NCT06079398 · Ascendis Pharma A/S
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
What this study is about
This trial is a Phase 2, conducted at multiple hospitals, where neither patients nor doctors know which treatment is given, randomly assigned (ratio 2:1 TransCon CNP vs.
View original scientific description
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.
Interventions
DRUG
Navepegritide
Once-weekly subcutaneous injection of 100 µg/kg Navepegritide
DRUG
Placebo for Navepegritide
Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide
Primary outcome measures
To evaluate the safety and tolerability of Navepegritide
Time frame: 52 weeks
Incidence of treatment emergent adverse events (TEAEs)
To evaluate the effect of Navepegritide on growth
Time frame: 52 weeks
Change from baseline to 52 weeks in length/height Z-score
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 b
Where
- Saint Paul, Minnesota
- Houston, Texas
- Madison, Wisconsin
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 8, 2026 · Source of record for eligibility and locations