NCT04554940进行中（未招募）2 期
A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
- 试验地点
- 3
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
研究概览
简要总结
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
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
- None
入排标准
- 年龄范围
- 0 Months 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure.
- •Have ACH, documented by genetic testing.
- •Are willing and able to perform all study procedures as physically possible.
- •Age 0 to ≤ 12 months, at study entry (Day 1). Given that any potential impact of vosoritide therapy on the foramen magnum is dependent on treating as early as possible and as long as possible while the synchondroses at the base of the skull are still open. For subjects > 6 months of age at enrollment, a discussion between the investigator and the Medical Monitor should occur with the goal of limiting the number of subjects in the range of > 6 months to ≤ 12 months of age.
- •Parent(s) or caregiver(s) are willing to administer daily injections to the subject and complete the required training.
- •Have evidence of CMC that "may" require surgical intervention
排除标准
- •Have hypochondroplasia or short-stature condition other than achondroplasia (eg, trisomy 21, pseudoachondroplasia, etc).
- •Have CMC that either does not require surgical intervention (for example foramen magnum narrowing with preservation of the cerebrospinal fluid space) or does require immediate surgical intervention .
- •Have any of the following: Untreated congenital hypothyroidism or maternal history of hyperthyroidism, Insulin-requiring neonatal diabetes mellitus, Autoimmune inflammatory disease, Inflammatory bowel disease, Autonomic neuropathy.
- •Have a history of any of the following:Renal insufficiency, Chronic anemia,Baseline systolic blood pressure below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low blood pressure generally accompanied by symptoms eg, pallor, cyanosis, irritability, poor feeding) and Cardiac or vascular disease.
- •Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F ≥ 450 msec on screening ECG.
- •Have been treated with growth hormone, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
- •Have ever had prior cervicomedullary decompression surgery.
- •Have had a fracture of the long bones or spine within 6 months prior to Screening.
研究组 & 干预措施
Vosoritide + Standard of Care
Experimental
Standard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of recommended dose of vosoritide based on weight-band dosing.
干预措施: vosoritide (Biological)
Standard of Care Alone
No Intervention
Institutional standard of care monitoring and treatment for cervicomedullary compression
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: Through Week 260
次要结局
- Evaluate the effect of Vosoritide on total foramen magnum volume (in cm3) by MRI volumetric measurement software(Through Week 260)
研究者
研究点 (3)
Loading locations...
相似试验
已完成
2 期
A Study to Investigate the Novel Agent BNT111 and Cemiplimab in Combination or as Single Agents in Patients With Advanced Melanoma That Has Not Responded to Other Forms of TreatmentMelanoma Stage IIIMelanoma Stage IVUnresectable MelanomaNCT04526899BioNTech SE184
已完成
2 期
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaAchondroplasiaNCT02055157BioMarin Pharmaceutical35
已完成
2 期
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaAchondroplasiaNCT03583697BioMarin Pharmaceutical75
已完成
2 期
Efficacy and Safety of BN101 in Subjects With Chronic Graft Versus Host Disease (cGVHD)GVHD, ChronicNCT04930562BioNova Pharmaceuticals (Shanghai) LTD.30
进行中（未招募）
2 期
A Clinical Trial Investigating the Safety, Tolerability, and Therapeutic Effects of BNT113 in Combination With Pembrolizumab Versus Pembrolizumab Alone for Patients With a Form of Head and Neck Cancer Positive for Human Papilloma Virus 16 and Expressing the Protein PD-L1Unresectable Head and Neck Squamous Cell CarcinomaMetastatic Head and Neck CancerRecurrent Head and Neck CancerNCT04534205BioNTech SE358