Paroxysmal Nocturnal HemoglobinuriaNCT06606314

Specified Drug-use Surveillance of Fabhalta Capsules

An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 91 sites in Japan.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Trial 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. Read more about this study

Who is it for?: Ages 0 to 100 years
Conditions the study is for: Paroxysmal Nocturnal Hemoglobinuria
How many people: 132 participants
Where: 91 sitesJapan
Study dates: 18 Sep 2024to 30 Sep 2028
Phase: Observational
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 0 to 100 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: All patients who received Fabhalta.

Who may be able to join

All patients who received Fabhalta.
·

Who cannot join

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

Trial locations and contacts

91 sites in 1 country.

City, postcode or hospital

91 sites in 1 country

Japan91 sites

Novartis Investigative Site
Aizu-Wakamatsu, Fukushima, 9693482
Novartis Investigative Site
Amagasaki, Hyōgo, 6608511
Novartis Investigative Site
Ashiya, Hyōgo, 6590012
Novartis Investigative Site
Beppu, Oita Prefecture, 8740840
Novartis Investigative Site
Bunkyo Ku, Tokyo, 1138431
Novartis Investigative Site
Chiba, Chiba, 2608677
Novartis Investigative Site
Fukaya, Saitama, 3660052
Novartis Investigative Site
Fukuoka, Fukuoka, 8108563
Novartis Investigative Site
Fukushima, 9601295
Novartis Investigative Site
Fukuyama, Hiroshima, 7200001
Novartis Investigative Site
Funabashi, Chiba, 2738556
Novartis Investigative Site
Habikino, Osaka, 5830872
Novartis Investigative Site
Hachinohe, Aomori, 0391104
Novartis Investigative Site
Hakodate, Hokkaido, 0418680
Novartis Investigative Site
Handa, Aichi-ken, 4750817
Novartis Investigative Site
Higashimurayama, Tokyo, 1898511
Novartis Investigative Site
Hikone, Shiga, 5220057
Novartis Investigative Site
Himeji, Hyōgo, 6708540
Novartis Investigative Site
Ikoma, Nara, 6300293
Novartis Investigative Site
Isehara, Kanagawa, 2591193
Novartis Investigative Site
Ishikawa, 9208641
Novartis Investigative Site
Itabashi Ku, Tokyo, 1738606
Novartis Investigative Site
Itabashi-ku, Tokyo, 1738610
Novartis Investigative Site
Iwata, Shizuoka, 4388550
Novartis Investigative Site
Izumi, Osaka, 5940071
Novartis Investigative Site
Kagoshima, 8920853
Novartis Investigative Site
Kanoya, Kagoshima-ken, 8930024
Novartis Investigative Site
Kashiwa, Chiba, 2778567
Novartis Investigative Site
Kawagoe, Saitama, 3508550
Novartis Investigative Site
Kobe, Hyōgo, 6500047
Novartis Investigative Site
Kobe, Hyōgo, 6510072
Novartis Investigative Site
Kofu, Yamanashi, 4008506
Novartis Investigative Site
Kohriyama, Fukushima, 9638022
Novartis Investigative Site
Kumamoto, Kumamoto, 8608556
Novartis Investigative Site
Kurume, Fukuoka, 8308543
Novartis Investigative Site
Kyoto, Kyoto, 6038151
Novartis Investigative Site
Kyoto, 6048845
Novartis Investigative Site
Kyoto, 6068507
Novartis Investigative Site
Maebashi, Gunma, 3710811
Novartis Investigative Site
Matsumoto, Nagano, 3908621
Novartis Investigative Site
Matsuyama, Ehime, 7908524
Novartis Investigative Site
Meguro City, Tokyo, 1538581
Novartis Investigative Site
Minato-ku, Tokyo, 1058471
Novartis Investigative Site
Miyazaki, Miyazaki, 8808510
Novartis Investigative Site
Moriguchi, Osaka, 5708540
Novartis Investigative Site
Morioka, Iwate, 0200066
Novartis Investigative Site
Nagano, Nagano, 3808582
Novartis Investigative Site
Nagaoka, Niigata, 9402085
Novartis Investigative Site
Nagoya, Aichi-ken, 4538511
Novartis Investigative Site
Nagoya, Aichi-ken, 4668650
Novartis Investigative Site
Narita, Chiba, 2868523
Novartis Investigative Site
Nerima City, Tokyo, 1130033
Novartis Investigative Site
Nerima City, Tokyo, 1790072
Novartis Investigative Site
Niigata, 9518520
Novartis Investigative Site
Nishinomiya, Hyōgo, 663-8241
Novartis Investigative Site
Nishinomiya, Hyōgo, 6638501
Novartis Investigative Site
Nobeoka, Miyazaki, 8820835
Novartis Investigative Site
Okayama, 7008558
Novartis Investigative Site
Okazaki, Aichi-ken, 4448553
Novartis Investigative Site
Ōmura, Nagasaki, 8568562
Novartis Investigative Site
Osaka, 5420081
Novartis Investigative Site
Ōsaki, Miyagi, 9896183
Novartis Investigative Site
Ōta-ku, Tokyo, 1438541
Novartis Investigative Site
Ōtsu, Shiga, 5200804
Novartis Investigative Site
Rittō, Shiga, 5203046
Novartis Investigative Site
Saga, Saga-ken, 8498501
Novartis Investigative Site
Sakaidechō, Kagawa-ken, 7628550
Novartis Investigative Site
Sapporo, Hokkaido, 0030006
Novartis Investigative Site
Sapporo, Hokkaido, 0640804
Novartis Investigative Site
Shibuya-ku, Tokyo, 1508935
Novartis Investigative Site
Shimotsuga Gun, Tochigi, 3210293
Novartis Investigative Site
Shimotsuke, Tochigi, 3290498
Novartis Investigative Site
Shinagawa Ku, Tokyo, 1418625
Novartis Investigative Site
Shinagawa-ku, Tokyo, 1428666
Novartis Investigative Site
Shinjuku Ku, Tokyo, 1600023
Novartis Investigative Site
Shizuoka, 4228527
Novartis Investigative Site
Suita, Osaka, 5650871
Novartis Investigative Site
Suwa, Nagano, 3928510
Novartis Investigative Site
Tanabe, Wakayama, 6468588
Novartis Investigative Site
Tokorozawa, Saitama, 3590037
Novartis Investigative Site
Toyama, Toyama, 9300859
Novartis Investigative Site
Toyohashi, Aichi-ken, 4418570
Novartis Investigative Site
Toyota, Aichi-ken, 4700343
Novartis Investigative Site
Toyota, Aichi-ken, 4718513
Novartis Investigative Site
Tsukuba, Ibaraki, 3002622
Novartis Investigative Site
Tsukuba, Ibaraki, 3004321
Novartis Investigative Site
Tsukuba, Ibaraki, 3058576
Novartis Investigative Site
Uji, Kyoto, 6110042
Novartis Investigative Site
Uruma, Okinawa, 9042293
Novartis Investigative Site
Utsunomiya, Tochigi, 3210974
Novartis Investigative Site
Yokohama, Kanagawa, 2320024

