Dupilumab for Alopecia Areata

(PEDAL Trial)

Recruiting in Palo Alto (17 mi)

+4 other locations

Overseen by Emma Guttman-Yassky

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Icahn School of Medicine at Mount Sinai

Must not be taking: Immunosuppressives, Corticosteroids, JAK inhibitors, others

Disqualifiers: Pregnancy, Severe asthma, Infections, others

Prior Safety Data

Breakthrough Therapy

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests a new treatment for children and adolescents with significant hair loss due to Alopecia Areata. The treatment likely helps the immune system stop attacking hair follicles, allowing hair to regrow. Oral tofacitinib has shown promising results in treating alopecia areata in children.

Do I have to stop taking my current medications for the trial?

Yes, you will need to stop taking certain medications. Specifically, you must not use systemic immunosuppressive medications or certain topical treatments within 4 weeks before the trial starts, and you must stop using topical corticosteroids, tacrolimus, or pimecrolimus 1 week before the trial begins.

What data supports the effectiveness of the drug Dupilumab for treating alopecia areata?

Research shows that Dupilumab, originally used for atopic dermatitis, has helped some patients with alopecia areata regrow hair, as seen in cases where patients experienced significant hair regrowth and improvement in their skin condition. ^{1 2 3 4 5}

Is Dupilumab generally safe for humans?

Dupilumab has been used to treat conditions like atopic dermatitis and has shown a good safety profile in some cases, with no side effects reported in a child with severe combined immunodeficiency. However, there have been reports of hair loss (alopecia) and eye-related side effects, suggesting that while it is generally safe, some people may experience adverse effects. ^{1 3 5 6 7}

How does the drug Dupilumab differ from other treatments for alopecia areata?

Dupilumab is unique because it is a biologic drug that targets specific immune pathways by blocking interleukin-4 and interleukin-13 receptors, which are involved in inflammation. Unlike conventional treatments, it has shown promise in improving alopecia areata in patients with atopic dermatitis, although its effects can vary, with some reports of hair regrowth and others of hair loss. ^{1 3 4 5 6}

Eligibility Criteria

Children and teens aged 6-18 with moderate to severe Alopecia Areata (AA), affecting over half of the scalp, who weigh at least 30 kg. They must have had some hair regrowth in the past 7 years and either a personal or family history of allergies or related conditions. Participants need to be generally healthy, able to follow the study schedule, and girls able to bear children must test negative for pregnancy.

Inclusion Criteria

I am between 6 and 18 years old and have my parent's or guardian's consent to participate.

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

I weigh at least 30 kilograms.

Exclusion Criteria

I am using or plan to use anti-retroviral therapy during the study.

I have or might have a blood cancer, or had another cancer in the last 5 years, except for certain skin and cervical cancers.

I have ongoing severe infections or a history of them.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment

Participants receive either Dupilumab or placebo injections every 2 or 4 weeks based on weight for 48 weeks

48 weeks

Visits every 8-16 weeks for clinical assessments and monitoring

Open-label extension

Participants receive open-label Dupilumab treatment for an additional 48 weeks

48 weeks

Visits every 8-16 weeks for clinical assessments and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Treatment Details

Interventions

• Dupilumab (Monoclonal Antibodies)
• Placebo (Other)

Trial Overview The trial is testing Dupilumab against a placebo in kids with AA. It's set up so neither doctors nor patients know who gets what treatment (double-blind). The study will last up to 116 weeks including screening, treatment phases, and follow-up. Patients are randomly chosen for either Dupilumab or placebo.

Participant Groups

Group I: Dupilumab Experimental Treatment 1 Intervention

200 mg or 300 mg SC injections every 2 or 4 weeks (weight based)

Group II: Placebo Placebo Group 1 Intervention

Injections without active ingredients

Dupilumab is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Dupixent for:

• Atopic dermatitis
• Asthma
• Chronic rhinosinusitis with nasal polyps
• Eosinophilic esophagitis

🇪🇺 Approved in European Union as Dupixent for:

• Atopic dermatitis
• Asthma
• Chronic rhinosinusitis with nasal polyps
• Eosinophilic esophagitis

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Yale University New Haven, CT

Icahn School of Medicine at Mount Sinai New York, NY

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, IL

Northwestern University Feinberg School of Medicine Chicago, IL

More Trial Locations

Who Is Running the Clinical Trial?

