NCT05732987

Brief Summary

This study is to identify rare, disease-causing mutations of several rare neutrophil dermatoses. To identify associations between NMID and variants in the genome next generation sequencing, mainly whole exome sequencing, will be used. In a second approach the expression level of already known inflammatory proteins in skin samples will be investigated.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
3,370

participants targeted

Target at P75+ for all trials

Timeline
40mo left

Started Feb 2023

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress50%
Feb 2023Sep 2029

First Submitted

Initial submission to the registry

January 30, 2023

Completed
4 days until next milestone

Study Start

First participant enrolled

February 3, 2023

Completed
14 days until next milestone

First Posted

Study publicly available on registry

February 17, 2023

Completed
6.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2029

Last Updated

August 22, 2023

Status Verified

August 1, 2023

Enrollment Period

6.6 years

First QC Date

January 30, 2023

Last Update Submit

August 21, 2023

Conditions

Keywords

inflammasomeinflammation-driving pathwaygene variantsnext generation sequencing

Outcome Measures

Primary Outcomes (1)

  • Number of protein-coding rare variants associated with forms of NMID

    The primary endpoint consists in the determination of association between newly identified or previously reported rare gene variants and one or more forms of NMIDs. The discovery of such genetic variants will lead to the identification of defective molecular mechanisms involved in abnormal cutaneous immune reactions in these patients: - Statistically significant association between genetic data and NMID * Detection of protein-coding rare variants associated with forms of NMID * Identification of inflammasome activation in different stages of NMID

    one time assessment at baseline

Secondary Outcomes (5)

  • Imaging Mass Cytometry

    one time assessment at baseline

  • RNA expression

    one time assessment at baseline

  • Immune cell count

    one time assessment at baseline

  • Rate of mean fluorescence intensity of immune cells

    one time assessment at baseline

  • Protein quantification (ELISA)

    one time assessment at baseline

Study Arms (4)

Biobank- samples from NMID patients

600 samples from NMID patients from the Biobank Dermatology Unispital Basel (USB) (Biobank USB) and from the biobank of the Dermatology Department of the University Hospital Zürich (USZ) (Biobank USZ) will be included.

Other: Analysis of samples

Formalin-fixed and paraffin-embedded (FFPE) samples

About 50 of formalin-fixed and paraffin-embedded (FFPE) samples from the Dermatology USB collected before 2014 for RNA and protein expression analyses will be reused. The patients in questions are informed about the study and the coded use of their samples.

Other: Analysis of samples

Biobank- samples from controls

2'700 anonymized control genomes as well as 150 anonymized control samples for the proteomics approach can be used as control samples from the biobank of the Dermatology Department of the University Hospital Zürich (Biobank Dermatology USZ).

Other: Analysis of samples

Fresh skin samples from healthy donors

A maximum of 20 fresh skin samples from healthy donors are required per skin location. They will be requested from healthy volunteers after information about the study and receiving the informed consent.

Other: Analysis of samples

Interventions

DNA extraction from blood or saliva samples for the identification of gene variants by next generation sequencing;

Biobank- samples from NMID patientsBiobank- samples from controlsFormalin-fixed and paraffin-embedded (FFPE) samplesFresh skin samples from healthy donors

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

The study participants will largely be recruited by a.) prospectively collected biopsies of patients from biobanks or b.) analyzing, contacting, and including/excluding the existing patient population of our clinic (FFPE tissues). Healthy skin is obtained from patients undergoing plastic surgery at the USB. These patients will be informed and their informed consent obtained before surgery.

You may qualify if:

  • written consent of the participating person
  • diagnosis of a disease in the NMID form group or proband of the control group

You may not qualify if:

  • Missing informed consent if samples collected after 2014
  • no diagnosis of NMID
  • Missing informed consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Hospital Basel, Clinic of Dermatology

Basel, 4031, Switzerland

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

All samples originating from biobanks or Dermatology USB (fixed tissue) will be returned to origin after finishing of study.

Study Officials

  • Alexander Navarini, Prof. Dr. med.

    University Hospital Basel, Clinic of Dermatology

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Alexander Navarini, Prof. Dr. med.

CONTACT

Emmanuel Contassot, Dr.

CONTACT

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 30, 2023

First Posted

February 17, 2023

Study Start

February 3, 2023

Primary Completion (Estimated)

September 1, 2029

Study Completion (Estimated)

September 1, 2029

Last Updated

August 22, 2023

Record last verified: 2023-08

Locations