Ascendis Pharma、小児軟骨無形成症患者の自然歴研究を開始 / Ascendis Pharma started Observational Study of Children With Achondroplasia

Ascendis Pharmaは、小児の軟骨無形成症の治療薬TransConCNP を踏まえた自然歴研究をアメリカ、カナダ、イギリス、ドイツ、オーストラリアで開始します。対象は0-8歳までの小児軟骨無形成症患者、想定被験者数は200名、2024年3月完了とされます。具体的な施設は、2019年3月23日現在、上記の国においては10施設とされるものの施設名およびコンタクト先は未公表となっています。アメリカ以外の施設では被験者の募集は開始されていませんが、今後随時更新されるものです。

Ascendis Pharma starts their natural history study for TransCon CNP in US, Canada, UK, Germany and Australia. Their TransCon CNP is expected as a treatment for children with Achondroplasia. Ages eligible for study is up to 8 years and the number of estimated enrollment would be 200. The estimated primary completion date would be June 2024. As of 24th March, They are going to start 10 clinical centers among the countries but they have not disclosed their names and contacts. The States of the clinical sites except US are not recruiting yet but things are definitely updated at any time.





本研究は、軟骨無形成症(ACH)の小児を対象とした長期にわたる多施設共同縦断観察研究です。 研究目的は、ACHの小児の成長速度と合併症を研究することです。自然歴研究であり、治験薬は投与されません。

最重要測定項目: 1. 小児軟骨無形成症患者の身長の平均速度(cm/年)[期間:最長5年]


副次的評価項目:1. 小児軟骨無形成症患者の自然歴の収集[期間:最長5年間]



A Multi-center, Longitudinal, Observational Study of Children with Achondroplasia


Brief Summary:

This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and no study medication will be administered.


Outcome Measures

Primary Outcome Measures  : 1.Annualized height velocity (centimeters/year) in children with achondroplasia [ Time Frame: Up to 5 years ]

Subjects will undergo a series of height measurements (in centimeters) on Day 1 and then every 6 months over the study period with height velocity reported in cm/year

Secondary Outcome Measures  : 1.Collection of natural history of achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 5 years ]

To characterize achondroplasia symptoms in children with achondroplasia