JPRN-jRCT2061220040已完成2 期
A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
Kawai Norisuke0 个研究点目标入组 63 人开始时间: 2022年7月7日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 63
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 2age old 至 < 11age old（—）
- 性别
- All
入选标准
- •Inclusion Criteria:
- •Main cohort: Aged >=2 years to <11 years (up to the day before 11th birthday inclusive) at time of enrollment; or exploratory cohort: aged >=3 months to <2 years (up to the day before 2nd birthday inclusive) at time of enrollment
- •Documented, confirmed genetic diagnosis of achondroplasia from historical medical records prior to entry into this trial (test must have been performed at a laboratory fully accredited for genetic testing under local regulations).
- •Completed the C4181001 natural history study with at least 2 valid height/length measurements (at least 3 months apart) prior to enrollment in this study. One of these measurement timepoints must be within the 3 months prior to enrollment in C4181005.
- •Tanner stage 1 based on investigator assessment during physical examination (must include assessment of breast development for females, testicular stage for males).
- •Able to stand independently for height measurements (if >=2 years of age at enrollment).
- •If aged <2 years at enrollment, has a documented historical MRI brain/cervical spine performed in the previous 12 months.
排除标准
- •Exclusion Criteria:
- •*Presence of co-morbid conditions or circumstances that, in the opinion of the investigator, would affect interpretation of growth data or ability to complete the trial procedures.
- •*Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
- •*Presence of severe obesity (BMI >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].14
- •*Known closure of long bone growth plates (cessation of height growth).
- •*Body weight <7 kg or >30 kg.
- •*Moderate or Severe renal impairment CrCL GFR <60 mL/min/1.73m2 (Calculated GFR based on updated bedside Schwartz formula for pediatric patients (CrCL (mL/min/1.73 m2) = 0.413 x Height (cms)/ Serum cr (mg/dL) or hepatic impairment (AST/ALT >1.5 ULN).
- •*History of hypersensitivity to study intervention or any excipients.
- •*History of any prior treatment with human growth hormone or related products (including insulin-like growth factor 1 [IGF-1]).
- •*History of receipt of any treatment that are known to potentially affect growth (including oral steroids >5 days in the last 6 months, high dose inhaled corticosteroids (>800 mcg/day beclametasone equivalent) and medication for attention deficit hyperactivity disorder).
- •*History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
- •*Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
- •*Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
- •*Presence of any internal guided growth plates/devices.
- •*History of removal of internal guided growth plates/devices within less than 6 months.
- •*History of receipt of any investigational product for achondroplasia or that may affect growth/interpretation of growth parameters.
- •*History of receipt of an investigational product (not for achondroplasia/growth affecting) within the last 30 days or 5 half-lives (whichever is longer).
研究者
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