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Parallel Sequencing of Fetal Genome and RNA in the Presence of Ultrasound Warning Signs: a Complementary Approach for the Prenatal Diagnosis of Rare Diseases. (PrenOmics)

2026年8月25日 更新者:Assistance Publique - Hôpitaux de Paris

Parallel Sequencing of the Fetal Genome and RNA in the Presence of Ultrasound Warning Signs: a Complementary Approach for the Prenatal Diagnosis of Rare Diseases.

Prenatal exome sequencing (ES) is increasingly used for fetuses with ultrasound-detected anomalies but yields 10-15% variants of uncertain significance (VUS), limiting diagnostic performance, particularly in prenatal settings with incomplete phenotypes.

This study aims to evaluate the added value of combined prenatal genome sequencing (GS) and RNA sequencing (RNA-Seq), which are not currently part of routine care. Conducted at AP-HP, it will compare the diagnostic yield of GS + RNA-Seq with the current standard approach (chromosomal microarray analysis + ES), according to variant type (coding, non-coding, and structural). The contribution of systematic RNA-Seq to rapid VUS resolution will be specifically assessed.

Overall, this project will assess the feasibility, diagnostic performance, and clinical utility of implementing GS + RNA-Seq in prenatal diagnosis, supporting future integration into routine care in France.

調査の概要

詳細な説明

Couples will be enrolled during a "pre-test" genetic consultation by a physician or a research-trained collaborator (genetic counselor or another investigator).

As part of routine clinical care: chromosomal microarray analysis (CMA) and exome sequencing (ES) will be performed locally, with interpretation by a biologist from the site providing follow-up to the couple.

For research purposes, after obtaining written consent from both the pregnant woman and her partner: samples will be prepared for pre-analytical processing at Pitié-Salpêtrière and sequenced at SeqOIA. Interpretation will be conducted by a biologist at a site different from the one performing the exome analysis, using the Gleaves-P interface dedicated to the project (MOABI).

Transcriptomic analysis (RNA-Seq) will be performed on RNA extracted from amniotic fluid cultures, with and without emetine (a NMD inhibitor). Sequencing will be carried out after Agilent capture at the Genetics Laboratory of Necker-Enfants Malades Hospital (AP-HP). Bioinformatics analysis will be performed by the Imagine platform. Study biologists will interpret results using the PolyRNASEQ interface (qualitative splice junction analysis) and IGV (semi-quantitative analysis and expression).

All results from CMA + ES and GS + RNA-Seq will be reviewed during weekly meetings. In cases of discordant or unexpected results (anticipated for ~10 couples), a reference method will be applied to provide clinically validated results through an accredited prenatal diagnostic center, ensuring no loss of care opportunity.

Results will be discussed in a multidisciplinary prenatal diagnosis meeting (RCP) and integrated into the prognostic discussion. Couples will be seen during a "post-test" genetic consultation for communication of results.

For couples included at Necker-Enfants Malades Hospital only: two additional maternal blood tubes will be collected during routine pregnancy follow-up before amniocentesis. Circulating DNA will be extracted from maternal plasma and subjected to genome sequencing under the same conditions as the amniotic DNA. The generated data will be analysed using bioinformatic approaches integrating artificial intelligence methods to improve the distinction between fetal- and maternal-derived DNA fragments. Interpretation will be performed via the Gleaves-P interface. Non-invasive prenatal genome sequencing results are strictly for research purposes: they will not be communicated to couples and will not be used for clinical decision-making. Their objective is to evaluate the feasibility, accuracy, and performance of non-invasive genome sequencing.

研究の種類

観察的

入学 (推定)

100

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

  • 名前:Sarah BOUCHARD, Project Manager
  • 電話番号:+33 01 42 19 28 79
  • メール:sarah.bouchard@aphp.fr

研究連絡先のバックアップ

研究場所

    • Île-de-France Region
      • Paris、Île-de-France Region、フランス、75015
        • Hôpital Necker Enfants malades
        • コンタクト:
        • コンタクト:

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

サンプリング方法

非確率サンプル

調査対象母集団

Couples whose indication for prenatal exome sequencing has been reviewed and approved by a multidisciplinary prenatal diagnostic center.

説明

Inclusion Criteria:

  • Participating couple aged ≥ 18 years
  • Ongoing pregnancy for which the indication for exome sequencing has been discussed and validated by a Multidisciplinary Prenatal Diagnosis Center (CPDPN).

While a strict list of indications is not appropriate in the prenatal setting, examples include multiple anomalies not related to a malformation sequence, persistent increased nuchal translucency, hydrops fetalis (anasarca), cleft palate, multiple contractures/arthrogryposis, skeletal dysplasia, bowed femurs, or brain anomalies.

