Dupilumab for Itchy Skin Diseases

Overseen By Amy Paller

Age: Any Age

Sex: Any

Trial Phase: Phase < 1

Sponsor: Northwestern University

Must not be taking: Immunosuppressives, Biologics, Retinoids, others

Disqualifiers: Ichthyosis vulgaris, Active infections, Malignancies, others

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

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: Unknown

Duration: 16 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called dupilumab (an injectable medication) to determine its effectiveness in reducing severe itch in people with genetic skin disorders. Researchers aim to find out if dupilumab can improve itching and ensure its safety for individuals with various skin conditions. The trial includes a treatment phase and a possible long-term extension for those who benefit. Individuals with a genetic skin disorder experiencing significant itching may be suitable candidates for this study. As an Early Phase 1 trial, this research focuses on understanding how dupilumab works in people, offering participants the chance to contribute to groundbreaking research.

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

Yes, you may need to stop certain medications. You must stop using immunosuppressive/immunomodulating drugs, systemic anti-inflammatory medication, or phototherapy at least 4 weeks before the trial. Other biologics must be stopped within 5 half-lives or 16 weeks before the trial. Topical or systemic retinoids and topical anti-inflammatory agents must be started at least 4 weeks before the trial and continued throughout. Consult with the study investigator for specific guidance.

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

Research has shown that dupilumab is generally safe for people with skin conditions. In one study involving adults with moderate-to-severe atopic dermatitis, participants who received dupilumab showed significant improvement without serious safety issues. Specifically, over 41% of these patients experienced a noticeable reduction in itchiness, compared to just 12% of those who received a placebo.

The treatment has already been approved for certain skin conditions, suggesting general safety. While this approval doesn't guarantee safety for all skin disorders, it offers some reassurance. Discussing potential risks and benefits with a healthcare provider is important before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Dupilumab is unique because it targets a specific pathway involved in inflammatory responses by blocking the interleukin-4 (IL-4) and interleukin-13 (IL-13) signaling. These pathways are often overactive in itchy skin diseases like eczema. Unlike standard treatments that might rely on general immunosuppressants or topical steroids, Dupilumab provides a more targeted approach, potentially reducing inflammation with fewer side effects. Researchers are excited about Dupilumab because it offers a novel way to tackle the underlying causes of itchiness, which could lead to more effective and lasting relief for patients.

What evidence suggests that dupilumab might be an effective treatment for itchy skin diseases?

Research has shown that dupilumab, the treatment under study in this trial, can effectively reduce severe itching in skin conditions. One study found that 41% of adults using dupilumab experienced a significant decrease in itching, compared to just 12% who took a placebo. Another study reported that 58.8% of patients noticed an improvement in itching after 24 weeks of using dupilumab. Furthermore, after one year of treatment, 81% of patients achieved the best possible relief from itching. These results suggest that dupilumab can greatly improve itching symptoms in various skin disorders. ^{1 5 6 7 8}

Who Is on the Research Team?

Amy Paller

Principal Investigator

Northwestern University

Are You a Good Fit for This Trial?

This trial is for individuals over 6 months old with severe itch due to genetic skin disorders, who have an average itch score of ≥4. Participants must not have ichthyosis vulgaris as the only disorder, no recent serious infections or malignancies, and should not be on certain medications like biologics or immunosuppressants recently.

Inclusion Criteria

Subject, parent/caregiver or legal guardians, as appropriate, are able to understand and complete the study requirements and study-related questionnaires

I have had an average itchiness of 4 or more, with it reaching at least 5 at its worst in the past week.

Must be willing and able to adhere to the prohibitions and restrictions specified in this protocol

Exclusion Criteria

I am under 6 months old.

I can give my own consent or have someone legally allowed to do so if I'm under 18.

I haven't used certain medications, including steroids or immunosuppressants, within the last 4 weeks or longer.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive dupilumab during a 16-week open-label phase to assess changes in itch

16 weeks

Long-term extension

Participants who qualify and wish to continue receive dupilumab for an additional 20 months

20 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Dupilumab

Trial Overview The study tests Dupilumab's effectiveness in reducing itch in various pruritic genetic inflammatory skin conditions. It includes a 16-week open-label phase followed by a long-term extension up to 26 months for those responding positively and willing to continue.

How Is the Trial Designed?

Group I: Open-Label Dupilumab Experimental Treatment 1 Intervention

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?

Northwestern University

Lead Sponsor

Trials

1,674

Recruited

989,000+

Published Research Related to This Trial

In a study of 100 patients with atopic dermatitis, 36% of those treated with dupilumab developed ocular diseases, significantly higher than the 10% in the reference group.

Severe allergic conjunctivitis and blepharitis were notably more common in the dupilumab group, with 30% and 22% of patients affected, respectively, indicating a need for careful monitoring and potential ophthalmologist consultation before starting treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prevalence of ocular disease during dupilumab treatment for atopic dermatitis: a bicentric retrospective comparative cohort study. Barbé, J., Poreaux, C., Remen, T., et al.[2021]

Dupilumab, a monoclonal antibody that inhibits the Th2 signaling pathway, is used to treat atopic dermatitis but can lead to the development of psoriasis in some patients, as seen in a case study of a 40-year-old man.

