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 range
18 Years – 65 Years
Sex
ALL
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment-emergent Adverse Events
Timeframe: Screening (Day 0) up to Week 45 (end of study)