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Growth Hormone Therapy

Navepegritide for Achondroplasia

Phase 2
Recruiting
Research Sponsored by Ascendis Pharma A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks

Summary

"This trial will test the safety and effectiveness of a new medication called Navepegritide in infants under 2 years old with a specific genetic condition. The medication will be given once a week

Who is the study for?
This trial is for infants under 2 years with achondroplasia, a form of dwarfism. Parents must consent and be able to give weekly shots at home. Babies up to 1 year old need daily Vitamin D; older ones with low levels must start supplements before joining.
What is being tested?
The trial tests Navepegritide (TransCon CNP) against a placebo in babies with achondroplasia. It's given as a shot once a week for a year. The goal is to see if it's safe and works better than no treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To evaluate the effect of Navepegritide on growth
To evaluate the safety and tolerability of Navepegritide

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: NavepegritideExperimental Treatment1 Intervention
Once weekly double-blinded treatment with SC injection of 100 µg/kg of Navepegritide for 52 weeks
Group II: Placebo for NavepegritidePlacebo Group1 Intervention
Once weekly double-blinded treatment with SC injection of 100 µg/kg of Placebo for Navepegritide for 52 weeks

Find a Location

Who is running the clinical trial?

Ascendis Pharma A/SLead Sponsor
14 Previous Clinical Trials
1,688 Total Patients Enrolled
4 Trials studying Achondroplasia
365 Patients Enrolled for Achondroplasia
Ascendis Pharma Growth Disorders A/SLead Sponsor
3 Previous Clinical Trials
246 Total Patients Enrolled
3 Trials studying Achondroplasia
246 Patients Enrolled for Achondroplasia
~40 spots leftby Mar 2026