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New treatment option for Down Syndrome
Official title Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1
ClinicalTrials.gov ID: NCT07598643
What this study is testing
What is Tofacitinib Oral Solution?
Tofacitinib Oral Solution is an investigational medicine, being studied as a potential treatment for down syndrome.
Also referred to as XELJANZ.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This protocol describes a phase 2, double-blind, randomized, placebo-controlled clinical trial for Janus kinase (JAK) inhibition in Down syndrome (DS). This trial will evaluate the safety and efficacy of a 6-month treatment with the JAK1/3 inhibitor tofacitinib (XELJANZ) in individuals ages 6-22 (inclusive) with DS.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 6 months
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 22
You may be able to join if
- Individuals with DS aged 6 years (inclusive) to 22 years (inclusive). All forms of DS will qualify, including complete trisomy 21, Robertsonian...
- Available parent(s) or guardian(s) legally able to sign the consent form and who can complete study materials as appropriate.
- Body weight is at least 10 kgs.
You likely can't join if
- Prior treatment with a JAK inhibitor or with an investigational agent, device, or procedure within 21 days of enrollment.
- Current or planned use of a JAK inhibitor during the 6-month study period.
- Known allergies, hypersensitivity, or intolerance to tofacitinib.
- Active, uncontrolled, or life-threatening infection that at the determination of the treating physician would preclude safe use of tofacitinib.
- History of gastrointestinal perforation.
- Vaccination with live attenuated virus within six weeks of inclusion in the study or planned during the study. Note on vaccines: Participants not yet...
See the full eligibility criteria
- 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)...
- Available parent(s) or guardian(s) legally able to sign the consent form and who can complete study materials as appropriate.
- Body weight is at least 10 kgs.
- Prior treatment with a JAK inhibitor or with an investigational agent, device, or procedure within 21 days of enrollment.
- Current or planned use of a JAK inhibitor during the 6-month study period.
- Known allergies, hypersensitivity, or intolerance to tofacitinib.
- Active, uncontrolled, or life-threatening infection that at the determination of the treating physician would preclude safe use of tofacitinib.
- History of gastrointestinal perforation.
- 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...
- Concomitant treatment with any of the following:
- Concomitant treatment with other immunosuppressants (e.g., methotrexate, azathioprine, tacrolimus, cyclosporine).
- Strong CYP3A4 inhibitors (e.g., ketoconazole).
- Strong CYP3A4 Inducers (e.g., rifampin).
- Moderate CYP3A4 inhibitor(s) with a strong CYP2C19 inhibitor(s) (e.g., fluconazole).
- Other supplements or medications that at the determination of the treating physician would preclude safe use of tofacitinib.
- Evidence of severe organ dysfunction, including severe renal impairment, that at the determination of the treating physician would preclude safe administration of tofacitinib.
- Any history of leukemia, lymphoma, or unresolved transient myeloproliferative disorder.
- Any current, recurrent, or metastatic forms of cancer.
- Any cancer treatment within five years prior to study entry.
- Known personal history of thrombosis or bleeding disorder.
- History of tuberculosis, disseminated herpes zoster, disseminated herpes simplex, or recurrent localized herpes zoster.
- Intravenous antimicrobial therapy within 3 months of inclusion in the study.
- History of organ or bone marrow transplant.
- History of myocardial infarction or stroke.
- Evidence of lipid disorder, including but not limited to LDL \> 190 mg/dL, per discretion of the treating physician.
- Participant received blood or plasma products within 30 days of the Baseline visit.
- Treatment with intravenous immunoglobulin (IVIG) within 8 weeks of the Baseline visit.
- Hospitalization longer than 6 months in the last year.
- History of neurological syndrome that in the opinion of the study doctors would inhibit successful participation in the study.
- Less than 6 weeks post-surgery at Baseline appointment.
- Total vision or hearing loss (with no corrective devices available).
- Participant must be able to attempt the neurodevelopment assessment battery at Baseline and caregiver must be able to complete proxy reports for neurodevelopmental assessments.
- Poor venous access not allowing repeated blood tests or non-compliance with venipuncture requirements.
- Participants may be excluded for other unforeseen reasons at the study doctor's discretion.
- Pregnancy or breastfeeding.
- Use of estrogen-containing oral contraceptives.
The study team makes the final eligibility decision.
Where it's taking place
- Aurora, Colorado, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 6 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 22 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Aurora, Colorado, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.