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Could a common arthritis drug help people with down syndrome think and learn better?

NCT ID NCT07598643

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 04, 2026 · Updated 4 times

Summary

This study tests whether tofacitinib, a drug used for arthritis, can improve thinking skills and immune function in people with Down syndrome aged 6 to 22. Half of the 92 participants will receive the drug for 6 months, and the other half a placebo. Researchers will measure changes in intelligence, attention, and daily living skills, while closely watching for side effects.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Tofacitinib (XELJANZ), a JAK inhibitor drug
What this could lead to
If successful, this could point toward a treatment that improves thinking, learning, and overall health in people with Down syndrome.
What could go wrong
This is an early phase 2 trial with only 92 participants, so results may not apply to everyone. The drug also carries risks like infection and requires careful monitoring.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 92 people

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

Started

May 2026

Expected to finish

Aug 2030

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

6 to 22 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: 1. Individuals with DS aged 6 years (inclusive) to 22 years (inclusive). All forms of DS will qualify, including complete trisomy 21, Robertsonian translocation trisomy 21, partial trisomy 21 (segmental duplication), and/or mosaic trisomy 21. 2. Available parent(s) or guardian(s) legally able to sign the consent form and who can complete study materials as appropriate. 3. Body weight is at least 10 kgs. Exclusion Criteria: 1. Prior treatment with a JAK inhibitor or with an investigational agent, device, or procedure within 21 days of enrollment. 2. Current or planned use of a JAK inhibitor during the 6-month study period. 3. Known allergies, hypersensitivity, or intolerance to tofacitinib. 4. Active, uncontrolled, or life-threatening infection that at the determination of the treating physician would preclude safe use of tofacitinib. 5. History of gastrointestinal perforation. 6. Vaccination with live attenuated virus within six weeks of inclusion in the study or planned during the study. Note on vaccines: Participants not yet vaccinated for MMR-V should consider their timeline for MMR-V vaccination. Specifically, the study team recommends MMR-V vaccination as soon as possible and delay study start until 6 weeks after MMR-V vaccinations. 7. Concomitant treatment with any of the following: 1. Concomitant treatment with other immunosuppressants (e.g., methotrexate, azathioprine, tacrolimus, cyclosporine). 2. Strong CYP3A4 inhibitors (e.g., ketoconazole). 3. Strong CYP3A4 Inducers (e.g., rifampin). 4. Moderate CYP3A4 inhibitor(s) with a strong CYP2C19 inhibitor(s) (e.g., fluconazole). 5. Other supplements or medications that at the determination of the treating physician would preclude safe use of tofacitinib. 8. Evidence of severe organ dysfunction, including significantly abnormal laboratory values or severe renal impairment, that at the determination of the treating physician would preclude safe administration of tofacitinib. 9. Any history of leukemia, lymphoma, or unresolved transient myeloproliferative disorder. 10. Any current, recurrent, or metastatic forms of cancer. 11. Any cancer treatment within five years prior to study entry. 12. Known personal history of thrombosis or bleeding disorder. 13. History of tuberculosis, disseminated herpes zoster, disseminated herpes simplex, or recurrent localized herpes zoster. 14. Intravenous antimicrobial therapy within 3 months of inclusion in the study. 15. History of organ or bone marrow transplant. 16. History of myocardial infarction or stroke. 17. Evidence of lipid disorder, including but not limited to LDL \> 190 mg/dL, per discretion of the treating physician. 18. Participant received blood or plasma products within 30 days of the Baseline visit. 19. Treatment with intravenous immunoglobulin (IVIG) within 8 weeks of the Baseline visit. 20. Hospitalization longer than 6 months in the last year. 21. History of neurological syndrome that in the opinion of the study doctors would inhibit successful participation in the study. 22. Less than 6 weeks post-surgery at Baseline appointment. 23. Total vision or hearing loss (with no corrective devices available). 24. Participant must be able to attempt the neurodevelopment assessment battery at Baseline and caregiver must be able to complete proxy reports for neurodevelopmental assessments. 25. Poor venous access not allowing repeated blood tests or non-compliance with venipuncture requirements. 26. Participants may be excluded for other unforeseen reasons at the study doctor's discretion. 27. Pregnancy or breastfeeding. 28. Use of estrogen-containing oral contraceptives.

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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.

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. 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 ↗

  4. 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

  • CU Anschutz, Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

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