An early phase II 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) 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 trial in the assent document (if possible)
- •(2) Has achondroplasia, diagnosed by genetic diagnosis
- •(3) Aged 5 to 14 years, inclusive, at study entry
- •(4) Able to lead a normal life without assistance
- •(5) Able to perform all the tests and administration of investigational product described in the protocol, judged by Investigator
- •(6) Participates in the observational study and has a height measurement record at the trial site at least 26 weeks (6 months) before study entry
排除标准
- •(1) Has short stature condition other than achondroplasia
- •(2) Has congenital diseases other than achondroplasia
- •(3) Has history of treatment with growth hormone or VOXZOGO within 38 weeks before study entry
- •(4) Has history of limb-lengthening surgery
- •(5) With a closed growth plate
- •(6) Has any of the following disorders that require surgical treatment on MRI or CT scans, or has clinically significant neurological symptoms or obstructive sleep apnea syndrome caused by these disorders: foramen magnum stenosis, spinal stenosis, hydrocephalus, spinal cord and cauda equina compression, etc (However, patients H146who have undergone surgical treatment for these disorders at least 52 weeks (12 months) before study entry and have no clinical problems can be enrolled)
- •(7) Has a history of allergies against the following products containing RBM-007 components (e.g., nucleic acid, polyethylene glycol, mannitol): prescribed medicines, vaccines, over-the-counter medicines, cosmetics, foods, etc
- •(8) Has history of cardiac or vascular disease
- •(9) Shows clinically significant abnormalities in the clinical laboratory test result, which is related to hepatic function or renal function before study entry
- •(10) Had a fracture of the long bones or spine within 52 weeks (12 months) before study entry (except for fracture of digits or toes)
- •(11) Has history of hip surgery, severe hip dysplasia or serious hip injury
- •(12) Highly likely to be unable to comply with RBM-007 treatment or to complete the trial, judged by the Investigator
- •(13) Used any other IP or investigational medical device within 52 weeks (12 months) before study entry
- •(14) Inadequate as a patient for this trial for any other reason than above, considered by the Investigator
结局指标
主要结局
Height growth velocity
时间窗: at least 26 weeks [6 months] each
Comparison of height growth velocity before and after administration
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)
Condition of elbow and hip joints
Condition of 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
Blood concentration profile of RBM-007 and pharmacokinetic parameters
Blood concentration profile of RBM-007 and pharmacokinetic parameters
次要结局
未报告次要终点