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}

Research Team

Emma Guttman-Yassky

Principal Investigator

Icahn School of Medicine at Mount Sinai

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

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

Trials

933

Recruited

579,000+

Dr. Brendan Carr

Icahn School of Medicine at Mount Sinai

Chief Executive Officer since 2024

MD, MA, MS

Dr. Vicki LoPachin

Icahn School of Medicine at Mount Sinai

Chief Medical Officer

MD, FACP, MBA

Findings from Research

Dupilumab, a biologic drug used for moderate-to-severe atopic dermatitis, has shown potential in improving alopecia areata, a condition characterized by hair loss.

The case reported indicates significant improvement in alopecia areata symptoms after starting Dupilumab treatment for atopic dermatitis, suggesting a possible beneficial effect of this medication beyond its primary use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improvement of alopecia areata with Dupilumab in a patient with severe atopic dermatitis and review the literature. Magdaleno-Tapial, J., Valenzuela-Oñate, C., García-Legaz-Martínez, M., et al.[2021]

Dupilumab, an immune-modulating drug, has shown long-term efficacy in treating alopecia areata in a 16-year-old patient, leading to complete hair regrowth and significant improvement in atopic dermatitis over three years.

This case suggests that dupilumab's mechanism of action, which involves blocking Th2 immune response signaling, may have broader applications in treating autoimmune conditions, although further research is needed to fully understand its effects on alopecia areata.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-Term Efficacy of Dupilumab in Alopecia Areata. Kulkarni, M., Rohan, CA., Travers, JB., et al.[2022]

A 12-year-old boy with severe combined immunodeficiency and severe atopic dermatitis showed excellent clinical improvement after receiving dupilumab, achieving complete remission within a year.

The treatment was well-tolerated with no reported side effects, indicating a favorable safety and efficacy profile for dupilumab in this patient.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prurigo-like atopic dermatitis in a child with CARD11-associated severe combined immunodeficiency successfully treated with dupilumab. Gualdi, G., Lougaris, V., Amerio, P., et al.[2023]

Dupilumab, an interleukin-4 receptor alpha antagonist, has been shown to significantly improve atopic dermatitis (AD).

In addition to its effects on AD, dupilumab treatment has also led to notable resolution of alopecia areata, alopecia universalis, and alopecia totalis, indicating its potential as a treatment for these hair loss conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Improvement of atopic dermatitis and alopecia universalis with dupilumab. Alotaibi, L., Alfawzan, A., Alharthi, R., et al.[2022]

A 42-year-old woman with severe atopic dermatitis experienced a reactivation of alopecia areata after starting treatment with dupilumab, a medication for her dermatitis, which had been in remission for over 5 years.

This case suggests that dupilumab may have an unrecognized adverse effect of triggering alopecia areata in patients, highlighting the need for further investigation into its safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Reactivation of Alopecia Areata After Dupilumab Therapy for Atopic Dermatitis. Carnicle, JM., Hendricks, AJ., Shi, VY.[2022]

In a study analyzing 20,548 adverse event reports related to dupilumab, 462 cases (2.2%) were linked to hair disorders, primarily hair loss, indicating a notable side effect of this medication.

Dupilumab was found to be associated with hair disorders more frequently in women and showed a higher reporting rate in Europe, suggesting a need for further investigation into its effects on hair health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Short communication: Comments on hair disorders associated with dupilumab based on VigiBase. Park, S., Park, SH., Byun, YJ., et al.[2022]

Dupilumab (Dupixent) has been associated with a risk of ocular adverse reactions, highlighting the importance of monitoring patients for eye-related side effects during treatment.

Prompt management of any ocular issues is essential for patients receiving dupilumab, ensuring their safety and well-being while benefiting from the medication.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety update: dupilumab and ocular adverse reactions. [2022]

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]

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

Long-Term Efficacy of Dupilumab in Alopecia Areata. [2022]

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]

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

Improvement of atopic dermatitis and alopecia universalis with dupilumab. [2022]

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

Reactivation of Alopecia Areata After Dupilumab Therapy for Atopic Dermatitis. [2022]

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

Short communication: Comments on hair disorders associated with dupilumab based on VigiBase. [2022]

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

Safety update: dupilumab and ocular adverse reactions. [2022]