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New PNH drug iptacopan tested in Real-World chinese study

NCT ID NCT06931691

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes in 80 patients across China. The goal is to see if iptacopan improves disease control and reduces the need for healthcare resources.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 80 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Adult PNH patients receiving iptacopan for the first time, including those who are complement inhibitor naive and treated

Ages

18 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria For Cohort 1, Patient who meets all the following criteria can be included in this study: 1. Age ≥ 18 years at the time of signing the ICF; 2. Patient with a documented diagnosis of PNH; 3. Patient who has never received complement inhibitor therapy; 4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF; 5. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible. 6. Patient who has signed the ICF. For Cohort 2, Patient who meets all the following criteria can be included in this study: 1. Age ≥ 18 years at the time of signing the ICF; 2. Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment; 3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF; 4. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; 5. If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible. 6. Patient who has signed the ICF. Exclusion criteria For Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study: 1. Participating in an interventional PNH clinical study; 2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose; 3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose; 4. Documented with a history of HIV infection; 5. Women who are pregnant or breastfeeding or intending to conceive during the study period; 6. Existence of bone marrow failure (reticulocytes \< 100 × 109/L, platelets \< 30 × 109/L, and neutrophils \< 0.5 × 109/L) determined by the investigator; 7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Paroxysmal nocturnal hemoglobinuria

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    14 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Novartis Investigative Site

    RECRUITING

    Hefei, Anhui, 230022, China

  • Novartis Investigative Site

    RECRUITING

    Guangzhou, Guangdong, 510000, China

  • Novartis Investigative Site

    RECRUITING

    Guangzhou, Guangdong, 510080, China

  • Novartis Investigative Site

    RECRUITING

    Shijiazhuang, Hebei, 050000, China

  • Novartis Investigative Site

    RECRUITING

    Zhengzhou, Henan, 450003, China

  • Novartis Investigative Site

    RECRUITING

    Zhengzhou, Henan, 450008, China

  • Novartis Investigative Site

    RECRUITING

    Wuhan, Hubei, 430030, China

  • Novartis Investigative Site

    RECRUITING

    Nantong, Jiangsu, 226000, China

  • Novartis Investigative Site

    RECRUITING

    Nanchang, Jiangxi, 330006, China

  • Novartis Investigative Site

    RECRUITING

    Changchun, Jilin, 130021, China

  • Novartis Investigative Site

    RECRUITING

    Jinan, 250012, China

  • Novartis Investigative Site

    RECRUITING

    Tianjin, 300020, China

  • Novartis Investigative Site

    RECRUITING

    Tianjin, 300052, China

  • Novartis Investigative Site

    RECRUITING

    Wuhan, 430022, China

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