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

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.

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Key information

Conditions

Achondroplasia Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Endocrine System Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias

Age range

2 year–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Ascendis Pharma Investigational Site, Parkville, Victoria, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 < 4ml for boys) at Screening Visit
Able to stand without assistance
Caregiver willing and able to administer subcutaneous injections of study drug

Exclusion criteria

Clinically significant findings at Screening that:

are expected to require surgical intervention during participation in the trial or
are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or
otherwise are considered by investigator or Medical Monitor/Medical Expert to make a participant unfit to receive study drug or undergo trial related procedures
Have received treatment (>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

Treatment and study plan

TransCon CNP

Drug

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.

Placebo for TransCon CNP

Drug

Weekly subcutaneously injection of placebo.

Primary outcomes

Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment

Time frame: 52 weeks

The primary efficacy analysis compared the difference in the primary efficacy endpoint between the TransCon CNP treatment group and the pooled placebo group using an ANCOVA model with the annualized height velocity (AHV) at Week 52 as the response variable, treatment (dose groups and placebo) and sex as factors, baseline age and baseline height SDS as the covariates, and based on the Full Analysis Set.

Sponsors and collaborators

Lead sponsor

Ascendis Pharma A/S

Industry

Registry information

Official study 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

Important dates

Study start: 2020
Primary completion: 2022
Study completion: 2024
First posted: Sep 11, 2019
Registry last updated: Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed