New hope for hunter syndrome: Long-Term drug trial launches

NCT ID NCT06075537

First seen Sep 30, 2025 · Last updated May 07, 2026 · Updated 32 times

Summary

This study looks at the long-term safety and effects of an experimental drug called DNL310 for people with Hunter syndrome (MPS II), a rare genetic disease. About 99 participants who finished earlier studies will receive the drug for up to 5 years. The goal is to see if the drug remains safe and helps control the disease over time.

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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.

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Contacts and locations

Locations

  • ASST di Cremona

    Cremona, Italy

  • Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia

    Udine, Italy

  • Birmingham Women's and Children's NHS Foundation Trust

    Birmingham, United Kingdom

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Drottning Silvias Barn Och Ungdomssjukhus

    Gothenburg, Sweden

  • Erasmus Medical Center - Sophia Children's Hospital

    Rotterdam, 3015 GD, Netherlands

  • Gazi Universitesi Tip Fakultes

    Ankara, Turkey (Türkiye)

  • Great Ormond Street Hospital

    London, WC1N 3JH, United Kingdom

  • Hopital Jeanne De Flandre - Metabolic Diseases Unit

    Lille, France

  • Hospit U. Vall d'Hebron - PPDS

    Barcelona, Spain

  • Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Huntsman Cancer Hospital

    Salt Lake City, Utah, 84112, United States

  • McGill University Health Center

    Montreal, Quebec, H4A3J1, Canada

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • Sanatorio Mater Dei

    Buenos Aires, Argentina

  • SpinCS GmbH

    Höchheim, Germany

  • The University of Texas Medical School at Houston

    Houston, Texas, 77030, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • UNC Children's Research Institute

    Chapel Hill, North Carolina, 27599, United States

  • UZ Brussel

    Jette, Belgium

  • Universitair Ziekenhuis Antwerpen

    Edegem, Antwerpen, 2650, Belgium

  • University Medical Faculty Balcali Hospital

    Adana, Turkey (Türkiye)

  • University of Alberta - Faculty of Medicine & Dentistry

    Edmonton, Alberta, Canada

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, Germany

  • Vseobecna Fakultni Nemocnice V Praze

    Prague, 120 00, Czechia

Conditions

Explore the condition pages connected to this study.