Monitoring the use of Fabhalta Capsules in patients with Paroxysmal Nocturnal Hemoglobinuria

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

This study is tracking how well Fabhalta capsules work and their safety for people with Paroxysmal Nocturnal Hemoglobinuria over 48 weeks.

Quick facts

• Study type: Observational
• Enrollment: 100 (estimated)
• Ages: 0 Years to 100 Years
• Sex: All
• Sponsor: Novartis (Industry-sponsored)
• Locations: 92 sites (Handa, Aichi-ken and 91 other locations)
• Trial ID: NCT06606314 on ClinicalTrials.gov

What this trial studies

This observational study aims to monitor the use of Fabhalta capsules in patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH) over a period of 48 weeks. It involves a multicenter approach where data will be collected on adverse events and concomitant medications for patients receiving Fabhalta. The study will track patients from the start of treatment and continue monitoring for 30 days after treatment discontinuation. The goal is to gather comprehensive safety data on the drug's use in a real-world setting.

Who should consider this trial

Good fit: Ideal candidates for this study are patients who have been prescribed Fabhalta for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

Not a fit: Patients receiving Fabhalta for unapproved indications will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the safety and effectiveness of Fabhalta for patients with PNH.

How similar studies have performed: While this study is observational and focuses on drug-use surveillance, similar studies have successfully monitored drug safety and efficacy in other conditions.

Eligibility criteria

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.

Where this trial is running

Handa, Aichi-ken and 91 other locations

• Novartis Investigative Site — Handa, Aichi-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Nagoya, Aichi-ken, Japan (Recruiting)
• Novartis Investigative Site — Nagoya, Aichi-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Okazaki, Aichi-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Toyohashi, Aichi-ken, Japan (Recruiting)
• Novartis Investigative Site — Toyota, Aichi-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Toyota, Aichi-ken, Japan (Recruiting)
• Novartis Investigative Site — Hachinohe, Aomori, Japan (Active_not_recruiting)
• Novartis Investigative Site — Chiba, Chiba, Japan (Active_not_recruiting)
• Novartis Investigative Site — Funabashi, Chiba, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kashiwa, Chiba, Japan (Active_not_recruiting)
• Novartis Investigative Site — Narita, Chiba, Japan (Active_not_recruiting)
• Novartis Investigative Site — Matsuyama, Ehime, Japan (Active_not_recruiting)
• Novartis Investigative Site — Fukuoka, Fukuoka, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kurume, Fukuoka, Japan (Active_not_recruiting)
• Novartis Investigative Site — Aizu-Wakamatsu, Fukushima, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kohriyama, Fukushima, Japan (Active_not_recruiting)
• Novartis Investigative Site — Maebashi, Gunma, Japan (Completed)
• Novartis Investigative Site — Fukuyama, Hiroshima, Japan (Recruiting)
• Novartis Investigative Site — Hakodate, Hokkaido, Japan (Active_not_recruiting)
• Novartis Investigative Site — Sapporo, Hokkaido, Japan (Active_not_recruiting)
• Novartis Investigative Site — Sapporo, Hokkaido, Japan (Active_not_recruiting)
• Novartis Investigative Site — Amagasaki, Hyōgo, Japan (Active_not_recruiting)
• Novartis Investigative Site — Ashiya, Hyōgo, Japan (Completed)
• Novartis Investigative Site — Himeji, Hyōgo, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kobe, Hyōgo, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kobe, Hyōgo, Japan (Active_not_recruiting)
• Novartis Investigative Site — Nishinomiya, Hyōgo, Japan (Active_not_recruiting)
• Novartis Investigative Site — Nishinomiya, Hyōgo, Japan (Completed)
• Novartis Investigative Site — Tsukuba, Ibaraki, Japan (Completed)
• Novartis Investigative Site — Tsukuba, Ibaraki, Japan (Active_not_recruiting)
• Novartis Investigative Site — Tsukuba, Ibaraki, Japan (Active_not_recruiting)
• Novartis Investigative Site — Morioka, Iwate, Japan (Active_not_recruiting)
• Novartis Investigative Site — Sakaidechō, Kagawa-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Kanoya, Kagoshima-ken, Japan (Active_not_recruiting)
• Novartis Investigative Site — Isehara, Kanagawa, Japan (Completed)
• Novartis Investigative Site — Isehara, Kanagawa, Japan (Active_not_recruiting)
• Novartis Investigative Site — Yokohama, Kanagawa, Japan (Completed)
• Novartis Investigative Site — Kyoto, Kyoto, Japan (Completed)
• Novartis Investigative Site — Uji, Kyoto, Japan (Active_not_recruiting)
• Novartis Investigative Site — Ōsaki, Miyagi, Japan (Completed)
• Novartis Investigative Site — Miyazaki, Miyazaki, Japan (Active_not_recruiting)
• Novartis Investigative Site — Nobeoka, Miyazaki, Japan (Active_not_recruiting)
• Novartis Investigative Site — Matsumoto, Nagano, Japan (Recruiting)
• Novartis Investigative Site — Nagano, Nagano, Japan (Active_not_recruiting)
• Novartis Investigative Site — Suwa, Nagano, Japan (Active_not_recruiting)
• Novartis Investigative Site — Ōmura, Nagasaki, Japan (Active_not_recruiting)
• Novartis Investigative Site — Ikoma, Nara, Japan (Active_not_recruiting)
• Novartis Investigative Site — Nagaoka, Niigata, Japan (Active_not_recruiting)
• Novartis Investigative Site — Beppu, Oita Prefecture, Japan (Active_not_recruiting)

Study contacts

• Study coordinator: Novartis Pharmaceuticals

Conditions Paroxysmal Nocturnal Hemoglobinuria; paroxysmal nocturnal hemoglobinuria; PNH; Fabhalta