An early-stage clinical study of a potential new medicine for patients with Netherton Syndrome

Phase 1

Recruiting

• Conditions: etherton's Syndrome; MedDRA version: 20.0Level: PTClassification code: 10062909Term: Netherton's syndrome Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: CTIS2022-502853-32-00
• Lead Sponsor: Daiichi Sankyo Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-03-31
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Male or female patients aged 18 to 65 years with clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria: 1. Neonatal erythroderma; 2. Bamboo hair and/or alopecia; 3. Chronic atopy specified as food allergy and/or asthma and/or rhino-conjunctivitis and/or eczema for at least 2 years; 4. Ichthyosis linearis circumflexa or scaling erythroderma or equivalent. Eligible participants are those who have received treatment in the past that did not work and so need the further care., Immunohistochemistry documentation of absence of lympho-epithelial Kazal-type-related inhibitor (LEKTI) in the skin or confirmed serine peptidase inhibitor of Kazal type 5 (SPINK5) gene mutations., NS involvement of =20% of Body Surface Area (BSA) at both Screening and Baseline., Patients must give written informed consent to participation in the study prior to Screening., Patients must be willing to have skin tape harvests collected from lesional and non-lesional skin areas

Exclusion Criteria

Any skin disease that may interfere with the diagnosis or evaluation of NS., Any infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, or antifungals within 2 weeks before Screening visit.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified