Natural History of Netherton Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

12 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-10-09

Study Completion Date

2025-03-31

Brief Summary

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The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).

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Detailed Description

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Conditions

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Netherton Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Patients diagnosed with Netherton Syndrome

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. Confirmed diagnosis of NS by at least one of the following:

* Genetic testing of mutations in Serine Protease Inhibitor of Kazal Type 5 (SPINK5);
* Absence or major deficiency of the protein Lympho-Epithelial Kazal-Type-Related Inhibitor (LEKTI) in skin biopsy;
* Clinical assessment (signs and symptoms).
2. Provision of consent or assent (i.e., by parent or legal guardian) as required by local regulations:

* \[Part 1\] to authorise access to existing medical records for study data collection;
* \[Part 2\] to participate in the longitudinal 52-week evaluation of disease severity and clinical outcome assessments.

\[for Part 2 only\]
3. Not participating in a clinical trial at the time of study enrolment for Part 2.

Exclusion Criteria

1. Patient who has died prior to 2002.
2. Patient whose last known survival status is dated prior to 2002 (i.e., patient has been lost to clinical follow-up since 2002).

Eligible Sex

ALL

Accepts Healthy Volunteers

No