Abrocitinib for Atopic Dermatitis and Alopecia Areata

Overseen By Giselle Singer

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Icahn School of Medicine at Mount Sinai

Must not be taking: Immunosuppressants, Monoclonal antibodies, Corticosteroids, JAK inhibitors

Disqualifiers: Pregnancy, Active infections, Immunodeficiency, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 44

56 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called Abrocitinib, a JAK inhibitor, to determine its effectiveness for people with Down Syndrome who have skin conditions like atopic dermatitis (a chronic itchy rash) or alopecia areata (patchy hair loss). The researchers aim to discover if taking Abrocitinib daily can improve these skin issues over time. Participants with Down Syndrome who have struggled with these conditions for at least six months might be suitable for this study. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to potentially benefit from an innovative therapy.

Is there any evidence suggesting that Abrocitinib is likely to be safe for humans?

Research shows that Abrocitinib is generally safe for people with atopic dermatitis. Studies have confirmed its safety for treating this condition. In clinical trials, serious side effects rarely occurred, and most participants did not experience severe adverse effects.

Abrocitinib is approved by the FDA for atopic dermatitis, indicating it has passed strict safety tests for this condition. However, researchers are still studying its safety for people with Down Syndrome who have skin conditions like atopic dermatitis or alopecia areata.

In summary, past research has demonstrated a good safety record for Abrocitinib. However, more research is needed to fully understand its safety for those with Down Syndrome. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for atopic dermatitis and alopecia areata focus on reducing symptoms like inflammation and itchiness through topical steroids or immunosuppressants. But Abrocitinib works differently, targeting specific enzymes called Janus kinases (JAKs) that play a key role in the inflammatory process. By selectively inhibiting these enzymes, Abrocitinib can potentially reduce inflammation more effectively and with fewer side effects. Researchers are excited about this treatment because it offers a new way to manage these conditions, especially for patients who may not respond well to existing therapies. Plus, Abrocitinib is an oral medication, which can be more convenient compared to topical treatments.

What evidence suggests that Abrocitinib might be an effective treatment for Atopic Dermatitis and Alopecia Areata?

Studies have shown that Abrocitinib effectively treats both atopic dermatitis (AD) and alopecia areata (AA). In this trial, participants will receive Abrocitinib, starting at 100 mg, with the option to increase to 200 mg if they do not respond by Week 12. Research has indicated that patients taking either 200 mg or 100 mg of Abrocitinib for 12 weeks showed significant symptom improvement. For AD, many patients experienced a 75% reduction in the Eczema Area and Severity Index (EASI), which measures skin inflammation. For AA, patients often achieved a SALT score of 20 or lower, indicating significant hair regrowth. Abrocitinib has also proven effective for patients who did not respond well to other treatments like Dupilumab. These findings suggest that Abrocitinib can be a promising option for managing these conditions. ^{1 2 3 6 7}

Who Is on the Research Team?

Emma Guttman

Principal Investigator

Icahn School of Medicine at Mount Sinai

Are You a Good Fit for This Trial?

This trial is for individuals with Down Syndrome who also have atopic dermatitis (a skin condition causing inflammation and itchiness) or alopecia areata (an autoimmune disorder leading to hair loss). Participants must be able to take a daily oral medication and commit to the study's duration.

Inclusion Criteria

Participant is able to adhere to the study visit schedule and other protocol requirements

I am at least 12 years old and have consent from a parent or guardian to participate.

I have been diagnosed with Down Syndrome.

Exclusion Criteria

Participant has increased risk of developing venous thromboembolism

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Phase 1

Participants receive Abrocitinib 100 mg daily for 12 weeks. Responders continue on this dose.

12 weeks

Visits at weeks 0, 4, 8, and 12

Treatment Phase 2

Non-responders switch to Abrocitinib 200 mg daily for another 12 weeks.

12 weeks

Visits at weeks 16, 20, and 24

Maintenance

All AD and AA patients maintained on respective dose of Abrocitinib from Week 24 through Week 60.

36 weeks

Visits at weeks 28, 36, 48, and 60

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Abrocitinib

Trial Overview The trial is testing Abrocitinib, an oral medication, given at 100 mg daily for 12 weeks. Depending on their response, participants may continue this dose or increase to 200 mg daily. The treatment period extends from week 24 through week 60.

How Is the Trial Designed?

Group I: Open Label Abrocitinib Experimental Treatment 1 Intervention

Participants with DS with either AD or AA will be given Abrocitinib 100mg. In the case where patients is not responsive to treatment at Week 12 per protocol defined non-responder criteria, the treatment dosage will change to Abrocitinib 200 mg.

Who Is Running the Clinical Trial?

Icahn School of Medicine at Mount Sinai

Lead Sponsor

Trials

933

Recruited

579,000+

Columbia University

Collaborator

Trials

1,529

Recruited

2,832,000+

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Collaborator

Trials

508

Recruited

1,090,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11616915/

Abrocitinib: A Comprehensive Review of its Efficacy and ... Patients who took abrocitinib 200 mg and 100 mg for 12 weeks demonstrated improvements of at least 75% in the Eczema Area and Severity Index (EASI), and at ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT07242638

Treatment of Atopic Dermatitis and Alopecia Areata With ... Patients will receive abrocitinib 100 mg daily for 12 weeks. Responders (defined as achieving EASI75 response for AD, or SALT ≤20 for AA) will ...

3. jaad.org jaad.org/article/S0190-9622(24)02876-7/fulltext

Oral abrocitinib for treatment of refractory alopecia areata Efficacy end points were defined as the percentage of patients achieving a SALT score ≤ 20, SALT50 (50% of hair regrowth), and SALT75 (75% of ...

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa2019380

Abrocitinib versus Placebo or Dupilumab for Atopic ... In this trial, abrocitinib at a dose of either 200 mg or 100 mg once daily resulted in significantly greater reductions in signs and symptoms of moderate-to- ...

5. medicaljournalssweden.se medicaljournalssweden.se/actadv/article/view/19454

Real-world Experience of Abrocitinib Treatment in Patients ... Abrocitinib is an effective treatment for atopic dermatitis and can be effective for patients with previous inadequate response to dupilumab or upadacitinib.

6. medicaljournalssweden.se medicaljournalssweden.se/actadv/article/view/42990/49877

Real-world Effectiveness and Safety of Janus Kinase ... In the total cohort of 72 patients, 44 (61%) achieved substantial regrowth: 4/8 (50%) on abrocitinib, 15/27 (56%) on baricitinib, 3/6 (50%) on ...

7. hcplive.com hcplive.com/view/real-world-findings-incidence-adverse-events-highlighted-jak-inhibitors

Real-World Findings on Incidence of Adverse Events ... There were 2 upadacitinib studies in atopic dermatitis which highlighted 2 cases of malignancy, and no deaths were recorded in any of the 43 ...