New hope for rare immune disorders: upadacitinib trial launches
NCT ID NCT07670156
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time
Summary
This study tests the drug upadacitinib (Rinvoq) in 30 people with rare genetic disorders that overactivate the JAK-STAT immune pathway. The trial has four phases over 12 months, including an open-label phase to find the best dose and a randomized withdrawal phase to compare the drug against placebo. The goal is to see if the drug is safe and can prevent disease flares.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Upadacitinib (Rinvoq), a JAK inhibitor taken as a daily tablet
- What this could lead to
- If successful, this could provide a first approved treatment option for people with rare JAK-STAT disorders, helping control immune symptoms.
- What could go wrong
- This is an early-phase trial with only 30 participants, so results may not apply broadly. The drug may cause side effects or fail to prevent disease flares.
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
Locations
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Columbia University
New York, New York, 10032, United States
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Washington University in St.Louis
St Louis, Missouri, 63110, United States