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

COMPLETED

NCT04085523

PHASE2

Sponsor: Ascendis Pharma A/S

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.

Official title: 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

Key Details

Gender

All

Age Range

2 Years - 10 Years

Study Type

INTERVENTIONAL

Enrollment

57

Start Date

2020-06-24

Completion Date

2024-10-01

Last Updated

2026-07-02

Healthy Volunteers

No

Conditions

Achondroplasia

Interventions

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.