka, Miyazaki, 882-0835, Japan

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Matsumoto, Nagano, 3908621, Japan

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Nagano, Nagano, 3808582, Japan

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Suwa, Nagano, 392-8510, Japan

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Ōmura, Nagasaki, 856-8562, Japan

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Ikoma, Nara, 630-0293, Japan

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Nagaoka, Niigata, 940-2085, Japan

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Beppu, Oita Prefecture, 874-0840, Japan

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Uruma, Okinawa, 904-2293, Japan

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Habikino, Osaka, 583-0872, Japan

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Izumi, Osaka, 594-0071, Japan

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Moriguchi, Osaka, 570-8540, Japan

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Suita, Osaka, 565-0871, Japan

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Saga, Saga-ken, 849-8501, Japan

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Fukaya, Saitama, 366-0052, Japan

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Kawagoe, Saitama, 3508550, Japan

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Tokorozawa, Saitama, 359-0037, Japan

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Hikone, Shiga, 522-0057, Japan

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Ōtsu, Shiga, 520-0804, Japan

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Rittō, Shiga, 520-3046, Japan

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Iwata, Shizuoka, 438-8550, Japan

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Shimotsuga Gun, Tochigi, 3210293, Japan

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Shimotsuke, Tochigi, 329-0498, Japan

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Utsunomiya, Tochigi, 321-0974, Japan

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Bunkyo Ku, Tokyo, 1138431, Japan

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Higashimurayama, Tokyo, 189-8511, Japan

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Novartis Investigative Site

Itabashi Ku, Tokyo, 1738606, Japan

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Novartis Investigative Site

Itabashi-ku, Tokyo, 1738610, Japan

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Novartis Investigative Site

Meguro City, Tokyo, 153-8581, Japan

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Minato-ku, Tokyo, 105-8471, Japan

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Nerima City, Tokyo, 113-0033, Japan

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Nerima City, Tokyo, 179-0072, Japan

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Ōta-ku, Tokyo, 1438541, Japan

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Shibuya City, Tokyo, 150-8935, Japan

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Shinagawa Ku, Tokyo, 1418625, Japan

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Shinagawa-ku, Tokyo, 1428666, Japan

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Shinjuku Ku, Tokyo, 160-0023, Japan

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Toyama, Toyama, 930-0859, Japan

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Tanabe, Wakayama, 646-8588, Japan

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Kofu, Yamanashi, 400-8506, Japan

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Fukushima, 9601295, Japan

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Ishikawa, 9208641, Japan

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Kagoshima, 892-0853, Japan

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Kyoto, 604-8845, Japan

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Kyoto, 6068507, Japan

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Novartis Investigative Site

Niigata, 9518520, Japan

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Okayama, 7008558, Japan

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Osaka, 542-0081, Japan

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Novartis Investigative Site

Shizuoka, 422-8527, Japan

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

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: June 16, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

JP (91)