  • Sequencing performed on an amniotic fluid sample (chorionic villus sampling is excluded)
  • Attendance at a genetic counseling consultation
  • Written informed consent obtained for study participation

Exclusion Criteria:

  • Couple not covered by the social security system
  • Couple deprived of liberty or under legal guardianship or curatorship
  • Monoparental pregnancies

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

コホートと介入

グループ/コホート
介入・治療
Genome Sequencing (GS) + RNA-Sequencing (RNA-Seq)
Couples (pregnant woman and partner) with a fetal indication requiring prenatal exome sequencing, reviewed and approved by a multidisciplinary prenatal diagnosis center, will be included. Fetal samples (amniotic fluid, including cell cultures) and parental blood samples collected during routine care will be used for standard genetic analyses (chromosomal microarray and exome sequencing) and for research analyses, including genome sequencing (GS), RNA sequencing (RNA-Seq), and bioinformatic evaluations. Non-invasive genome sequencing performed from maternal blood samples (for couples included at Necker-Enfants Malades Hospital only) will be conducted for research purposes only; results will not be returned to couples and will not influence clinical management.

Couples whose indication for trio exome sequencing is approved by a multidisciplinary prenatal diagnostic center are enrolled during a pre-test genetic consultation, during which written informed consent is obtained from both partners.

As part of routine care, fetal samples are collected by amniocentesis and parental blood samples are obtained for chromosomal microarray analysis (CMA) and trio exome sequencing (ES), analyzed locally. Amniotic fluid cell cultures are systematically prepared and stored.

For research purposes, portions of fetal and parental samples are processed at Pitié-Salpêtrière and sequenced at SeqOIA for trio genome sequencing (GS). Bioinformatics analysis is performed using the MOABI platform, with interpretation via the Gleaves-P interface.

RNA sequencing (RNA-Seq) is performed on RNA extracted from amniotic fluid cell cultures at Necker Hospital.

For couples included at Necker only, maternal plasma DNA is also sequenced. Non-invasive GS results are for rese

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
Evaluation of the diagnostic contribution of RNA sequencing (RNA-Seq) performed in parallel with genome sequencing (GS) in prenatal diagnosis, compared with chromosomal microarray analysis (CMA) and exome sequencing (ES).
時間枠:18 months
Comparison of variants identified by each strategy and the number of additional diagnoses achieved using genome sequencing (GS) combined with RNA sequencing (RNA-Seq) compared with chromosomal microarray analysis (CMA) and exome sequencing (ES) in the prenatal setting. Diagnostic yield will be analyzed according to variant type (single-nucleotide and structural variants) and genomic location (coding vs non-coding regions).
18 months

二次結果の測定

結果測定
メジャーの説明
時間枠
Comparison of turnaround times for result delivery between genome sequencing combined with RNA sequencing (GS + RNA-Seq) and the current standard approach of chromosomal microarray analysis and exome sequencing (CMA + ES) in prenatal diagnosis.
時間枠:18 months
Time interval between test prescription and receipt of results for GS + RNA-Seq compared to CMA + ES in prenatal diagnosis
18 months
Comparison of the costs associated with GS + RNA-Seq versus CMA + ES in prenatal diagnostic settings.
時間枠:18 months
Evaluation and comparison of the costs associated with GS + RNA-Seq and CMA + ES approaches.
18 months
Evaluation of the feasibility and analytical performance of non-invasive prenatal genome sequencing performed on circulating cell-free fetal DNA extracted from maternal blood.
時間枠:18 months
Correlation of diagnostic results between invasive foetal genome sequencing performed on DNA extracted from amniotic fluid and non-invasive genome sequencing performed on circulating cell-free foetal DNA extracted from maternal plasma.
18 months
Identification and characterization of potential challenges and obstacles to the implementation of these diagnostic methods in clinical practice.
時間枠:18 months
List of difficulties encountered at each stage, from pre- to post-analytical.
18 months

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

捜査官

  • 主任研究者:Lucile BOUTAUD, Pharm.D PhD、Hôpital Necker Enfants Malades AP-HP

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年9月1日

一次修了 (推定)

2028年3月1日

研究の完了 (推定)

2028年3月1日

試験登録日

最初に提出

2026年8月6日

QC基準を満たした最初の提出物

2026年8月25日

最初の投稿 (実際)

2026年8月27日

学習記録の更新

投稿された最後の更新 (実際)

2026年8月27日

QC基準を満たした最後の更新が送信されました

2026年8月25日

最終確認日

2026年8月1日

詳しくは

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