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Hope for rare immune disorder: long-term drug safety tested

NCT ID NCT02859727

Terminated Disease control Sponsor: Pharming Technologies B.V. Source: ClinicalTrials.gov ↗

First seen Mar 31, 2026 · Last updated May 23, 2026 · Updated 12 times

Summary

This study looked at the long-term safety and effectiveness of the drug CDZ173 in people with APDS/PASLI, a rare genetic condition that weakens the immune system. Participants had either been in a previous study or had taken similar drugs before. The goal was to see how well the drug controlled the disease over time, but the study was stopped early.

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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

  • Pharming Investigative Site

    Bethesda, Maryland, 20892, United States

  • Pharming Investigative Site

    Minsk, 223053, Belarus

  • Pharming Investigative Site

    Prague, CZE, 15006, Czechia

  • Pharming Investigative Site

    Dresden, 01307, Germany

  • Pharming Investigative Site

    Brescia, BS, 25123, Italy

  • Pharming Investigative Site

    Palermo, PA, 90127, Italy

  • Pharming Investigative Site

    Rotterdam, 3000 CA, Netherlands

  • Pharming Investigative Site

    Moscow, 117198, Russia

Conditions

Explore the condition pages connected to this study.

ACTIVATED PI3KDELTA SYNDROME (APDS); PASLI DISEASE