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Hope for kids with rare blood disease: new pill under study

NCT ID NCT06934967

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

What the study statuses mean

This study's is highlighted.

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 07, 2026 · Updated 1 time

Summary

This study tests a medicine called iptacopan in children aged 2 to under 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. About 12 children will take the medicine, and doctors will monitor side effects and blood changes. This is not a cure, but aims to control the disease.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 12 people

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

Started

Oct 2025

Expected to finish

Nov 2031

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.

Ages

2 to 18 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: * Male and female participants 2 to \< 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg. * Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator. * Patients who are anti-C5 treatment naive: mean hemoglobin level \< 10 g/dL confirmed by central laboratory assessment during screening. * Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) \> 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab. * Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated. * Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan. Exclusion Criteria: * History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes. * Known or suspected hereditary complement deficiency at screening. * History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1). * Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100 x 10 to the ninth/L; platelets \< 30 × 10 to the ninth/L; neutrophils \< 0.5 × 10 to the ninth/L). * Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration. * Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration. Other protocol-defined inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

anemia 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, PNH

How to take part

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

  1. The places running it

    15 sites in 6 countries. 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

  • Cancer Institute of New Jersey

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

  • Childrens Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30342, United States

  • Childrens Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104-4399, United States

  • Novartis Investigative Site

    RECRUITING

    Brasília, Federal District, 70684-831, Brazil

  • Novartis Investigative Site

    RECRUITING

    Natal, Rio Grande do Norte, 59012 300, Brazil

  • Novartis Investigative Site

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-003, Brazil

  • Novartis Investigative Site

    RECRUITING

    Santo André, São Paulo, 09090-401, Brazil

  • Novartis Investigative Site

    RECRUITING

    São Paulo, São Paulo, 01323001, Brazil

  • Novartis Investigative Site

    RECRUITING

    São Paulo, São Paulo, 04038-002, Brazil

  • Novartis Investigative Site

    RECRUITING

    Cali, Valle del Cauca Department, 760012, Colombia

  • Novartis Investigative Site

    RECRUITING

    Cologne, North Rhine-Westphalia, 50937, Germany

  • Novartis Investigative Site

    RECRUITING

    Berlin, 13353, Germany

  • Novartis Investigative Site

    RECRUITING

    Genova, GE, 16147, Italy

  • Novartis Investigative Site

    RECRUITING

    Utrecht, 3584 CX, Netherlands

  • St Jude Childrens Research Hospital

    RECRUITING

    Memphis, Tennessee, 38105, United States

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