A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants… (NCT06079398) | Clinical Trial Compass
RecruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
United States, Australia, Austria72 participantsStarted 2024-01-23
Plain-language summary
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 ÎĽg 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.
Who can participate
Age range0 Years – 2 Years
SexALL
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Inclusion Criteria:
* 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.
* Compliance to daily Vitamin D supplementation for infants aged 14 days to 1 year. All participants older than 1 year of age with serum 25-hydroxyvitamin D (25OHD) measured below lower limit of reference range at screening should start daily Vitamin D supplementation prior to randomization.
* Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG, imaging, and clinical laboratory tests performed during the screening period.
Exclusion Criteria:
* 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 gesta…
What they're measuring
1
To evaluate the safety and tolerability of Navepegritide