Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1

Part of paid clinical trials in Aurora, Colorado.

Sponsor
University of Colorado, Denver
Study ID
NCT07598643
Phase
PHASE2
Status
Recruiting
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Conditions

  • Down Syndrome

Eligibility Criteria

Sex
ALL
Age
6 Years - 22 Years
Healthy Volunteers
Not accepted

Interventions

  • Tofacitinib Oral Solution — DRUG
    JAK1/3 inhibitor
  • Placebo — DRUG
    The placebo will be compounded by Children's Hospital of Colorado Investigational Drug Services using commercially available syrup with added flavoring to mimic the active product.

Study Details

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. There will be two main arms for this study: a treatment arm and a placebo control arm. Participants will be randomized into the treatment or placebo arm. Those completing 6 months in the placebo arm may be eligible to participate in a cross-over, open-label extension arm to receive 6 months of tofacitinib treatment. Participants will be evaluated during a Screening visit to determine eligibility, complete a Baseline visit if eligible, and be monitored via safety clinical laboratories and in-person evaluations by study doctors at 1 month, 3 months (mid-point visit) and 6 months (endpoint visit). An interim analysis of safety will be completed by an independent Data and Safety Monitoring Board (DSMB) after 40 participants have completed 6 months of treatment or placebo (20 in each arm).

Key Dates

Start date
May 31, 2026
Status verified
May 2026
Primary completion
Aug 31, 2030
Completion
Aug 31, 2030

Study Design

Enrollment
92 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Arm
    Participants enrolled in the treatment arm will receive a 6-month treatment with the JAK1/3 inhibitor tofacitinib (XELJANZ) to define the safety and efficacy of this medicine relative to placebo.
  • Placebo Comparator: Placebo arm
    Participants in the placebo arm will complete the same study activities as the participants in the treatment arm. Placebo will be an oral solution to mimic the active product. At the end of 6 months of activities, unblinding will occur and if eligible, participants in the placebo arm may be offered to participate in the cross-over arm to undergo 6 months of treatment with tofacitinib in an open-label design.

Primary Outcome Measure

Number and percentage of subjects experiencing treatment-emergent adverse events. [ Time Frame: From screening to 1 month after end of treatment ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
CU Anschutz, Children's Hospital ColoradoAuroraColorado80045
Erika M Chelales, PhD
[email protected]
303-724-5017
Constance Brecl
[email protected]
303-724-6214
Joaquin M Espinosa, PhD (PRINCIPAL_INVESTIGATOR)
Jessica L Bloom, MD (PRINCIPAL_INVESTIGATOR)

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By research site
CU Anschutz, Children's Hospital Colorado· Aurora, CO

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