Actively Recruiting
A Phase 2b Trial Evaluating Weekly Subcutaneous Navepegritide for 52 Weeks in Adolescents (12-18 Years) With Achondroplasia
Led by Ascendis Pharma A/S · Updated on 2026-04-21
24
Participants Needed
5
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effectiveness and safety of once-weekly subcutaneous doses of navepegritide at 100 micrograms per kilogram compared to a placebo in adolescents aged 12 to 18 years with Achondroplasia. The main focus is to measure the annualized growth velocity after a 52-week treatment period. Participants will receive either navepegritide or a placebo through weekly injections under the skin for one year. The study is randomized, double-blinded, and placebo-controlled, ensuring that neither the participants nor the researchers know who receives the active medication or placebo during the 52 weeks. During the trial, participants will be monitored regularly to assess growth and other health parameters. Researchers will measure growth rates and height z-scores over the course of the year. Safety and any side effects will be closely observed throughout the study. The trial is sponsored by Ascendis Pharma A/S and aims to provide detailed information about the effects of navepegritide in this adolescent population.
CONDITIONS
Brief 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 informed consent or assent from participant and parent/legal guardian as required
- Male or female aged between 12 and 18 years at randomization
- Clinical diagnosis of Achondroplasia with genetic confirmation
- Parent or guardian willing and able to give 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 within 3 months before screening if receiving investigational treatment
- Decreased growth velocity below 1.5 cm/year or evidence of growth plate closure
- Known or suspected allergy to investigational product or related substances
- Presence of other growth disorders or medical conditions causing short stature besides Achondroplasia
- Severe FGFR3 gene mutation or severe Achondroplasia with developmental delay
- Previous treatment or surgery affecting growth or body proportions
- Need for chronic or repeated systemic corticosteroids during trial
- History of bone injuries or surgeries affecting growth potential within specified timeframes
- Significant medical conditions or surgeries that may affect trial participation or safety
- Cardiac abnormalities or arrhythmias affecting heart function
- QTcF interval equal or above 450 msec at screening
- Conditions impacting blood flow stability
- Chronic anemia not adequately treated
- Chronic renal insufficiency with eGFR below 60 mL/min/1.73 m2 for over 3 months
- Chronic or recurrent illness affecting hydration or nutrition
- History of malignant disease
- Low vitamin D levels below 30 nmol/L at screening without supplementation
- Any condition that may prevent trial completion or confound results
- Sexually active participants of childbearing potential not using effective contraception
- Pregnant, lactating, or breastfeeding females
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 weekly subcutaneous injections of navepegritide or placebo for 52 weeks.
Weekly visits for 52 weeks
Trial Site Locations
Total: 5 locations
1
Ascendis Investigational Site
Montreal, Canada, H3T 1C5
Actively Recruiting
2
Ascendis Investigational Site
Copenhagen, Denmark, 2100
Actively Recruiting
3
Ascendis Investigational Site
Paris, France, 75743
Actively Recruiting
4
Ascendis Investigational Site
Dublin, Ireland, D01 YC76
Actively Recruiting
5
Ascendis Investigational Site
Norwich, United Kingdom, NR4 7UY
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
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