ABOUT THIS STUDY
- 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.
- 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
- 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.
- Severe renal impairment (eGFR <60 mL/min/1.73m2) or hepatic impairment (AST/ALT >1.5
- 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
- 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).
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