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Looking for participantsPhase2

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

Sponsor: Ascendis Pharma A/S

NCT ID: NCT06079398

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Navepegritide (drug), Placebo for Navepegritide (drug)
How long the study runs
Study runs about 59 months (dates as stated)
About the drug or intervention
Navepegritide — drug: Once-weekly subcutaneous injection of 100 µg/kg Navepegritide · Placebo for Navepegritide — drug: Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide
Patient visit burden
Not specified by the sponsor

In plain English

This trial is testing a medicine called TransCon CNP against a dummy medicine (placebo) in babies and children under 2 years old who have achondroplasia, a genetic condition that affects bone growth. The trial is looking at how well the medicine works and how safe it is. It is run by Ascendis Pharma A/S.

Who can take part

  • Parents or caregivers must give written, signed permission for their child to take part
  • Boys or girls younger than 2 years old when they join the trial (or, for a small first group on open treatment, when they first get the medicine)
  • A clinical diagnosis of achondroplasia confirmed by a genetic test during screening
  • Parents or caregivers must be willing to follow the trial instructions, including giving a weekly injection under the skin at home
  • Babies aged 14 days to 1 year must take daily vitamin D; children over 1 year with low vitamin D levels on screening must start daily vitamin D before joining
  • The child must be judged fit to take part based on their medical history, physical checks, vital signs, heart tracing (electrocardiogram), scans, and blood and urine tests during screening

Who may not be able to

  • Known or suspected allergy to the trial medicine or its ingredients (trehalose, tris[hydroxymethyl]aminomethane, succinate, and polyethylene glycol)
  • Achondroplasia where the child has two copies of the faulty gene (homozygous)
  • Born early (premature) before 32 weeks of pregnancy
  • Born between 32 and 37 weeks of pregnancy, unless more than 6 months old at screening and growing well in weight, as confirmed by the trial doctor and medical monitor
  • Likely to need surgery during the trial, especially surgery to relieve pressure where the skull meets the spine (cervicomedullary decompression), based on examination, sleep apnoea tests, or an MRI scan; common operations such as grommets, removing tonsils or adenoids, or ear tubes are allowed
  • Another growth condition or medical problem, apart from achondroplasia, that causes short height or abnormal growth
  • Ever having taken prescribed medicines or trial products meant to affect height, growth, or body proportions, including growth hormone or vosoritide
  • Needing or likely to need corticosteroid tablets for more than 4 weeks, more than twice a year, or high doses of inhaled corticosteroids during the trial
  • Past or current injury or disease of the growth plates (other than achondroplasia), such as certain fractures (Salter-Harris) or recent bone surgery
  • Serious heart problems, including certain congenital heart conditions, repaired or unrepaired narrowing of the main body artery (coarctation), or a particular heart tracing result (QTcF of 450 milliseconds or more)
  • Conditions affecting blood pressure or circulation stability, such as autonomic dysfunction or orthostatic intolerance
  • Long-term anaemia, long-term kidney problems, or long-term or repeated illness affecting hydration or fluid levels
  • Past or current cancer
  • Any other condition or family situation that, in the trial doctor's view, could stop the child finishing the trial, affect the results, or put the child at extra risk

What taking part involves

  • • The trial medicine TransCon CNP or a dummy medicine (placebo), given by the parent or caregiver as a weekly injection under the skin
  • • Daily vitamin D for babies aged 14 days to 1 year, and for older children with low vitamin D levels
  • • Screening checks including medical history, physical examination, vital signs, heart tracing (electrocardiogram), imaging, and laboratory tests

Time commitment: Weekly injections under the skin given at home by the parent or caregiver, daily vitamin D, and screening tests including an ECG, imaging, and blood tests; total trial length and number of clinic visits: Not stated — ask the trial team.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
0 Years to 2 Years
Who
All
Number of participants
72
Started
2024-01-23
Last checked
2026-04

Plain English Summary

What is this study?

  • • Testing a new treatment for achondroplasia
  • • Phase2 - 72 participants
  • • This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs

Who can take part?

  • • Ages 0 Years to 2 Years
  • • Diagnosed with achondroplasia

Where?

  • • London - Ascendis Investigational Site

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Achondroplasia

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 0 Years - 2 Years
  • Who can join: All genders

What the study is looking for

  • ✓Male or female younger than 2 years of age at the time of randomization; or for open label sentinel participants, at...
  • ✓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...
  • ✓Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG,...

Who cannot take part

  • ✗Known or suspected hypersensitivity to the the the study treatment or related products (trehalose,...
  • ✗Genetic confirmation of ACH homozygous genotype.
  • ✗Premature birth with gestational age \< 32 weeks.
  • ✗Physical examination (e.g., neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
  • ✗Evidence of uncontrolled sleep apnea as confirmed by local the usual treatment assessment (e.g. polysomnography or...
See the full criteria
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. * MRI performed at screening indicating presence of severe cervicomedullary compression (CMC) or spinal cord damage. Presence of abnormal MRI T2 signal intensity at and immediately above and below the cervicomedullary junction should be considered high risk for requiring surgery and the participant is not eligible for trial participation. 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. * Requires or anticipated to require chronic (\> 4 weeks) or repeated treatment (more than twice/year) with oral corticosteroids, or high-dose inhaled corticosteroids during trial participation. * History or presence of injury or disease of the growth plate(s), other than ACH, affecting growth potential of long bones, including Salter-Harris fracture and recent bone-related surgery, as determined by Investigator and confirmed with the Medical Monitor. * Have a clinically significant finding indicating abnormal cardiac function, including but not limited to: * Repaired or unrepaired coarctation. * Moderate or greater complexity congenital heart disease including tetralogy of Fallot, atrioventricular septal defects, truncus arteriosus, total anomalous pulmonary venous return, double outlet right ventricle, or single ventricle heart disease. * QTcF ≥ 450 msec on screening 12-lead ECG. * History or presence of a condition impacting hemodynamic stability (such as autonomic dysfunction and orthostatic intolerance). * History or presence of the following: * Chronic anemia. * Chronic renal insufficiency. * Chronic or recurrent illness that can affect hydration or volume status, including conditions associated with decreased nutritional intake or increased volume loss. * History or presence of malignant disease. * Any disease or condition that, in the opinion of the Investigator, may make the participant unlikely to fully complete the trial, not adhering to trial procedures, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, comorbid conditions, or medications that might impact safety or be considered confounding.

Where Is This Study? (1 UK site)

Ascendis Investigational Site

London SE1 7EH, United Kingdom

Recruiting

How to Get in Touch

Ascendis Registry Inquiries

Sponsor contact

CONTACT

+45 61242484 asnd_registryinquiries@ascendispharma.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-04