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

NCT07598643 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 92

Last updated 2026-05-20

No results posted yet for this study

Summary

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).

Conditions

  • Down Syndrome

Interventions

DRUG

Tofacitinib Oral Solution

JAK1/3 inhibitor

DRUG

Placebo

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.

Sponsors & Collaborators

  • Anschutz Acceleration Initiative

    collaborator UNKNOWN

  • GLOBAL Down Syndrome Foundation

    collaborator UNKNOWN

  • University of Colorado, Denver

    lead OTHER

Principal Investigators

  • Joaquin Espinosa, PhD · Linda Crnic Institute for Down Syndrome, CU Anschutz

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-31
Primary Completion
2030-08-31
Completion
2030-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07598643 on ClinicalTrials.gov