A long-term study of RBM-007 in children with achondroplasia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- RIBOMIC Inc.
- 入组人数
- 12
- 主要终点
- height growth velocity
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomized Controlled Trial
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 5age old over 至 14age old under（—）
- 性别
- All
入选标准
- •(1) Has completed the early phase II study, has completed the safety and efficacy evaluation (height growth velocity, etc.) by Investigator, and has been judged to have no safety issues
- •(2) Able to obtain written informed consent from surrogate decision maker (parents, relatives or legal representative) before study entry
- •Able to obtain written informed assent from the patient after explaining the contents of this study in the assent document (if possible)
- •(3) Able to perform all the tests and administration of investigational product described in the protocol, judged by Investigator
排除标准
- •(1) Inadequate as a patient for this study based on examinations, observations, etc. at the end of the early phase II study
- •(2) Highly likely to be unable to complete this study due to poor compliance treated with RBM-007 or poor implementation of regular examinations and observation items, judged by the Investigator
- •(3) Diagnosed with a closed growth plate in the examination at the end of the early phase II study
- •(4) Has a positive pregnancy test at the end of the early phase II study (only female patients)
- •(5) Inadequate as a patient for this study for any other reason than above, considered by the Investigator
结局指标
主要结局
height growth velocity
Comparison of height growth velocity in the observational study and height growth velocity after the early phase II study entry
serum ALP and NT-proCNP
Change in serum ALP and NT-proCNP
body weight, head circumference and chest circumference
Change in body weight, head circumference and chest circumference
BMI
Change in BMI
body proportion ratios
Change in body proportion ratios (ratio of upper arm length to forearm length, ratio of thigh length to lower leg length, ratio of arm span to height)
elbow and hip joints
Condition of the elbow and hip joints based on clinical findings
bone age
Bone age
morphological shape of the femoral growth plate
Morphological shape of the femoral growth plate
Tanner Staging Classification
Tanner Staging Classification
adverse event
Adverse event
次要结局
未报告次要终点