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Janus Kinase (JAK) Inhibitor

Tofacitinib for Immune Skin Conditions in Down Syndrome

Phase 2
Recruiting
Led By Joaquin Espinosa, PhD
Research Sponsored by University of Colorado, Denver
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Moderate-to-severe hidradenitis suppurativa
Moderate-to-severe vitiligo.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to week 18
Awards & highlights

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.

Who is the study for?
This trial is for adolescents and adults with Down syndrome, aged 12-50 years, weighing at least 40 kg, who have moderate-to-severe immune skin conditions like psoriasis or eczema. Participants must avoid pregnancy or fathering children and need a study partner or legal guardian to assist with the study.Check my eligibility
What is being tested?
The trial tests Tofacitinib's safety and effectiveness in improving skin conditions associated with increased interferon signaling in individuals with Down syndrome. It also examines the drug's impact on molecular markers, cognitive abilities, and quality of life.See study design
What are the potential side effects?
Potential side effects of Tofacitinib may include infections due to weakened immune response, digestive issues, headaches, respiratory tract infections, high blood pressure, and increases in cholesterol levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have moderate-to-severe skin condition with painful lumps.
Select...
I have moderate to severe vitiligo.
Select...
I have moderate-to-severe eczema.
Select...
I have lost at least 25% of my hair.
Select...
I am between 12 and 50 years old, have Down syndrome, and weigh at least 40 kg.
Select...
I have moderate to severe psoriasis.
Select...
I am between 12 and 50 years old, have Down syndrome, and weigh at least 40 kg.
Select...
I have a severe skin condition like eczema, psoriasis, or vitiligo.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to week 18
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 18 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in Interferon (IFN) Scores in the Transcriptome of White Blood Cells
Safety as Assessed by Number of Serious Adverse Events (SAE)
Secondary outcome measures
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
+5 more

Side effects data

From 2017 Phase 3 trial • 209 Patients • NCT02281552
12%
Nasopharyngitis
1%
Interstitial lung disease
1%
Anaemia
1%
Pneumocystis jirovecii pneumonia
1%
Pneumonia bacterial
1%
Pneumonia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Tofacitinib Immediate Release (IR)
Tofacitinib Modified Release (MR)

Trial Design

1Treatment groups
Experimental Treatment
Group I: On TreatmentExperimental Treatment1 Intervention
Tofacitinib 5mg oral tablets twice daily for 16 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tofacitinib
2018
Completed Phase 3
~39970

Find a Location

Who is running the clinical trial?

University of Colorado, DenverLead Sponsor
1,748 Previous Clinical Trials
2,163,919 Total Patients Enrolled
8 Trials studying Down Syndrome
3,571 Patients Enrolled for Down Syndrome
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH
485 Previous Clinical Trials
1,086,862 Total Patients Enrolled
Joaquin Espinosa, PhDPrincipal InvestigatorLinda Crnic Institute, University of Colorado Anschutz Medical Campus
4 Previous Clinical Trials
2,566 Total Patients Enrolled
2 Trials studying Down Syndrome
2,566 Patients Enrolled for Down Syndrome

Media Library

Tofacitinib (Janus Kinase (JAK) Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04246372 — Phase 2
Down Syndrome Research Study Groups: On Treatment
Down Syndrome Clinical Trial 2023: Tofacitinib Highlights & Side Effects. Trial Name: NCT04246372 — Phase 2
Tofacitinib (Janus Kinase (JAK) Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04246372 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Have prior studies been conducted on the efficacy of Tofacitinib?

"Tofacitinib is being explored in 34 different clinical studies, with 7 of those trials currently at Phase 3. Although the majority are based out of Shanghai, there are 662 sites conducting research on this medication worldwide."

Answered by AI

What is the desired outcome of this research endeavor?

"The fundamental objective of this 18 week study is to gauge any alterations in interferon scores within white blood cells. Secondary endpoints include changes in Psoriasis Area and Severity Index (PASI) Score, Eczema Area and Severity Index (EASI), as well as Modified Sartorius Score (MSS). PASI scoring quantifies the area and severity of psoriasis based on parameters like erythema, induration, desquamation; while EASI utilizes 4 measurements such as erythema, infiltration, excoriations and lichenification for assessing eczema's"

Answered by AI

How many enrollees are being accepted for this medical experiment?

"Correct. Data on clinicaltrials.gov verifies that this research endeavour, which was initially listed on October 21st 2020, is actively recruiting patients. Approximately 47 individuals need to be enrolled from 1 centre."

Answered by AI

Is this clinical research actively seeking participants?

"Indeed, records from clinicaltrials.gov disclose that the trial is currently recruiting participants. It was published on October 21st 2020 and underwent its most recent update on July 27th 2022; 47 people are needed for this study across 1 clinic location."

Answered by AI

Is Tofacitinib a risk-free treatment option for patients?

"Tofacitinib has been evaluated in Phase 2 trials and is thought to be reasonably safe, so it earned a rating of 2. No clinical data currently exists demonstrating efficacy however."

Answered by AI

Is this research study accessible to individuals aged forty and above?

"According to the enrollment guidelines, prospective patients should be between 12 and 50 years of age. There are 530 trials for those under 18 while 1448 studies have been developed with seniors in mind."

Answered by AI

Am I eligible to join the experiment?

"Patients with hidradenitis suppurativa are being recruited for this trial if they fall in the 12 to 50 year age range. Currently, 47 participants have been accepted into the study."

Answered by AI

What primary applications is Tofacitinib employed for?

"Tofacitinib is primarily prescribed for children suffering from juvenile arthritis. Additionally, it can be a useful treatment option in cases where patients are intolerant to methotrexate and have not responded well enough to traditional therapies."

Answered by AI

Who else is applying?

What state do they live in?
California
Ontario
Tennessee
How old are they?
18 - 65
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
What site did they apply to?
Linda Crnic Institute for Down Syndrome
How many prior treatments have patients received?
1
3+
~2 spots leftby Aug 2024