Identification of Genetic Variants Associated With Lichen Sclerosus (GENITALS)
Lichen Sclerosus (LS) is a common genital skin condition that severely impacts on daily living. LS occurs worldwide but may be more common in the white population. The extragenital skin is involved in about 10% of reported patients, exact numbers are not known. LS is estimated to affect 0.1-0.3% of new patients in a general hospital patient population and 1.7% of patients referred to general gynaecological practice, however, the exact prevalence and incidence is not known. LS has a major impact on the quality of life, as symptoms of itching, pain and discomfort can make it difficult to sit, walk and go to the toilet. Having sex becomes painful because of erosions and fissures (break down of the skin), sometimes impossible because of irreversible fusion (sticking together) and sclerosis (hardening) of the genital skin. There is an increased risk of genital cancer in individuals with LS, this seems higher in familial cases. Next to a genetic background leading to a dysregulation of the immune system, certain external trigger mechanisms seem to play an important role in the development of LS.
In this project the investigators propose to identify pathogenic variants in novel protein-coding genes that may be involved in Lichen sclerosus using samples from families with members manifesting LS. Through elucidating underlying pathomechanims which have not yet been fully explored the development of novel treatments may be possible.
調査の概要
詳細な説明
The project plan is to identify genetic variants associated with lichen sclerosus. The investigators intend to identify differences in the genome in family members affected by LS and those who are not affected by LS.
The investigators hypothesize that enrollment and sequencing the genome of families with LS would lead to the discovery of novel genetic factors underlying LS. "Success" as measured by discovery of novel Mendelian genes will be inherent to our capacity to recruit a large number of families preferably with multiple affected individuals. This "opportunistic" and "somewhat untargeted" approach is essential to reach our aim of identifying novel LS-associated genes.
The investigators' project does not have classical primary and secondary endpoints as one or multiple parameters are not followed and because the study is not performed within a clinical trial frame. The investigators' primary endpoint will be having sequenced the exome/genome of families with LS and solved the segregation of variants of interest in the rest of the pedigree, "Number of participants with potential LS genes". The secondary endpoint will be the identification of novel LS genes, "Number of genes associated with LS identified".
研究の種類
入学 (推定)
連絡先と場所
研究連絡先
- 名前:Gudula Kirtschig, Medical doctor
- 電話番号:0041527230202
- メール:g.kirtschig@gmail.com
研究連絡先のバックアップ
- 名前:Hirotsugu Oda, Prof. Dr.
- 電話番号:+49 221 478 84088
- メール:hoda@uni-koeln.de
研究場所
-
-
Thurgau
-
Frauenfeld、Thurgau、スイス、8500
- 募集
- Medbase
-
コンタクト:
- Gudula Kirtschig, medical doctor
- 電話番号:0041 527230202
- メール:g.kirtschig@gmail.com
-
-
参加基準
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
サンプリング方法
調査対象母集団
説明
Inclusion Criteria:
- Individual with clinical or /and histological Lichen sclerosus
- Family member of an individual with lichen sclerosus
Exclusion Criteria:
- No Family member with lichen sclerosus
研究計画
研究はどのように設計されていますか?
デザインの詳細
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Identification of novel Lichen sclerosus genes
時間枠:5 years
|
Our project does not have classical primary and secondary endpoints as we are not following one or multiple parameters and because we are not within a clinical trial frame.
Our primary endpoint will be having sequenced the exome/genome of families with LS and solved the segregation of variants of interest in the rest of the pedigree "Number of Participants with potential LS genes".
The secondary endpoint will be the identification of novel LS genes "Number of genes associated with LS identified".
|
5 years
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Novel Lichen sclerosus genes
時間枠:5 years
|
The secondary endpoint will be the identification of novel LS genes "Number of genes associated with LS identified".
|
5 years
|
その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Novel LS genes
時間枠:5 years
|
Our project does not have classical primary and secondary endpoints as we are not following one or multiple parameters and because we are not within a clinical trial frame.
Our primary endpoint will be having sequenced the exome/genome of families with LS and solved the segregation of variants of interest in the rest of the pedigree.
The secondary endpoint will be the identification of novel LS genes.
|
5 years
|
協力者と研究者
スポンサー
協力者
捜査官
- 主任研究者:Gudula Kirtschig, Dr.、Medbase
出版物と役立つリンク
一般刊行物
- Kirtschig G, Kinberger M, Kreuter A, Simpson R, Gunthert A, van Hees C, Becker K, Ramakers MJ, Corazza M, Muller S, von Seitzberg S, Boffa MJ, Stein R, Barbagli G, Chi CC, Dauendorffer JN, Fischer B, Gaskins M, Hiltunen-Back E, Hofinger A, Kollmann NH, Kuhn H, Larsen HK, Lazzeri M, Mendling W, Nikkels AF, Promm M, Rall KK, Regauer S, Sardy M, Sepp N, Thune T, Tsiogka A, Vassileva S, Voswinkel L, Wolber L, Werner RN. EuroGuiderm guideline on lichen sclerosus-Treatment of lichen sclerosus. J Eur Acad Dermatol Venereol. 2024 Oct;38(10):1874-1909. doi: 10.1111/jdv.20083. Epub 2024 Jun 1.
- Kirtschig G, Kinberger M, Kreuter A, Simpson R, Gunthert A, van Hees C, Becker K, Ramakers MJ, Corazza M, Muller S, von Seitzberg S, Boffa MJ, Stein R, Barbagli G, Chi CC, Dauendorffer JN, Fischer B, Gaskins M, Hiltunen-Back E, Hofinger A, Kollmann NH, Kuhn H, Larsen HK, Lazzeri M, Mendling W, Nikkels AF, Promm M, Rall KK, Regauer S, Sardy M, Sepp N, Thune T, Tsiogka A, Vassileva S, Voswinkel L, Wolber L, Werner RN. EuroGuiderm guideline on lichen sclerosus-introduction into lichen sclerosus. J Eur Acad Dermatol Venereol. 2024 Oct;38(10):1850-1873. doi: 10.1111/jdv.20082. Epub 2024 Jun 1.
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- BASEC Nr. 2025-01049
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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