NCT04085523

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

ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 57 (actual)
• Sponsor: Ascendis Pharma A/S · Industry
• Sex: All
• Age: 2 Years – 10 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Achondroplasia

Interventions

Type	Name	Description
DRUG	TransCon CNP	TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.
DRUG	Placebo for TransCon CNP	Weekly subcutaneously injection of placebo.

Timeline

• Start date: 2020-06-24
• Primary completion: 2022-09-27
• Completion: 2024-10-01
• First posted: 2019-09-11
• Last updated: 2025-05-22
• Results posted: 2024-03-07

Locations

15 sites across 8 countries: United States, Australia, Austria, Denmark, Germany, Ireland, New Zealand, Portugal

Regulatory

• FDA-regulated drug study