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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
Led by Ascendis Pharma A/S · Updated on 2026-04-08
72
Participants Needed
18
Research Sites
253 weeks
Total Duration
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AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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...
- Parent or caregiver has provided written informed consent
- Male or female younger than 2 years old at randomization or first study drug administration
- Clinical diagnosis of achondroplasia with genetic confirmation of heterozygous genotype
- Parent or caregiver willing to follow study protocol and administer weekly injections
- Compliance with daily Vitamin D supplementation for infants aged 14 days to 1 year; older infants with low vitamin D must start supplementation before randomization
- Eligible based on medical history, physical exam, vital signs, ECG, imaging, and lab tests during screening
You will not qualify if you...
- Known or suspected allergy to the study drug or related substances
- Genetic confirmation of homozygous achondroplasia
- Premature birth before 32 weeks gestation
- Premature birth between 32 and 37 weeks gestation unless over 6 months old and well-nourished
- Expected to require surgery during the study, including cervicomedullary decompression
- Presence of severe cervicomedullary compression or spinal cord damage on MRI
- Growth disorders other than achondroplasia
- Prior use of medications affecting growth or body proportions
- Need for chronic or frequent corticosteroid treatment during the study
- History of growth plate injury or recent bone surgery
- Significant heart abnormalities or congenital heart disease
- Conditions affecting blood flow stability
- Chronic anemia or kidney problems
- Chronic or recurrent illnesses affecting hydration or nutrition
- History of cancer
- Any condition likely to prevent study completion or affect safety or results as judged by the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 18 locations
1
Ascendis Investigational Site
Saint Paul, Minnesota, United States, 55102
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2
Ascendis Investigational Site
Houston, Texas, United States, 77030
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3
Ascendis Investigational Site
Madison, Wisconsin, United States, 53705
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4
Ascendis Investigational Site
Parkville, Australia, 3052
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5
Ascendis Investigational Site
Linz, Austria, 4020
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6
Ascendis Investigational Site
Montreal, Canada, H3T 1C5
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7
Ascendis Investigational Site
Copenhagen, Denmark, 2100
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8
Ascendis Investigational Site
Helsinki, Finland, 00029
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9
Ascendis Investigational Site
Paris, France, 75743
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10
Ascendis Investigational Site
Berlin, Germany, 13353
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11
Ascendis Investigational Site
Cologne, Germany, 50931
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12
Ascendis Investigational Site
Dublin, Ireland, D01 YC76
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13
Ascendis Investigational Site
Milan, Italy, 20132
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14
Ascendis Investigational Site
Auckland, New Zealand, 1023
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15
Ascendis Investigational Site
Oslo, Norway, 0372
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16
Ascendis Investigational Site
Coimbra, Portugal, 3000-602
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17
Ascendis Investigational Site
Stockholm, Sweden, 17176
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18
Ascendis Investigational Site
London, United Kingdom, SE1 7EH
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Research Team
A
Ascendis Registry Inquiries
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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