Ascendis Pharma、TransCon CNPフェーズ2開始へ、スタディデザイン発表 / Ascendis Pharma announced the study design to start TransCon CNP Phase 2

Ascendis Pharma、TransCon CNPフェーズ2開始へ、スタディデザイン発表 / Ascendis Pharma announced the study design to start TransCon CNP Phase 2

Ascendis Pharma(本社:デンマーク)が、小児の軟骨無形成症患者へ週次CNP投与として開発進めるTransCon CNPのプロトコルがClinical Traials.govにて2019年9月11日付で公表されました。投与群は、TransCon CNP 6 mcg CNP/kg・20 mcg CNP/kg・50 mcg CNP/kg・100 mcg CNP/kg、100 mcg超CNP/kgの5つで、いずれの群もプラセボランダマイズ(TransCon CNP:Placebo=3:1)とされます。2019年9月12日現在、米国内の5つの施設が想定されるもののリクルーティングステータスは(Not yet recruiting)です。


12月9日時点でのClinical Traials.govの仮訳および原文の抜粋を掲載します。

The protocol of TransCon CNP, which Ascendis Pharma (Headquarters: Denmark) is developing as a weekly CNP administration for children with achondroplasia, was disclosed on September 11, 2019 at Clinical There are five arms, TransCon CNP 6 mcg CNP / kg, 20 mcg CNP / kg, 50 mcg CNP / kg, 100 mcg CNP / kg, and over 100 mcg CNP / kg, Placebo randomized (Placebo = 3: 1). As of September 12, five centers in the US are expected, but the recruiting status is still “Not yet recruiting”.

In a growth study of young healthy monkeys in basic research, the company demonstrated that the effect of TransConCNP is higher than that of CNP analogs on daily dose, and confirmed that it was well tolerated in phase I clinical trials for adults male.

Here is a Japanese translation into Japanese and an excerption in English.


思春期前の小児軟骨形成無形成症患者への週次投与薬TransCon CNPの安全性、有効性、および薬物動態を評価する用量拡大試験




この試験は、多施設共同無作為化二重盲検プラセボ対照試験であり、2〜10歳の思春期前の小児軟骨無形成症患者に、週1回のTransCon CNPを皮下投与する。

状態/疾患 介入/治療Intervention/treatment 試験フェーズ
軟骨無形成症 Drug: TransCon CNP
Drug: Placebo for TransCon CNP
Phase 2


試験タイプ : 介入
想定参加者 : 60名
割当: 無作為
介入モデル: 並行群間比較試験
介入モデルの詳細: 5つ被験者群について、TransCon CNPまたはプラセボのいずれかを3:1の比率で投与するよう無作為化された約60人の被験者で構成
マスキング: 4重盲目 (参加者、保護者、調査者、結果評価者)
主な目的: 治療
公式タイトル: フェーズ2、多施設、二重盲検、無作為化、プラセボ対照、用量漸増試験、軟骨無形成症の思春期前小児に12か月間、週1回投与されるTransCon CNPの皮下投与の安全性、有効性、および薬物動態の評価
調査開始予定: 2019年11月
初期研究終了予定 : 2021年6月
研究終了予定 : 2021年6月



主な効果の測定 :


毎週1回のTransCon CNP治療またはプラセボの安全性と忍容性


TransCon CNPまたはプラセボの52週間にわたってセンチメートルで測定された年単位の高さ速度











治験への参加中に外科的介入を必要とするか、または Salter-Harris骨折や重度の股関節痛などの筋骨格系の性質あるいは治験責任医師/メディカルモニター/医療専門家が、参加者が治験薬を服用したり、治験関連の手続きを受けるのに適していないと認めた場合






A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

Study Description

Brief Summary:

The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.

Condition or disease Intervention/treatment Phase
Achondroplasia Drug: TransCon CNP
Drug: Placebo for TransCon CNP
Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: There are 5 cohorts enrolling approximately 60 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratio
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Accomplish: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 12 Months in Prepubertal Children With Achondroplasia
Estimated Study Start Date : November 2019
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Outcome Measures

Primary Outcome Measures :

  1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: 52 weeks ]

Safety and tolerability of once weekly TransCon CNP treatment or placebo

  1. Annualized height velocity (centimeters/year) at 52 weeks [ Time Frame: 52 weeks ]

Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo

Eligibility Criteria


Inclusion Criteria:

  1. Clinical diagnosis of ACH with genetic confirmation
  2. Age between 2 to 10 years old (inclusive) at Screening Visit
  3. Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit
  4. Able to stand without assistance
  5. Caregiver willing and able to administer subcutaneous injections of study drug

Exclusion Criteria:

  1. Clinically significant findings at Screening that:
    • are expected to require surgical intervention during participation in the trial or
    • are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or
    • otherwise are considered by investigator or Medical Monitor/Medical Expert to make a participant unfit to receive study drug or undergo trial related procedures
  1. Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
  2. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
  3. Have received any study drug or device intended to affect stature or body proportionality at any time
  4. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

Source: Clinical



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