A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
- Conditions
- Achondroplasia
- Registration Number
- JPRN-jRCT2061220040
- Lead Sponsor
- Kawai Norisuke
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 63
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.
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).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method