Study of Tofacitinib for Down Syndrome
This study is testing a medication called tofacitinib (Xeljanz) in people with Down syndrome aged 6 to 22 years. Tofacitinib is a JAK1/3 inhibitor, which means it blocks certain signals in the immune system. The goal is to see if this medication is safe and effective in improving outcomes and brain development in individuals with Down syndrome. You would either receive tofacitinib or a placebo (an inactive substance that looks and tastes like the study drug) for 6 months. Researchers will measure safety by tracking any side effects, and they will assess effectiveness by looking at changes in verbal and nonverbal intelligence scores. The study aims to enroll up to 92 participants, but the current recruitment status is unclear.
- Study design
- This is a Phase 2, double-blind, randomized, placebo-controlled study. Up to 92 participants will be randomly assigned to receive either tofacitinib or a placebo.
- What's involved
- Participants will complete identical activities over 6 months, including a Baseline visit, a 3-month visit, and a 6-month visit. Safety monitoring will involve self-reporting, lab tests, and doctor assessments.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety will be monitored from screening until one month after the end of treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Joaquin Espinosa, PhD · PRINCIPAL_INVESTIGATOR · Linda Crnic Institute for Down Syndrome, CU Anschutz
Who to contact
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What this trial measures
- Number and percentage of subjects experiencing treatment-emergent adverse events.From screening to 1 month after end of treatment
Number, percentage, type, and severity of treatment-emergent adverse events (TEAEs) will be annotated over the 6-month period in the treatment arm and placebo arm.
- Change in Kaufman Brief Intelligence Test, 2nd Edition Revised (KBIT-2 Revised) - Verbal IntelligenceBaseline, 6 months
Raw scores for Verbal Intelligence
- Change in Kaufman Brief Intelligence Test, 2nd Edition Revised (KBIT-2 Revised) - Nonverbal IntelligenceBaseline, 6 months
Raw scores for Nonverbal Intelligence
- Change in Leiter 3 - Attention Sustained subtestBaseline, 6 months
The raw score is the correct number of targets minus errors made across four trials.
- Change in Vineland Adaptive Behavior Scales 3 (VABS-3) - Sum of Domain Raw ScoresBaseline, 6 months
The sum of raw scores will be calculated as the applicable domain-level raw scores.
- Change in Clinical Global Impressions (CGI) Scale - Improvement in Health (CGI-I-H)Baseline, 6 months
The CGI-I scale, which ranges from 1 to 7, with 1 being "very much improved" and 7 being "very much worse" to assess changes in global health during the 6-month intervention period. Noteworthy, we will also collect the CGI-S score (severity) at each time point (baseline, 3 months - midpoint visit, and 6 months - endpoint visit). The CGI-I will be collected at 3 months and 6 months.