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New hope for rare immune disorders: upadacitinib trial launches

NCT ID NCT07670156

Disease control Sponsor: Lisa Satter Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 09, 2026 · Updated 3 times

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2026

An estimate. Start dates often move.

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * At least 30kg * Patients with a confirmed JAK/STATGOF mutation with evidence of immune dysregulation * Current disease status meeting criteria as defined in the disease scoring module * Expected survival of more than 12 months * Willingness to allow storage of biological samples for future research * Agreement to use highly effective contraception (for female participants) Exclusion Criteria: * Hypersensitivity to the study drug or any medication in the same class * Active infections * Central nervous system (CNS) manifestations * Pregnancy * Medical conditions or use of concomitant medications that may interfere with the effect or evaluation of the study drug * Laboratory abnormalities as specified in the protocol * Receipt of a live vaccine within 30 days prior to study treatment * High risk or history of osteoporosis, thrombosis, gastrointestinal (GI) perforation, or malignancy

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Conditions

The condition(s) this trial relates to.

autosomal recessive mendelian susceptibility to mycobacterial diseases due to partial jak1 deficiency growth hormone insensitivity with immune dysregulation 1, autosomal recessive inborn error of immunity STAT3-related early-onset multisystem autoimmune disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

JAK1 gof STAT1 gof STAT3 gof STAT5B gof STAT6 gof

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Columbia University

    New York, New York, 10032, United States

  • Washington University in St.Louis

    St Louis, Missouri, 63110, United States