In this case, the abrupt discontinuation of oral steroid therapy alongside dupilumab treatment was identified as potential triggers for the onset of psoriasis, highlighting the need for careful management when using this medication.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Psoriasis in dupilumab-treated atopic dermatitis]. Senner, S., Eicher, L., Aszodi, N., et al.[2020]

In a study of 818 adults with moderate-to-severe atopic dermatitis treated with dupilumab for at least 16 weeks, 12% of patients successfully spaced their doses or withdrew treatment while still maintaining effectiveness.

Key predictors for successful dose spacing or treatment withdrawal included having non-cutaneous atopic manifestations, a prurigo nodularis phenotype, and being older at the start of treatment, suggesting specific patient profiles can benefit from adjusted treatment regimens.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Identification of clinical predictors for dupilumab dose spacing in adults with atopic dermatitis: a real-world study. Chiricozzi, A., Dal Bello, G., Gori, N., et al.[2023]

Dupilumab is an effective treatment for atopic dermatitis, showing significant improvements in skin symptoms and overall disease control in clinical trials.

The medication works by inhibiting specific pathways in the immune system, which helps reduce inflammation and alleviate the symptoms of atopic dermatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab (Dupixent): An Interleukin-4 Receptor Antagonist for Atopic Dermatitis. D'Ippolito, D., Pisano, M.[2020]

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]

In a study of 46 adult patients with moderate-to-severe atopic dermatitis treated with dupilumab, 34.8% experienced ocular adverse events (OAEs), primarily mild to moderate dry eye disease and superficial punctate keratitis.

Patients with pre-existing conditions such as dry eye disease, eyelid eczema, or high IgE levels were more likely to develop OAEs, suggesting that these factors should be considered before starting dupilumab treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Incidence and risk factors for dupilumab associated ocular adverse events: a real-life prospective study. Touhouche, AT., Cassagne, M., Bérard, E., et al.[2021]

Dupilumab is a monoclonal antibody that targets the IL-4 receptor, effectively inhibiting the action of IL-4 and IL-13, which are key players in allergic diseases.

It received its first approval in March 2017 for treating moderate-to-severe atopic dermatitis in adults who do not respond well to topical treatments, and is also being developed for asthma, nasal polyps, and eosinophilic esophagitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab: First Global Approval. Shirley, M.[2022]

Dupilumab, a monoclonal antibody approved by the FDA in 2017, effectively treats moderate to severe atopic dermatitis by inhibiting IL-4 and IL-13 signaling, providing an alternative for patients not responding to topical therapies.

While dupilumab is generally effective, it can cause adverse effects such as injection site reactions and conjunctivitis, with rare cases like alopecia areata reported, highlighting the need for monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dupilumab side effect in a patient with atopic dermatitis: a case report study. Albader, SS., Alharbi, AA., Alenezi, RF., et al.[2020]

Citations

1. dupixenthcp.com dupixenthcp.com/atopicdermatitis/efficacy-safety/pruritus-nrs-clinical-trial

Adult Peak Pruritus NRS Efficacy Results 41 % of adults treated with DUPIXENT (n=213) achieved ≥4-point reduction in Peak Pruritus NRS vs 12% with placebo at Week 16 in SOLO 1 (n=212; P<0.001)

2. sciencedirect.com sciencedirect.com/science/article/pii/S2666328724000683

Dupilumab improves pruritus and skin lesions in patients ... At week 24, 90 (58.8%) dupilumab-treated vs 30 (19.0%) placebo-treated patients achieved clinically meaningful improvement in itch, 71 (46.4%) vs 27 (17.1%) ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02277769

NCT02277769 | Study of Dupilumab (REGN668/ ... This is a randomized, double-blind, placebo-controlled, parallel-group study to confirm the efficacy and safety of Dupilumab monotherapy in adults with moderate ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40971025/

Optimal Itch Response in Adults Treated with Dupilumab ... After 24 weeks of dupilumab treatment, 77% of patients achieved optimal itch response. This improved to 81% by week 52. The majority of patients ...

5. newsroom.regeneron.com newsroom.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-data-revolutionizing-atopic-dermatitis-rad

Dupixent® (dupilumab) Data at Revolutionizing Atopic ... Dupixent achieved 75% or greater improvement in overall disease severity, the primary endpoint, in more than three-quarters of treated patients.

6. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK565242/table/cl.app1.tab3/

Clinical Review Report: Dupilumab (Dupixent) - NCBI Bookshelf Indicated for the treatment of patients aged 12 years and older with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02210780

NCT02210780 | Study of Dupilumab and Immune ... This was a 32-week, randomized, double-blind, placebo-controlled, parallel-group study assessing immunization responses to vaccination in adults with moderate ...

8. cda-amc.ca cda-amc.ca/sites/default/files/cdr/clinical/sr0636-dupixent-clinical-review-report.pdf

Clinical Review Report - DUPILUMAB (DUPIXENT) Indication: Indicated for the treatment of patients aged 12 years and older with moderate-to-severe atopic dermatitis whose disease is not adequately controlled ...