Genetic Variants in Linear Localized Scleroderma

NCT ID: NCT02222038

Last Updated: 2020-11-10

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-08-31

Study Completion Date

2017-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to investigate the genetic architecture of Linear Localized Scleroderma (LLS) (linear morphea) by whole exome sequencing.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

At present the etiology of LLS is unknown, but a genetic background is suspected. Although LLS clearly classifies as a mosaic disorder, its genetics and protein machinery remain to be understood.

We are going to use a tailored approach to identify the genetic factors of LLS. In the first phase of the study we will investigate the genetic architecture in LLS. WES will analyze whole protein coding DNA in skin samples of 50 consenting LLS patient. The aim is to identify the key genes associated with LLS. In the second phase of the study subsequent functional experiments will be performed. Based on the identified candidate genes, knockdown and overexpression models will be created with relevant cell lines (fibroblasts) to identify the biological consequences and confirm the functional relevance of the identified genetic mutations in LLS. Further the protein network active in LLS will be investigated (proteomic analysis).

The described basic genetic studies combined with functional experiments will lay the groundwork for treatment trials to provide possibly novel treatment options.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Morphea

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

skin biopsy

4mm punch biopsy

Group Type OTHER

skin biopsy

Intervention Type OTHER

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

skin biopsy

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Male or female subjects ≥ 5 years of age with well phenotyped LLS
* Affecting their head and / or face "termed " en coup de sabre " type LLS or Hemiatrophia faciei or Parry-Romberg syndrome, with or without therapy
* Affecting any site of the body except the head or face, with or without therapy

Exclusion Criteria

* Patients with signs of systemic scleroderma
* Patients with localized scleroderma (morphea) other than the linear type ("plaque-type", "morphea profunda", "generalized morphea") Patients with diagnosed gadolinium induced scleroderma Patients with post-irradiation scleroderma Patients with missing consent
Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

University Children's Hospital, Zurich

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Lisa Weibel, MD

Role: PRINCIPAL_INVESTIGATOR

Department Pediatric Dermatology University Children's Hospital Zurich

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University Children's Hospital, Department of Pediatric Dermatology

Zurich, , Switzerland

Site Status

University Hospital, Department of Dermatology

Zurich, , Switzerland

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Switzerland

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

KISPI-UZH-LLS-2014

Identifier Type: -

Identifier Source: org_study_id