Down SyndromePhase 2MISSION-1NCT07598643
Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1
A Phase 2 study testing Tofacitinib Oral Solution in people with Down Syndrome, run by University of Colorado, Denver at 1 site in United States.
Trial 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 in individuals ages 6-22 (inclusive) with DS. Read more about this study
- Who is it for?
- Ages 6 to 22 years
- Conditions the study is for
- Down Syndrome
- What is being tested?
- Tofacitinib Oral Solution
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 92 participants
- Where
- 1 siteUnited States
- Study dates
- 28 May 2026to Aug 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- 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.
Who cannot join
- 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.
Show all 34Show fewer
- 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.
- 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 significantly abnormal laboratory values or 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
CU Anschutz, Children's Hospital Colorado
Aurora, Colorado, 80045
Erika M Chelales, PhD, 303-724-5017, DSresearch@cuanschutz.edu
Constance Brecl, 303-724-6214, DSresearch@cuanschutz.edu
About this study
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 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).
Read the detailed description
This is a phase 2 randomized, double-blind, placebo-controlled clinical trial for Janus kinase (JAK) inhibition in Down syndrome (DS). After successful enrollment, including informed consent and assessment of inclusion and exclusion criteria, participants will be enrolled and randomized into the treatment or placebo arms and complete identical activities over the course of 6 months.
Briefly, the study recruitment goal is 80 participants (n=40 per treatment and placebo arm) with up to 92 participants enrolled. Participants enrolled in the treatment arm will receive a 6-month treatment with the JAK1/3 inhibitor tofacitinib to define the safety and efficacy of this medicine relative to placebo.
Safety monitoring will be completed over the 6-month period through a combination of self-reporting, laboratory testing, and study doctor assessment. AEs will be annotated by the study team and classified per Common Terminology Criteria for Adverse Events (CTCAE 6.0).
Diverse metrics of neurodevelopment and overall health will be obtained at the Baseline visit, 3-month visit (midpoint) and 6-month visit (endpoint). The data obtained after 6 months of treatment or placebo will be used for all endpoint analyses.
Participants enrolled in the placebo arm will be eligible to continue in the trial for an additional 6 months of tofacitinib treatment in a cross-over, open-label extension arm. Data collected during the cross-over, open-label extension arm will not contribute to any of the primary endpoint analyses. Rather, the cross-over dataset will be used to complete exploratory analyses of longitudinal intra-individual variability while on placebo and tofacitinib. Activities during 6 months of treatment in the cross-over arm will be identical to the main treatment arm. The cross-over arm will also serve to incentivize participation by ensuring that all eligible participants will be able to receive the medicine at some point during the trial.
- Conditions
- Down Syndrome
- Being tested
- Tofacitinib Oral Solution, Placebo
- Keywords
- Down syndrome, JAK inhibition, Tofacitinib
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tofacitinib Oral SolutionDrug
Also called XELJANZ, Tofacitinib
JAK1/3 inhibitor
PlaceboDrug
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 group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | Participants enrolled in the treatment arm will receive a 6-month treatment with the JAK1/3 inhibitor tofacitinib to define the safety and efficacy of this medicine relative to placebo. Receives: Drug: Tofacitinib Oral Solution |
| Placebo arm | Placebo comparator | 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and percentage of subjects experiencing treatment-emergent adverse events.
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.
Measured: From screening to 1 month after end of treatment
Change in Kaufman Brief Intelligence Test, 2nd Edition Revised (KBIT-2 Revised) - Verbal Intelligence
Verbal Intelligence score, made up of the Verbal Knowledge and Riddles subtest, is a standardized measure of verbal cognitive ability. Raw scores,will be employed. Increase in scores indicate improvement in verbal cognitive performance.
Measured: Baseline, 6 months
Change in Kaufman Brief Intelligence Test, 2nd Edition Revised (KBIT-2 Revised) - Nonverbal Intelligence
The KBIT-2 Revised Nonverbal Intelligence score is a standardized measure of nonverbal reasoning ability and is made up of the Matrices subtest. We will be using the raw score. Increase in scores indicate improvement in nonverbal cognitive performance.
Measured: Baseline, 6 months
Change in Leiter 3 - Attention Sustained subtest
The Leiter-3 Attention Sustained subtest is a measure of inhibitory control. The raw score, calculated as the correct number of targets minus errors made across four trials, will be used. Increase in scores indicate improvement.
Measured: Baseline, 6 months
Change in Vineland Adaptive Behavior Scales 3 (VABS-3) - Sum of Domain Raw Scores
The Vineland Adaptive Behavior Scales, Third Edition (VABS-3) assesses adaptive functioning across communication, daily living skills, socialization, and motor domains. Domain raw scores will be summed to produce a composite raw score. . Increase in scores indicate improvement in adaptive functioning.
Measured: Baseline, 6 months
Change in Clinical Global Impressions (CGI) Scale - Improvement in Health (CGI-I-H)
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.
