Tofacitinib for Down Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Down Syndrome+5 MoreTofacitinib - Drug
Eligibility
12 - 50
All Sexes
What conditions do you have?
Select

Study Summary

This trial tests if a drug that blocks interferon signaling is safe and effective in people with Down syndrome and an autoimmune or autoinflammatory skin condition.

Eligible Conditions
  • Down Syndrome
  • Hidradenitis Suppurativa
  • Psoriasis
  • Eczema
  • Vitiligo
  • Alopecia Areata

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

2 Primary · 8 Secondary · Reporting Duration: Up to Week 18

Baseline and 16 weeks
A composite score generated using the Meso Scale Discovery (MSD) platform used to assess inflammatory changes in plasma.
Change in Dermatology Life Quality Index (DLQI)
Change in Eczema Area and Severity Index (EASI) Score in Participants with Atopic Dermatitis
Change in Interferon (IFN) Scores in the Transcriptome of White Blood Cells
Change in Investigator's Global Assessment (IGA)
Change in Modified Sartorius Score (MSS) Score in Participants with Hidradenitis Suppurativa
Change in Psoriasis Area and Severity Index (PASI) Score in Participants with Psoriasis
Change in Severity of Alopecia Tool (SALT) Score in Participants with Alopecia
Change in Vitiligo Extent Tensity Index (VETI) in Participants with Vitiligo
Up to Week 18
Safety as Assessed by Number of Serious Adverse Events (SAE)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

Side Effects for

Tofacitinib
47%Dyslipidemia
32%Upper respiratory tract infection
11%Acne
5%Constipation
5%Myalgia
5%Intestinal tuberculosis
5%Weight gain
This histogram enumerates side effects from a completed 2021 Phase 4 trial (NCT03800979) in the Tofacitinib ARM group. Side effects include: Dyslipidemia with 47%, Upper respiratory tract infection with 32%, Acne with 11%, Constipation with 5%, Myalgia with 5%.

Trial Design

1 Treatment Group

On Treatment
1 of 1

Experimental Treatment

47 Total Participants · 1 Treatment Group

Primary Treatment: Tofacitinib · No Placebo Group · Phase 2

On Treatment
Drug
Experimental Group · 1 Intervention: Tofacitinib · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tofacitinib
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: up to week 18

Who is running the clinical trial?

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH
447 Previous Clinical Trials
1,072,413 Total Patients Enrolled
University of Colorado, DenverLead Sponsor
1,591 Previous Clinical Trials
1,927,216 Total Patients Enrolled
8 Trials studying Down Syndrome
2,571 Patients Enrolled for Down Syndrome
Cory Dunnick, MDPrincipal InvestigatorDepartment of Dermatology, University of Colorado Anschutz Medical Campus
2 Previous Clinical Trials
116 Total Patients Enrolled
David Norris, MDPrincipal InvestigatorDepartment of Dermatology, University of Colorado Anschutz Medical Campus
Joaquin Espinosa, PhDPrincipal InvestigatorLinda Crnic Institute, University of Colorado Anschutz Medical Campus
4 Previous Clinical Trials
1,566 Total Patients Enrolled
2 Trials studying Down Syndrome
1,566 Patients Enrolled for Down Syndrome

Eligibility Criteria

Age 12 - 50 · All Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have moderate-to-severe atopic dermatitis.
You have moderate-to-severe psoriasis.
You must be at least 18 years old to complete this study.

Who else is applying?

What state do they live in?
Tennessee50.0%
California50.0%
How old are they?
18 - 65100.0%
What portion of applicants met pre-screening criteria?
Met criteria25.0%
Did not meet criteria75.0%
What site did they apply to?
Linda Crnic Institute for Down Syndrome100.0%