Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for kids with rare blood disorder: first pediatric trial of oral drug begins

NCT ID NCT06934967

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

Summary

This study is testing an oral medication called iptacopan in children and teenagers (ages 2 to 18) with a rare and serious blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). The main goals are to see how the drug behaves in children's bodies and to check if it is safe and well-tolerated. Researchers will also measure if the drug helps improve anemia and reduces the need for blood transfusions over a year of treatment.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Conditions

Explore the condition pages connected to this study.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)