Phase 2 N=57 Randomized Quadruple-blind Treatment

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

Achondroplasia

Bottom Line

View on ClinicalTrials.gov: NCT04085523 ↗

Enrolled (actual)

Serious AEs

3.5%

Results posted

Mar 2024

Primary outcome: Primary: Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment — 4.09; 4.52; 5.16; 5.42 cm/year — p== 0.6004

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: TransCon CNP (Drug); Placebo for TransCon CNP (Drug)
Age: Pediatric · 2+ yrs
Sex: All
Sponsor: Ascendis Pharma A/S
Primary completion: Sep 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment	4.09; 4.52; 5.16; 5.42; 4.35	= 0.6004

Summary

The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive, with Achondroplasia.

Eligibility Criteria

Inclusion Criteria

Clinical diagnosis of ACH with genetic confirmation
Age between 2 to 10 years old (inclusive) at Screening Visit
Prepubertal (Stage 1 breasts for girls or testicular volume 3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
Have received any study drug or device intended to affect stature or body proportionality at any time
History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04085523). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.