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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
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
Where Is This Study? (1 UK site)
Ascendis Investigational Site
London SE1 7EH, United Kingdom
How to Get in Touch
Ascendis Registry Inquiries
Sponsor contactCONTACT