Other trials in Japan

About this study

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

Read the 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.

Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: paroxysmal nocturnal hemoglobinuria, PNH, Fabhalta
Medical subject headings: Hemoglobinuria, Paroxysmal

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 48weeks

Secondary outcomes (3)

Occurrence status of serious hemolysis after discontinuation of Fabhalta.
Number of patients with serious hemolysis after discontinuation of Fabhalta.
Measured: 48weeks
Occurrence status of adverse events and adverse drug reactions.
Incidence of adverse events and adverse drug reactions during the treatment period.
Measured: 48weeks
Incidence of breakthrough hemolysis and change of hemolysis-related markers and status of red blood cell transfusion-free.
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.
Measured: 48weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Sep 202430 Sep 2028

First posted23 Sep 2024
Study start18 Sep 2024
Primary completion30 Sep 2028 (estimated)
Study completion30 Sep 2028 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Other
Enrolment: 132 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT06606314
Sponsor's protocol ID: CLNP023C11401
Status last verified: Sep 2026
First submitted: 18 Sep 2024
Last updated: 10 Sep 2026
Results posted: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Active, not recruitingPhase 3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria

All Paroxysmal Nocturnal Hemoglobinuria trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Specified Drug-use Surveillance of Fabhalta Capsules (Paroxysmal Nocturnal Hemoglobinuria, CLNP023C11401)
ClinicalTrials.gov ID: NCT06606314
Acronym / sponsor ID: CLNP023C11401
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: paroxysmal nocturnal hemoglobinuria, PNH, Fabhalta
Study type: Observational
Recruitment status: Active, not recruiting
Enrolment: 132 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 18 Sep 2024 (actual)
Primary completion: 30 Sep 2028 (estimated)
Study completion: 30 Sep 2028 (estimated)
First posted: 23 Sep 2024 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The observation period will be 48 weeks after the start of treatment with Fabhalta.

Study design

Observational model: Cohort
Time perspective: Other

Who may qualify

Ages: 0 to 100 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: All patients who received Fabhalta.

Key inclusion criteria (2 listed)

All patients who received Fabhalta.
·

Key exclusion criteria (1 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

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.

3 secondary outcomes

Occurrence status of serious hemolysis after discontinuation of Fabhalta.
Time frame: 48weeks
Occurrence status of adverse events and adverse drug reactions.
Time frame: 48weeks
Incidence of breakthrough hemolysis and change of hemolysis-related markers and status of red blood cell transfusion-free.
Time frame: 48weeks

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
Individual participant dataSharing planned: No

Related trials for Paroxysmal Nocturnal Hemoglobinuria

RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Active, not recruitingPhase 3
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal Hemoglobinuria

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

91 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 91 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 91 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Occurrence status of infections. (time frame: 48weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06606314.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026; AzTrials last refreshed its copy on 7 Nov 2026.