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A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia | Clinical Trials | Clareo Health

RECRUITINGPHASE2

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period

NCT06079398•Ascendis Pharma A/S

View on ClinicalTrials.gov

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 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.

Eligibility

Age

0 - 2 years

Sex

ALL

Healthy Volunteers

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 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.
•Anticipated, as assessed by Investigator and confirmed with Medical Monitor, to undergo surgical intervention during trial participation, including cervicomedullary decompression. Evaluation of immediate risk of requiring cervicomedullary decompression surgery will rely on the following assessments:
•* Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
•* Evidence of uncontrolled sleep apnea as confirmed by local standard of care assessment (e.g. polysomnography or simple sleep test) performed within 6 months prior to screening.
•Common surgeries, such as insertion of grommets, adenoidectomy, tonsillectomy, or myringotomy tube placement are permitted during trial participation.
•Have a growth disorder or medical condition, other than ACH, resulting in short stature or abnormal growth as determined by the Investigator and confirmed with the Medical Monitor.
•Have received any dose of prescription medications and/or investigational medicinal product or device intended to affect stature, growth, or body proportionality (including human growth hormone or vosoritide) at any time.
+13 more criteria

Locations (17)

Ascendis Investigational Site

Saint Paul, Minnesota, United States

Ascendis Investigational Site

Houston, Texas, United States

Ascendis Investigational Site

Madison, Wisconsin, United States

Ascendis Investigational Site

Parkville, Australia

Ascendis Investigational Site

Linz, Austria

Ascendis Investigational Site

Copenhagen, Denmark

Ascendis Investigational Site

Helsinki, Finland

Ascendis Investigational Site

Paris, France

Ascendis Investigational Site

Berlin, Germany

Ascendis Investigational Site

Cologne, Germany

Key Dates

Start Date

January 23, 2024

Primary Completion Date

December 1, 2027

Completion Date

December 1, 2028

Last Updated

February 20, 2026

Enrollment

ESTIMATED participants

Conditions

Achondroplasia

Interventions

Navepegritide

DRUG

Placebo for Navepegritide

DRUG

Contacts

Ascendis Registry Inquiries

CONTACT

+45 61242484 asnd_registryinquiries@ascendispharma.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 20, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Inclusion Criteria

Exclusion Criteria

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

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Radiological Analysis on Patients With Achondroplasia Disorder