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 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 57
- 试验地点
- 16
- 主要终点
- Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of ACH with genetic confirmation
- •Age between 2 to 10 years old (inclusive) at Screening Visit
- •Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit
- •Able to stand without assistance
- •Caregiver willing and able to administer subcutaneous injections of study drug
排除标准
- •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
- •Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
- •Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
- •Have received any study drug or device intended to affect stature or body proportionality at any time
- •History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones
研究组 & 干预措施
Placebo
Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection.
干预措施: Placebo for TransCon CNP (Drug)
TransCon CNP 100 mcg
TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection.
干预措施: TransCon CNP (Drug)
TransCon CNP 20 mcg
TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection.
干预措施: TransCon CNP (Drug)
TransCon CNP 50 mcg
TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection.
干预措施: TransCon CNP (Drug)
TransCon CNP 6 mcg
TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection.
干预措施: TransCon CNP (Drug)
Open-Label Extension Period: TransCon CNP
Participants who completed the 52-week blinded treatment period continued into the 104-week open-label extension period and received treatment with TransCon CNP (navepegritide) doses escalated up to a maximum of 100 mcg/kg delivered once weekly by subcutaneous injection.
干预措施: TransCon CNP (Drug)
结局指标
主要结局
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment
时间窗: 52 weeks
The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.
次要结局
未报告次要终点