Measured: Baseline, 6 months
Secondary outcomes (10)
Change in Peabody Picture Vocabulary Test, Fifth Edition (PPVT-5)
The Peabody Picture Vocabulary Test, Fifth Edition (PPVT-5) assesses receptive vocabulary. Growth Scale Values (GSV) are used to determine an individual's absolute progress over time and is sensitive to small changes . Increase in scores indicate improvement in receptive vocabulary ability.
A Naturalistic Language Sample will be utilized to measure spontaneous expressive language. We will analyze total utterances and mean length of utterance. Increase in scores indicate improvement in expressive language skills
Measured: Baseline, 6 months
Change in Naturalistic Language Sample
This measure evaluates spontaneous expressive language narration. We will analyze total utterances and mean length of utterance.
Measured: Baseline, 6 months
Change in Developmental Behavior Checklist 2 (DBC-2) or Achenbach Child (or Adult) Behavior Checklist
The DBC-2 reports a statistically significant change in Developmental Behavioral Checklist-2 (DBC-2) T-scores within or between treatment arms. A decrease in score indicates improvement.
Or the Achenbach is a caregiver-report measure of maladaptive behavior. This is a standardized questionnaire with available score norms by chronological age resulting in T-scores. We will analyze change in the internalizing and externalizing T-scores.
Measured: Baseline, 6 months
Change in Social Responsiveness Scale 2 (SRS-2), School Age and Adult
The Social Responsiveness Scale, Second Edition (SRS-2) generates T-scores with a mean of 50 and a standard deviation of 10. . Increase in scores indicates greater severity of autism-related symptoms (worse outcome). Both total and social communication T-scores will be analyzed.
Measured: Baseline, 6 months
Change in Modified Corsi Test
The Modified Corsi Test assesses working memory. Scores are summed based on total performance across all trials. Increase in scores indicates improvement in working memory performance.
Measured: Baseline, 6 months
Change in Dimensional Change Card Sort test
The Dimensional Change Card Sort Test measures cognitive flexibility. The total number of correct post-switch responses will be calculated across the last two trials. Increase in scores indicates improvement in cognitive flexibility.
Measured: Baseline, 6 months
Change in Beery Visual Motor Integration Scales (Beery VMI)
The Beery VMI, assesses visual-motor integration skills. Increase in scores indicates improvement in visual-motor integration ability.
Measured: Baseline, 6 months
Change in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) - Sum of Raw Scores
The Vineland-3 is composed of four domains: Communication, Daily Living Skills, Socialization, and Motor. Each domain score represents summed raw scores from subdomains. We will evaluate changes in the raw scores of the four domains.. Increase in scores indicate improvement of adaptive skills in communication, daily living skills, socialization, and motor skills.
Measured: Baseline, 6 months
Change in Composite Neurodevelopmental Improvement Scores
This composite score aggregates standardized changes across multiple assessments. Individual measures are transformed into standardized differences before averaging. The composite score is not bounded by a fixed range. Higher values indicate greater overall improvement in neurodevelopmental functioning
Measured: Baseline, 6 months
Change in Clinical Global Impression - Improvement in Neurodevelopment (CGI-I-ND)
This composite score aggregates standardized changes across multiple assessments. Individual measures are transformed into standardized differences before averaging. The composite score is not bounded by a fixed range. Higher values indicate greater overall improvement in neurodevelopmental functioning. CGI-I-ND is a scale to rate improvement.
Measured: Baseline, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2026
- Study start28 May 2026
- Primary completionAug 2030 (estimated)
- Study completionAug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Phase 2, double-blind, randomized, placebo-controlled clinical trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 92 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Anschutz Acceleration Initiative, GLOBAL Down Syndrome Foundation
- Responsible party
- Sponsor
- Investigators
- Joaquin Espinosa, PhD, Principal investigator, Linda Crnic Institute for Down Syndrome, CU Anschutz
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07598643
- Sponsor's protocol ID
- 24-0432
- Acronym
- MISSION-1
- Status last verified
- Aug 2026
- First submitted
- 13 May 2026
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified participant data will be made available for all primary outcome measures.
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1
- ClinicalTrials.gov ID
- NCT07598643
- Acronym / sponsor ID
- MISSION-1 / 24-0432
- Conditions
- Down Syndrome
- Keywords
- Down syndrome, JAK inhibition, Tofacitinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 92 participants (estimated)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Anschutz Acceleration Initiative, GLOBAL Down Syndrome Foundation
- Study start
- 28 May 2026 (actual)
- Primary completion
- Aug 2030 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 20 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 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).
Detailed description
This is a phase 2 randomized, double-blind, placebo-controlled clinical trial for Janus kinase (JAK) inhibition in Down syndrome (DS). After successful enrollment, including informed consent and assessment of inclusion and exclusion criteria, participants will be enrolled and randomized into the treatment or placebo arms and complete identical activities over the course of 6 months.
Briefly, the study recruitment goal is 80 participants (n=40 per treatment and placebo arm) with up to 92 participants enrolled. Participants enrolled in the treatment arm will receive a 6-month treatment with the JAK1/3 inhibitor tofacitinib to define the safety and efficacy of this medicine relative to placebo.
