Treatment of Atopic Dermatitis and Alopecia Areata With Abrocitinib in Individuals With Down Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorIcahn School of Medicine at Mount Sinai
About this trial
This is a single-center, open-label, basket phase 2b trial that will enroll Down Syndrome (DS) participants with at least one inflammatory skin condition (Atopic Dermatitis (AD) and/or Alopecia Areata (AA)). Patients will receive Abrocitinib 100 mg daily for 12 weeks. Responders (defined as achieving Eczema Area and Severity Index (EASI) 75 response for AD, or SALT \<= 20 for AA) will be kept on this dose, and non-responders based on these definitions, will initiate 200 mg daily for another 12 weeks. All AD and AA patients will be maintained on the respective dose of Abrocitinib from Week 24 through week 60.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female participants who are at least 12 years old, for whom signed informed consent can be provided by parent or legal guardian/LAR prior to participation in any study assessments or procedures.
Diagnosis of Trisomy 21 or translocation Down Syndrome.
Participant is able to adhere to the study visit schedule and other protocol requirements.
Females of childbearing potential (FCBP) must have a negative pregnancy test at Screening and Week 0/Baseline. While on investigational product and for at least 28 days after taking the last dose of investigational product (IP), FCBP who engage in activity in which conception is possible must use one of the approved contraceptive options described below: a. Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy; or b. Male or female condom (latex condom or non-latex condom NOT made out of natural [animal] membrane [for example, polyurethane]).
Disqualifiers
Inability or unwillingness of a participant's parent or legal guardian/LAR to give written informed consent or comply with study protocol.
Participant is pregnant or breastfeeding.
Participants with AA: A. cause of hair loss is indeterminable and/or they have concomitant causes of alopecia, such as traction, cicatricial, pregnancy-related, druginduced, telogen effluvium, or advanced androgenetic alopecia (i.e. Ludwig Type III or Norwood-Hamilton Stage ≥ V). B. Participant has a history of AA with no evidence of hair regrowth for ≥7 years since the last episode of hair loss
Participant has increased risk of developing venous thromboembolism, e.g. deep vein thrombosis or pulmonary embolism (history of venous thromboembolism, or first-degree relative with unprovoked venous thromboembolism (i.e. without known underlying cause such as trauma, surgery, immobilization, prolonged travel, pregnancy, hormone use, or plaster cast), that would suggest participant is at increased risk of inherited coagulation disorder (e.g. Factor V Leiden).
Trial design
Design model
Single group
Treatments tested in this trial
Abrocitinib
DrugAll participants will be started on 100mg Abrocitinib daily. Based on clinical response, non-responders will be increased to 200mg daily at week 12. Responders will continue to receive 100 mg dose of Abrocitinib. All AA and AD participants will be maintained on their dose of Abrocitinib through week 60.
Treatment groups
56 Participants
are divided into 1 treatment groupGroup A: Open Label AbrocitinibExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Rate of Serious Adverse Events (SAEs) at Week 24 and Week 60
Rate of Serious Adverse Events (SAEs) at week 24 and at 60 weeks to assesses the clinical safety of Abrocitinib in patients with DS and AD or AA.
Time frame
Week 24 and Week 60Secondary outcomes
1
Number and Severity of SAEs and TEAEs at Weeks 4, 8, 12
Overall number and severity of SAEs and Treatment-Emergent Adverse Events (TEAEs) at weeks 4, 8, and 12.
Time frame
Weeks 4, 8, 122
Number of SAEs and TEAEs
Number and severity of SAEs and Treatment-Emergent Adverse Events (TEAEs) at weeks 16, 28, 36, 48 and 60 in each dose group
Time frame
Weeks 16, 28, 36, 48, 603
Proportion of Participants with AD Reaching EASI 75
The proportion of subjects with AD achieving EASI 75 at week 24. The proportion of patients who achieve an improvement of 75% or greater at week 24. The EASI index assigns proportionate values to 4 body regions. Each region is assigned a score of 0 to 3, indicating none, mild, moderate, and severe clinical expression. The percentage of area involved is also assigned an eruption proportional score from 0 to 6. The total body score for each body region is obtained by multiplying the sum of the severity scores by the area score, then multiplying the result by the constant weighted value assigned to that body region. The sum of these scores gives the EASI total from 0-72, with higher score indicating more severity.
Time frame
Week 244
Proportion of Participants with AA reaching SALT ≤ 20
The proportion of subjects with AA achieving SALT ≤ 20 at week 24. SALT ≤ 20 is defined as the proportion of patients achieving ≤ 20% reduction in SALT score compared to baseline. Scalp is divided into 4 areas namely, Vertex - 40% (0.4) of scalp surface area; right profile of scalp - 18% (0.18) of scalp surface area; left profile of scalp - 18% (0.18) of scalp surface area; Posterior aspect of scalp - 24% (0.24) of scalp surface area. Percentage of hair loss in any of these areas is percentage hair loss multiplied by percent surface area of the scalp in that area. SALT score is the sum of percentage of hair loss in all above mentioned areas.
Time frame
Week 24Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Icahn School of Medicine at Mount Sinai
Lead sponsor
Columbia University
Collaborator
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Collaborator
Source ClinicalTrials.gov