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

NCT ID NCT06934967

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

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

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.

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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)

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-••••

Locations

  • Cancer Institute of New Jersey

    RECRUITING

    New Brunswick, New Jersey, 08901, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Childrens Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104 4399, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • 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

    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

    Contact Email: •••••@•••••

    Contact

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