Safety monitoring will be completed over the 6-month period through a combination of self-reporting, laboratory testing, and study doctor assessment. AEs will be annotated by the study team and classified per Common Terminology Criteria for Adverse Events (CTCAE 6.0).
Diverse metrics of neurodevelopment and overall health will be obtained at the Baseline visit, 3-month visit (midpoint) and 6-month visit (endpoint). The data obtained after 6 months of treatment or placebo will be used for all endpoint analyses.
Participants enrolled in the placebo arm will be eligible to continue in the trial for an additional 6 months of tofacitinib treatment in a cross-over, open-label extension arm. Data collected during the cross-over, open-label extension arm will not contribute to any of the primary endpoint analyses. Rather, the cross-over dataset will be used to complete exploratory analyses of longitudinal intra-individual variability while on placebo and tofacitinib. Activities during 6 months of treatment in the cross-over arm will be identical to the main treatment arm. The cross-over arm will also serve to incentivize participation by ensuring that all eligible participants will be able to receive the medicine at some point during the trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Phase 2, double-blind, randomized, placebo-controlled clinical trial
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Drug: Tofacitinib Oral SolutionParticipants enrolled in the treatment arm will receive a 6-month treatment with the JAK1/3 inhibitor tofacitinib to define the safety and efficacy of this medicine relative to placebo. |
| Placebo arm | Placebo comparator | Drug: PlaceboParticipants 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. |
Who may qualify
- Ages
- 6 to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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.
- 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.
Key exclusion criteria (34 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tofacitinib Oral SolutionAlso: XELJANZ, TofacitinibJAK1/3 inhibitor | Drug | Treatment Arm |
| PlaceboThe 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. | Drug | Placebo arm |
Primary outcome
Number and percentage of subjects experiencing treatment-emergent adverse events.
Time frame: 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 Intelligence
Time frame: Baseline, 6 months
Verbal Intelligence score, made up of the Verbal Knowledge and Riddles subtest, is a standardized measure of verbal cognitive ability. Raw scores,will be employed. Increase in scores indicate improvement in verbal cognitive performance.
Change in Kaufman Brief Intelligence Test, 2nd Edition Revised (KBIT-2 Revised) - Nonverbal Intelligence
Time frame: Baseline, 6 months
The KBIT-2 Revised Nonverbal Intelligence score is a standardized measure of nonverbal reasoning ability and is made up of the Matrices subtest. We will be using the raw score. Increase in scores indicate improvement in nonverbal cognitive performance.
Change in Leiter 3 - Attention Sustained subtest
Time frame: Baseline, 6 months
The Leiter-3 Attention Sustained subtest is a measure of inhibitory control. The raw score, calculated as the correct number of targets minus errors made across four trials, will be used. Increase in scores indicate improvement.
Change in Vineland Adaptive Behavior Scales 3 (VABS-3) - Sum of Domain Raw Scores
Time frame: Baseline, 6 months
The Vineland Adaptive Behavior Scales, Third Edition (VABS-3) assesses adaptive functioning across communication, daily living skills, socialization, and motor domains. Domain raw scores will be summed to produce a composite raw score. . Increase in scores indicate improvement in adaptive functioning.
Change in Clinical Global Impressions (CGI) Scale - Improvement in Health (CGI-I-H)
Time frame: 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.
10 secondary outcomes
Change in Peabody Picture Vocabulary Test, Fifth Edition (PPVT-5)
Time frame: Baseline, 6 months
Change in Naturalistic Language Sample
Time frame: Baseline, 6 months
Change in Developmental Behavior Checklist 2 (DBC-2) or Achenbach Child (or Adult) Behavior Checklist
Time frame: Baseline, 6 months
Change in Social Responsiveness Scale 2 (SRS-2), School Age and Adult
Time frame: Baseline, 6 months
Change in Modified Corsi Test
Time frame: Baseline, 6 months
Change in Dimensional Change Card Sort test
Time frame: Baseline, 6 months
Change in Beery Visual Motor Integration Scales (Beery VMI)
Time frame: Baseline, 6 months
Change in Vineland Adaptive Behavior Scales, Third Edition (VABS-3) - Sum of Raw Scores
Time frame: Baseline, 6 months
Change in Composite Neurodevelopmental Improvement Scores
Time frame: Baseline, 6 months
Change in Clinical Global Impression - Improvement in Neurodevelopment (CGI-I-ND)
Time frame: Baseline, 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Individual participant dataSharing planned: Yes. De-identified participant data will be made available for all primary outcome measures.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Joaquin Espinosa, PhD principal investigator
Linda Crnic Institute for Down Syndrome, CU Anschutz
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tofacitinib Oral Solution in people with Down Syndrome, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 34 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Constance Brecl (303-724-6214, constance.brecl@cuanschutz.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number and percentage of subjects experiencing treatment-emergent adverse events. (time frame: From screening to 1 month after end of treatment). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.