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A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Led by Ascendis Pharma A/S · Updated on 2026-04-21
24
Participants Needed
5
Research Sites
142 weeks
Total Duration
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AI-Summary
What this Trial Is About
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.
CONDITIONS
Official Title
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written, signed informed consent and/or assent as required by ethical committees
- Male or female aged 12 (inclusive) to 18 years at randomization
- Clinical diagnosis of Achondroplasia with documented genetic confirmation
- Parent(s)/legal guardian(s) able to administer weekly subcutaneous injections and follow protocol
- At least one standing height measurement from medical records taken 6 to 15 months before screening
You will not qualify if you...
- Participation in any interventional clinical trial with investigational medicinal product (IMP) within 3 months prior to screening
- Decreased growth velocity (less than 1.5 cm/year over at least 6 months) or evidence of growth plate closure
- Known or suspected hypersensitivity to the investigational medicinal product or related substances
- Presence of other growth disorders or medical conditions causing short stature such as SADDAN, hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudo-Achondroplasia, inflammatory bowel disease, celiac disease, thyroid disorders, or diabetes mellitus
- Severe FGFR3 gene mutations or severe Achondroplasia with developmental delay and acanthosis nigricans
- Prior treatment with medications or surgeries affecting growth or body proportions
- Requirement or expected need for chronic or repeated systemic corticosteroid treatment during the trial
- History of injury or disease affecting growth plates other than Achondroplasia
- Bone-related surgeries affecting growth potential, except certain reconstructive surgeries with full recovery
- Clinically significant conditions at screening that may affect trial participation or safety including severe untreated sleep apnea, certain fractures, or other investigator-assessed fitness issues
- Abnormal cardiac function or conduction disorders including repaired or unrepaired coarctation and complex congenital heart diseases
- QTcF interval equal to or greater than 450 msec at screening
- Conditions impacting hemodynamic stability such as autonomic dysfunction or orthostatic intolerance
- Chronic anemia (except resolved iron deficiency anemia), chronic renal insufficiency, or chronic/recurrent illnesses affecting hydration or nutrition
- History or presence of malignant disease
- Serum vitamin D levels below 30 nmol/L at screening (unless supplemented according to local standards)
- Any condition or situation that may prevent trial completion, confound results, or increase risk
- Sexually active participants and partners not using highly effective contraception during and 90 days after the trial
- Female participants who are pregnant, lactating, or breastfeeding
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 5 locations
1
Ascendis Investigational Site
Montreal, Canada, H3T 1C5
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2
Ascendis Investigational Site
Copenhagen, Denmark, 2100
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3
Ascendis Investigational Site
Paris, France, 75743
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4
Ascendis Investigational Site
Dublin, Ireland, D01 YC76
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5
Ascendis Investigational Site
Norwich, United Kingdom, NR4 7UY
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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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