A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome

NCT04244006 | Unknown Phase 2

• 1 other identifier: RC31/19/0045
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

Brief Summary

To date, there are no effective therapy for the management of Netherton Syndrome (NS) Patients use emollients with a limited efficacy on scaling and no efficacy on skin inflammation and pruritus. They may also use topical corticosteroids or calcineurin inhibitors in case of eczematous lesions. The use of therapies targeting skin inflammation has been reported in a few case reports. Their efficacy is very limited and their uses are limited because of the chronicity of the disease, the impaired skin barrier function and the risk for skin infections and skin cancers. Therefore, there is a huge medical need for novel therapies in NS.The expected consequences of this study are that a 16-week course of dupilumab will be more effective than placebo for the treatment of moderate to severe NS Dupilumab could therefore improve skin condition and quality of life.

Conditions

Netherton Syndrome

Outcome Measures

Primary Outcomes (1)

• The severity of the disease of the Netherton Area Severity Assessment score (NASA).

  NASA score

  Day 0 and week 16

Secondary Outcomes (9)

• Clinical efficacy severity of pruritus and pain

  Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

• Presence of infections (adverse event)

  Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

• Quantity of dermocorticosteroids used between each visit will be evaluated by questioning the patient.

  Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

• QOL score

  Day 0, Week 16 and 28

• Skin inflammation

  Day 0 and week 16

Study Arms (2)

Dupilumab

EXPERIMENTAL

The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of Dupilumab 300 mg (syringe of 2 mL for subcutaneous administration).

Drug: Dupilumab Prefilled Syringe

Placebo

PLACEBO COMPARATOR

The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of placebo (syringe of 2 mL for subcutaneous administration)..

Other: Placebo Prefilled Syringe

Interventions

Dupilumab Prefilled Syringe DRUG

administration of dupilumab corresponding to dupilumab arm

Dupilumab

Placebo Prefilled Syringe OTHER

administration of placebo corresponding to placebo arm

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult patients affiliated to a social insurance protection regimen.
• Clinical diagnosis of NS (7) (trichorrhexis invaginata, extensive scaling, skin inflammation, allergic manifestations) and absent or marked reduction of LEKTI staining.
• Patients able to understand the study procedures including the ability to complete patient-based self-assessment questionnaires.
• Patients who agree to sign the written informed consent.

You may not qualify if:

• Hypersensitivity to dupilumab or its excipients.
• Any other condition that according to the investigator will impair the ability to evaluate treatment effect.
• Known or suspected history of immunosuppression, including history of invasive opportunistic infections (e.g., tuberculosis, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystis, aspergillosis).
• Current infections including infection with helminthes.
• Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study. Women of childbearing age, potentially sexually active, and unwilling to use adequate birth control methods.
• Mental or physical incapacity to fill in the questionnaires.

Sponsors & Collaborators

• University Hospital, Toulouse: lead
• MedSharing: collaborator

Study Sites (2)

Dermatologie Necker

Paris, 75015, France

NOT YET RECRUITING

Dermatology

Toulouse, France

RECRUITING

MeSH Terms

Conditions

Netherton Syndrome

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Juliette MAZEREEUW-HAUTIER

  Toulouse Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nadège ALGANS

CONTACT

0561777204; algans.n@chu-toulouse.fr

Helene TEXIER

CONTACT

texier.h@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Dupilumab and placebo will be provided in identically matching 2 mL pre-filled syringes. To protect the blind, each treatment kit of 2 mL (dupilumab/placebo) glass pre-filled syringes will be prepared such that the treatments (dupilumab and its matching placebo) are identical and indistinguishable and will be labeled with a treatment kit number.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: one arm with dupilumab (2/3 of patients) and one arm with placebo (1/3 of patients)

Study Record Dates

• First Submitted: January 13, 2020
• First Posted: January 28, 2020
• Study Start: July 23, 2020
• Primary Completion: June 1, 2024
• Study Completion: June 1, 2024
• Last Updated: August 23, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)