NCT06067425终止2 期
A Phase 2, Open-label, Multi-center, 2-stage Sequential Cohort, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Subcutaneous SAR442501 in Pediatric Participants With Achondroplasia
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Sanofi
- 入组人数
- 16
- 试验地点
- 17
- 主要终点
- Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent period
研究概览
简要总结
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
详细描述
Up to approximately 275 weeks: 3 weeks Screening + 52 weeks primary treatment period + up to approximately 216 weeks extended treatment period+ 4 weeks follow-up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Days 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must have ACH with a confirmed mutation in the FGFR3 gene
- •Participants and/or parent(s) or legal representative(s) must be willing and able to perform all the study procedures to the best of their physical ability.
- •Parent(s) or legal representative(s) capable of giving signed informed consent and participants capable of giving assent when applicable.
排除标准
- •Have hypochondroplasia (or the N540K mutation) or short stature condition other than ACH (eg, trisomy 21, pseudochondroplasia)
- •Participants have received any dose of medications or investigational product, including human growth hormone, IGF-1, intended to affect participants' stature or body proportions between the completion of OBS16647 and enrollment (Week 0/Day 1/Visit 2).
- •Have a history of growth plate closure.
- •Long bone fracture within 3 months of enrollment (Week 0/Day 1/Visit 2)
- •Current evidence of corneal or retinal disorder/keratopathy.
- •Participants have had a previous surgical intervention involving the foramen magnum (Stage 2 only).
- •Hyperphosphatemia.
- •The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
研究组 & 干预措施
Cohort 2
Experimental
干预措施: SAR442501 (Drug)
Cohort 1
Experimental
干预措施: SAR442501 (Drug)
Cohort 3
Experimental
干预措施: SAR442501 (Drug)
结局指标
主要结局
Number of participants with adverse events (AE), serious adverse events (SAE), and adverse events of special interest (AESI) during the treatment-emergent period
时间窗: Baseline to Week 52
次要结局
- Change in upper-to-lower body segment ratio(Baseline to Week 26 and Week 52)
- Change in sitting to standing height ratio (crown-to-rump length to total length for infants)(Baseline to Week 26 and Week 52)
- Change in arm span to height ratio(Baseline to Week 26 and Week 52)
- Change in upper arm to forearm length ratio(Baseline to Week 26 and Week 52)
- Change in annualized growth velocity (AGV) Zscore(Baseline to Week 26 and Week 52)
- Change in AGV (cm/year)(Baseline to Week 26 and Week 52)
- Change in height Z score(Baseline to Week 26 and Week 52)
- Change in head circumference to height ratio(Baseline to Week 26 and Week 52)
- Change in volumetric parameter(Baseline to Week 52)
- Change in developmental score in the Achondroplasia Developmental Recording Form(Baseline to Week 52)
- Assessment of PK parameter: maximum plasma concentration observed (Cmax)(Baseline to Week 26 and 52)
- Assessment of PD parameter: change in osteocalcin levels(Baseline to Week 26 and Week 52)
- Change in upper to lower extremity ratio(Baseline to Week 26 and Week 52)
- Change in brainstem parameter(Baseline to Week 52)
- Change in upper leg to lower leg ratio(Baseline to Week 26 and Week 52)
- Change in skull parameter(Baseline to Week 52)
- Change in present pain and worst pain rating (PPQ) score(Baseline to Week 26 and Week 52)
- Assessment of PK parameter: concentration observed before treatment administration during repeated dosing (Ctrough)(Baseline to Week 26 and 52)
- Assessment of pharmacodynamics (PD) parameter: change in collagen X biomarker (CXM) levels(Baseline to Week 26 and Week 52)
- Assessment of PD parameter: change in collagen-type 1 C-Telopeptide (CTX) levels(Baseline to Week 26 and Week 52)
- Change in mobility and symptom rating (STEMS) score(Baseline to Week 26 and Week 52)
- Assessment of PD parameter: change in procollagen type 1 N-terminal propeptide (P1NP) levels(Baseline to Week 26 and Week 52)
- Change in spine morphometric parameter(Baseline to Week 52)
- Change in fatigue score in the PedsQL Multidimensional Fatigue Scale(Baseline to Week 26 and Week 52)
- Assessment of PK parameter: Area under the plasma concentration versus time curve calculated using the trapezoidal method during a dose interval (AUC0-t)(Baseline to Week 26 and 52)
- Number of participants with treatment-emergent anti-drug antibodies (ADA)(Baseline to Week 26 and Week 52)
- Change in overall health-related quality of life score in the PedsQL Inventory Generic Core Scale(Baseline to Week 26 and Week 52)
- Assessment of pharmacokinetic (PK) parameter: plasma concentration of SAR442501(Baseline to Week 26 and 52)
- Assessment of PK parameter: time to reach Cmax (Tmax)(Baseline to Week 26 and 52)
- Assessment of PD parameter: change in bone-specific alkaline phosphatase(Baseline to Week 26 and Week 52)
- Changes in neurological examination(Baseline through Week 26 and Week 52)
研究者
研究点 (17)
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