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PNH patients get extended access to iptacopan in safety follow-up

NCT ID NCT04747613

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

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

Summary

This study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side effects and how well the drug controls their disease. The goal is to provide ongoing treatment access while gathering more safety data.

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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

Contacts and locations

Locations

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Augusta University Georgia

    Augusta, Georgia, 30912, United States

  • City Of Hope

    Duarte, California, 91010, United States

  • City Of Hope National Med Center

    Duarte, California, 91010, United States

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Lakes Research

    Miami Lakes, Florida, 33014, United States

  • Montefiore Medical Center

    The Bronx, New York, 10461, United States

  • Novartis Investigative Site

    Santo André, São Paulo, 09090-790, Brazil

  • Novartis Investigative Site

    São Paulo, São Paulo, 01323-900, Brazil

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Tianjin, 300020, China

  • Novartis Investigative Site

    Tianjin, 300052, China

  • Novartis Investigative Site

    Ostrava, Poruba, 708 52, Czechia

  • Novartis Investigative Site

    Brno, 625 00, Czechia

  • Novartis Investigative Site

    Lille, 59037, France

  • Novartis Investigative Site

    Nantes, 44093, France

  • Novartis Investigative Site

    Nice, 06202, France

  • Novartis Investigative Site

    Paris, 75475, France

  • Novartis Investigative Site

    Toulouse, 31059, France

  • Novartis Investigative Site

    Dresden, Saxony, 01307, Germany

  • Novartis Investigative Site

    Aachen, 52074, Germany

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Hamburg, 20246, Germany

  • Novartis Investigative Site

    Riesa, 01589, Germany

  • Novartis Investigative Site

    Ulm, 89081, Germany

  • Novartis Investigative Site

    Ascoli Piceno, AP, 63100, Italy

  • Novartis Investigative Site

    Avellino, AV, 83100, Italy

  • Novartis Investigative Site

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    Bassano del Grappa, VI, 36061, Italy

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 453-8511, Japan

  • Novartis Investigative Site

    Isehara, Kanagawa, 259-1193, Japan

  • Novartis Investigative Site

    Suwa, Nagano, 392-8510, Japan

  • Novartis Investigative Site

    Niigata, Niigata, 951-8520, Japan

  • Novartis Investigative Site

    Suita, Osaka, 565-0871, Japan

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo, 160-0023, Japan

  • Novartis Investigative Site

    Fukushima, 9601295, Japan

  • Novartis Investigative Site

    Ishikawa, 9208641, Japan

  • Novartis Investigative Site

    Kyoto, 6068507, Japan

  • Novartis Investigative Site

    Vilnius, LT-08406, Lithuania

  • Novartis Investigative Site

    Kota Kinabalu, Sabah, 88586, Malaysia

  • Novartis Investigative Site

    Kuching, Sarawak, 93586, Malaysia

  • Novartis Investigative Site

    Nijmegen, Gelderland, 6500HB, Netherlands

  • Novartis Investigative Site

    Singapore, 119074, Singapore

  • Novartis Investigative Site

    Seoul, Seoul, 03080, South Korea

  • Novartis Investigative Site

    Seoul, Seoul, 06351, South Korea

  • Novartis Investigative Site

    Santiago Compostela, A Coruna, 15706, Spain

  • Novartis Investigative Site

    Donostia / San Sebastian, Gipuzkoa, 20014, Spain

  • Novartis Investigative Site

    Barcelona, 08036, Spain

  • Novartis Investigative Site

    Hualien City, 970, Taiwan

  • Novartis Investigative Site

    Taipei, 10002, Taiwan

  • Novartis Investigative Site

    Istanbul, Fatih, 34093, Turkey (Türkiye)

  • Novartis Investigative Site

    Leeds, West Yorkshire, LS9 7TF, United Kingdom

  • Novartis Investigative Site

    London, SE5 9RS, United Kingdom

  • Prisma Health Upstate

    Greenville, South Carolina, 29615, United States

  • USC Norris Cancer Center

    Los Angeles, California, 90033, United States

  • Univ Cali Irvine ALS Neuromuscular

    Orange, California, 92868, United States

  • Univ of California Irvine (Chao Family Comprehensive Cancer Center)

    Orange, California, 92868, United States