Icahn School of Medicine at Mount Sinai Lead Sponsor

References

1. Australia pubmed.ncbi.nlm.nih.gov

Improvement of alopecia areata with Dupilumab in a patient with severe atopic dermatitis and review the literature. [2021] Dupilumab, a biologic drug approved for the treatment of moderate-to-severe atopic dermatitis, has been associated with resolution or improvement in pre-existing alopecia areata. We report a case of significant improvement of AA after Dupilumab-onset treatment for atopic dermatitis and review the literature.

2. United States pubmed.ncbi.nlm.nih.gov

Long-Term Efficacy of Dupilumab in Alopecia Areata. [2022] BACKGROUND Dupilumab is a relatively new immune-modulating drug that has transformed the way clinicians treat common immunologic conditions, including atopic dermatitis, asthma, and chronic rhinosinusitis with nasal polyposis. Blocking signaling molecules involved within the Th2 immune response, dupilumab is a proven effective treatment for moderate to severe atopic dermatitis - a condition whose disease pathogenesis is heavily linked to the dysregulation of this immunologic pathway. Interestingly, dupilumab has found broader clinical utility, showing efficacy in treating other distinct dermatologic diseases, including alopecia areata. CASE REPORT A 16-year-old White male with a past medical history of moderate atopic dermatitis presented to our clinic with complete scalp hair, eyebrow, and eyelash loss. At this time, the patient was given a clinical diagnosis of alopecia totalis. Understanding that dupilumab has been previously used for treatment in adults of this specific autoimmune condition, we started this adolescent patient on dupilumab to concomitantly treat his atopic dermatitis and alopecia areata. The patient gradually experienced complete regrowth of his hair and almost complete resolution of his atopic dermatitis. Three years after starting dupilumab, the patient remains without signs of alopecia totalis. CONCLUSIONS This case report demonstrates the long-term efficacy of dupilumab use in alopecia areata. More investigation is required to understand dupilumab's broadening clinical indications. Additionally, this case highlights the complex relationship between dysregulation of the Th2 response and autoimmunity. Crosstalk between immune pathways within the disease spectrum of alopecia areata may explain why dupilumab has been reported to both treat and exacerbate alopecia areata.

3. United States pubmed.ncbi.nlm.nih.gov

Prurigo-like atopic dermatitis in a child with CARD11-associated severe combined immunodeficiency successfully treated with dupilumab. [2023] A 12-year-old boy affected by severe combined immunodeficiency due to a heterozygous variant in the CARD domain of CARD11, c.169G>A; p.Glu57Lys, developed severe atopic dermatitis and alopecia areata. After failure of conventional systemic therapy, dupilumab was administered at a dose of 400 mg subcutaneously, followed by 200 mg every 14 days. The patient had an excellent clinical response after 1 month and complete remission after a year, with the absence of side effects, demonstrating good efficacy and safety profile.

4. Italy pubmed.ncbi.nlm.nih.gov

Improvement of atopic dermatitis and alopecia universalis with dupilumab. [2022] Dupilumab is an interleukin-4 receptor alpha antagonist that showed significant improvement of atopic dermatitis (AD). Many reports have shown significant resolution of alopecia areata, alopecia universalis and alopecia totalis after dupilumab treatment for AD. We present one of reported cases that showed improvement of underlying alopecia universalis treated with dupilumab.

5. United States pubmed.ncbi.nlm.nih.gov

Reactivation of Alopecia Areata After Dupilumab Therapy for Atopic Dermatitis. [2022] We present a case report of a 42-year-old woman with severe atopic dermatitis (AD) and history of alopecia areata (AA), which had been in remission for more than 5 years. She was treated with dupilumab for AD, and 4 months after initiating treatment, she developed a rapidly progressive reactivation of AA in an androgenic alopecia pattern. Alopecia resolved after a single intramuscular triamcinolone injection while the patient remained on dupilumab. This is the seventh report to date of dupilumab-associated alopecia but is the first case documenting reactivation of previous AA. Alopecia occurring after dupilumab initiation raises suspicion for a possible unrecognized adverse effect of dupilumab in AD patients.

6. United States pubmed.ncbi.nlm.nih.gov

Short communication: Comments on hair disorders associated with dupilumab based on VigiBase. [2022] Dupilumab is a human antibody that blocks the signaling of both interleukin-4 and interleukin-13 receptors. It has been approved for the treatment of moderate-to-severe atopic dermatitis. However, several case reports have reported conflicting effects of dupilumab on alopecia.

7. England pubmed.ncbi.nlm.nih.gov

Safety update: dupilumab and ocular adverse reactions. [2022] Overview of: Medicines and Healthcare products Regulatory Agency. Dupilumab (Dupixent&#9660;): risk of ocular adverse reactions and need for prompt management. Drug Safety Update 2022;16(4): 1.