A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in… (NCT05979831) | Clinical Trial Compass
TerminatedPhase 1/2
A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome
Stopped: The study was terminated based on a business decision by the Sponsor.
France9 participantsStarted 2023-09-28
Plain-language summary
Netherton Syndrome (NS) is a severe rare disease characterized by generalized scaling, erythema, and epidermal barrier defects. This study assessed the safety, pharmacokinetics (PK), and efficacy of DS-2325a in patients with NS.
Who can participate
Age range18 Years – 65 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male or female participants aged 18 to 65 years with clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria:
* Neonatal erythroderma
* Bamboo hair and/or alopecia
* Chronic atopy specified as food allergy and/or asthma and/or rhino-conjunctivitis and/or eczema for at least 2 years
* Ichthyosis linearis circumflexa or scaling erythroderma or equivalent
* Immunohistochemistry documentation of absence of LEKTI in the skin or confirmed SPINK5 gene mutations
* NS involvement of ≥20% of Body Surface Area (BSA)
* Patients must give written informed consent to participation in the study prior to Screening
* Participants must be willing and able to understand and comply with study requirements
* Participants must be willing to have skin tape harvests collected from lesional and nonlesional 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
* Concomitant systemic disease not controlled by treatment. Stability for 3 months prior to Screening is required
* Kidney or liver disease with significant impairment of organ function (creatinine clearance \<30 mL/min, calculated using the Cockcroft-Gault Equation, and Child-Pugh Class C; ALT and AST \>2 × ULN range; total bilirubin \>1 × ULN).
* Concomitant disease or condition that m…
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events