Experimental drug shows promise for rare immune disorder, but study cut short
NCT ID NCT02859727
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the long-term safety and effectiveness of a drug called CDZ173 in people with APDS/PASLI, a rare genetic condition that causes a weak immune system and swollen lymph nodes. The 37 participants had already taken CDZ173 or a similar drug in a previous study. The trial was terminated early, so the full picture is unclear, but researchers were looking at side effects and quality of life improvements.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- CDZ173 (a drug that blocks a specific enzyme to help control immune system overactivity)
- What this could lead to
- If successful, this could provide a long-term treatment option to manage APDS/PASLI, reducing infections and improving quality of life.
- What could go wrong
- The study was terminated early, so results are limited. It is a small, open-label extension study, meaning no placebo group and potential bias. Long-term safety and efficacy are not fully established.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Pharming Investigative Site
Bethesda, Maryland, 20892, United States
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Pharming Investigative Site
Minsk, 223053, Belarus
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Pharming Investigative Site
Prague, CZE, 15006, Czechia
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Pharming Investigative Site
Dresden, 01307, Germany
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Pharming Investigative Site
Brescia, BS, 25123, Italy
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Pharming Investigative Site
Palermo, PA, 90127, Italy
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Pharming Investigative Site
Rotterdam, 3000 CA, Netherlands
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Pharming Investigative Site
Moscow, 117198, Russia
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