Actively Recruiting
A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-Controlled Trial Evaluating Weekly Subcutaneous TransCon CNP (Navepegritide) for 52 Weeks in Infants Under 2 Years with Achondroplasia with an Open Label Extension
Led by Ascendis Pharma A/S · Updated on 2026-04-08
72
Participants Needed
18
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and effectiveness of Navepegritide (TransCon CNP) in infants with genetically confirmed achondroplasia (ACH) who are younger than 2 years old. This Phase 2, multicenter, double-blind, randomized, placebo-controlled trial aims to compare weekly doses of Navepegritide with a placebo over a 52-week period to understand its impact on growth and safety in this population. Participants receive once-weekly subcutaneous injections of either 100 bcg/kg Navepegritide or a placebo for 52 weeks. The study is randomized in a 2:1 ratio, with some infants receiving the active drug and others receiving placebo injections. After the 52-week treatment period, there is an open-label extension phase allowing continued evaluation. During the study, infants will be monitored through medical history reviews, physical exams, vital signs, ECGs, imaging, and lab tests to evaluate safety and growth changes. The main outcomes measured are the safety and tolerability of Navepegritide and its effect on growth over 52 weeks. Parents or caregivers will administer weekly injections and follow study instructions, including vitamin D supplementation where applicable. The total participation time includes the initial 52 weeks of treatment followed by further observation during the extension period.
CONDITIONS
Brief Title
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent from parent(s) or caregiver(s)
- Male or female younger than 2 years at randomization
- Clinical and genetic confirmation of heterozygous achondroplasia
- Willingness to follow protocol and administer weekly subcutaneous injections
- Compliance with daily Vitamin D supplementation for infants aged 14 days to 1 year, or supplementation if Vitamin D levels are low in older infants
- Medically eligible based on history, physical exam, vital signs, ECG, imaging, and lab tests during screening
You will not qualify if you...
- Known or suspected allergy to the investigational product or related substances
- Genetic confirmation of homozygous achondroplasia
- Premature birth before 32 weeks gestation
- Premature birth between 32 and 37 weeks unless over 6 months old and in good nutritional status
- Expected surgical interventions during the trial including cervicomedullary decompression
- Growth disorders or conditions other than achondroplasia affecting stature
- Prior use of medications or investigational products affecting growth or body proportion
- Need for chronic or frequent corticosteroid use
- Growth plate injuries or recent bone surgeries
- Significant abnormal cardiac conditions or ECG abnormalities
- Conditions affecting blood flow stability
- Chronic anemia, kidney insufficiency, or illnesses affecting hydration
- History or presence of cancer
- Any condition likely to interfere with trial completion, protocol adherence, or safety as judged by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks
Participants receive a once-weekly subcutaneous injection of either Navepegritide or placebo for 52 weeks.
Weekly visits for 52 weeks
Trial Site Locations
Total: 18 locations
1
Ascendis Investigational Site
Saint Paul, Minnesota, United States, 55102
Actively Recruiting
2
Ascendis Investigational Site
Houston, Texas, United States, 77030
Actively Recruiting
3
Ascendis Investigational Site
Madison, Wisconsin, United States, 53705
Actively Recruiting
4
Ascendis Investigational Site
Parkville, Australia, 3052
Actively Recruiting
5
Ascendis Investigational Site
Linz, Austria, 4020
Actively Recruiting
6
Ascendis Investigational Site
Montreal, Canada, H3T 1C5
Actively Recruiting
7
Ascendis Investigational Site
Copenhagen, Denmark, 2100
Actively Recruiting
8
Ascendis Investigational Site
Helsinki, Finland, 00029
Actively Recruiting
9
Ascendis Investigational Site
Paris, France, 75743
Actively Recruiting
10
Ascendis Investigational Site
Berlin, Germany, 13353
Actively Recruiting
11
Ascendis Investigational Site
Cologne, Germany, 50931
Actively Recruiting
12
Ascendis Investigational Site
Dublin, Ireland, D01 YC76
Actively Recruiting
13
Ascendis Investigational Site
Milan, Italy, 20132
Actively Recruiting
14
Ascendis Investigational Site
Auckland, New Zealand, 1023
Actively Recruiting
15
Ascendis Investigational Site
Oslo, Norway, 0372
Actively Recruiting
16
Ascendis Investigational Site
Coimbra, Portugal, 3000-602
Actively Recruiting
17
Ascendis Investigational Site
Stockholm, Sweden, 17176
Actively Recruiting
18
Ascendis Investigational Site
London, United Kingdom, SE1 7EH
Actively Recruiting
Research Team
A
Ascendis Registry Inquiries
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here