This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.
Written, signed informed consent by the parent(s)/caregiver(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
Male or female younger than 2 years of age at the time of randomization; or for open label sentinel participants, at the time of first administration of IMP.
Clinical diagnosis of achondroplasia (ACH) with genetic confirmation of heterozygous genotype present during screening.
Parent(s)/caregiver(s) willing to follow the protocol and instructions provided, including being able to administer weekly subcutaneous injections of trial treatment.
Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris[hydroxymethyl]aminomethane, succinate, and polyethylene glycol [PEG]).
Genetic confirmation of ACH homozygous genotype.
Premature birth with gestational age < 32 weeks.
Premature birth with gestational age 32 to 37 weeks, unless time from birth is > 6 months at the time of screening and the child is in good nutritional status, defined as gain in body weight expected for age and diagnosis of ACH, as determined by the Investigator and confirmed with the Medical Monitor.
Parallel
Navepegritide
DrugOnce-weekly subcutaneous injection of 100 µg/kg Navepegritide
Placebo for Navepegritide
DrugOnce-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide
To evaluate the safety and tolerability of Navepegritide
Incidence of treatment emergent adverse events (TEAEs)
To evaluate the effect of Navepegritide on growth
Change from baseline to 52 weeks in length/height Z-score
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