Dupilumab for Alopecia Areata

(PEDAL Trial)

Recruiting at 4 trial locations

Overseen By Benjamin Ungar, MD

Age: < 18

Sex: Any

Trial Phase: Phase 2

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 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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 16

Duration: 48 weeks

76 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Dupilumab for alopecia areata, a condition causing hair loss, often in patches, on the scalp. The goal is to determine if Dupilumab can help individuals with moderate to severe hair loss regrow hair. Participants will receive either the actual treatment or a placebo (a substance with no active ingredients) through injections. Those with alopecia areata affecting 30% or more of their scalp and who have experienced some hair regrowth in the past seven years might be suitable for the trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important findings.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that dupilumab is generally safe for people. In studies treating atopic dermatitis (a skin condition that causes itchiness and redness), most side effects were mild, and no serious health problems were directly linked to the treatment. Another study involving individuals with alopecia areata (a condition that leads to hair loss) also found no serious side effects from dupilumab. While researchers continue to test dupilumab for alopecia areata, its use for other conditions suggests it is relatively safe. This should offer some reassurance about its safety for new participants, although individual experiences may vary. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for alopecia areata?

Unlike the standard treatments for alopecia areata, which often include corticosteroids and topical immunotherapies, Dupilumab offers a novel approach by targeting interleukin-4 and interleukin-13 pathways involved in inflammation. Researchers are excited about Dupilumab because it is already used successfully for other inflammatory conditions like eczema and asthma, suggesting it could be effective for alopecia areata too. Additionally, Dupilumab is administered through subcutaneous injections, providing a systemic treatment option that might offer more consistent results compared to topical treatments.

What evidence suggests that Dupilumab might be an effective treatment for alopecia areata?

Research suggests that Dupilumab, which participants in this trial may receive, can help treat alopecia areata (AA), a condition that causes hair loss. In one study, 70% of patients experienced hair regrowth, with new hair initially appearing white before darkening. Another study showed that a patient achieved almost full regrowth of scalp hair within eight months. Although more information is still being gathered, these early results indicate that Dupilumab might effectively help people with AA regrow their hair. ^{1 3 5 6 7}

Who Is on the Research Team?

Emma Guttman-Yassky

Principal Investigator

Icahn School of Medicine at Mount Sinai

Are You a Good Fit for This Trial?

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

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

I weigh at least 30 kilograms.

I have had severe hair loss covering more than half of my scalp for at least 6 months.

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Dupilumab
• Placebo

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.

How Is the Trial Designed?

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

Who Is Running the Clinical Trial?

Icahn School of Medicine at Mount Sinai

Lead Sponsor

Trials

933

Recruited

579,000+

Published Research Related to This Trial

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]

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]

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]

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, 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]

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]

Citations

1. tandfonline.com tandfonline.com/doi/full/10.1080/09546634.2024.2312245

Dupliumab therapy for alopecia areata: a case series and ... Notably, seven patients (70%) had white hair regrowth, with the white hair slowly decreasing over time and the proportion of pigmented black ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12293969/

Alopecia Areata Associated with Dupilumab The primary outcome assessed was the development of AA. As dupilumab can take up to 16 weeks to fully take effect, outcomes within 16 weeks of ...

3. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/dupilumab-regeneron-pharmaceuticals-alopecia-areata-likelihood-of-approval/

Dupilumab by Regeneron Pharmaceuticals for Alopecia ... According to GlobalData, Phase II drugs for Alopecia Areata have a 48% phase transition success rate (PTSR) indication benchmark for progressing ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT03359356

Treatment of Alopecia Areata (AA) With Dupilumab in ... Alopecia areata is a medical condition, in which the hair falls out in patches. The hair can fall out on the scalp or elsewhere on the face and body.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9235915/

Long-Term Efficacy of Dupilumab in Alopecia Areata - PMC Within 8 months of starting dupilumab, the patient had almost complete regrowth of his scalp hair, as seen in Figure 2B. After almost 3 years on this regimen, ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT02277769

NCT02277769 | Study of Dupilumab (REGN668/ ... Laboratory safety of dupilumab in moderate-to-severe atopic dermatitis: results from three phase III trials (LIBERTY AD SOLO 1, LIBERTY AD SOLO 2, LIBERTY ...

7. annallergy.org annallergy.org/article/S1081-1206(25)00203-0/fulltext

Patient outcomes and safety of combination biologic ... Combination biologic therapy with dupilumab seems to be well-tolerated, with most adverse effects being mild and no severe events directly attributed to ...