The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.
Written, signed informed consent and/or assent of the participant, participant parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent.
Male or female, between 12 (inclusive) and 18 years of age at the time of randomization
Clinical diagnosis of ACH with documented genetic confirmation available. Documentation of historic test results are acceptable for proof of diagnosis.
Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and to follow the protocol.
Participation (signed informed consent) in any interventional clinical trial within 3 months prior to Screening unless no doses of IMP was given.
Decreased growth velocity (AGV less than 1.5 cm/year based on measurement over a period of at least 6 months) or radiological evidence of growth plate closure.
Known or suspected hypersensitivity to the IMP or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and mPEG).
Have a growth disorder or medical condition other than ACH that results in short stature, or abnormal growth such as SADDAN, hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudo-ACH, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, or diabetes mellitus.