Use of Specific JAK Inhibition on Inflammatory Skin and Scalp Diseases in Down Syndrome R61AR084210
Icahn School of Medicine at Mount Sinai
Summary
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.
Description
This is a single-center, open-label, basket phase 2b trial that will enroll DS subjects with at least one inflammatory skin condition (Atopic Dermatitis \[AD\] and/or Alopecia Areata \[AA\]). As our goal is to have 51 patients complete the study, with an estimated dropout rate of \~10%, we will enroll a total of n=56 patients. Patients will receive abrocitinib 100 mg daily for 12 weeks. Responders (defined as achieving EASI75 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…
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Participants must meet all of the following criteria to be eligible as study participants: * 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 investiga…
Interventions
- DrugAbrocitinib
All 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.
Location
- Icahn School of Medicine at Mount SinaiNew York, New York