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

Completed · Phase 2 · Has a placebo group

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT04085523

Run by

Ascendis Pharma A/S

People needed

57

Starts

2020-06-24

Expected to finish

2024-10-01

Last updated by the study team

2026-07-02

Who can join

Age: 2 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Ascendis Pharma Investigational Site — Little Rock, Arkansas, United States
• Ascendis Pharma Investigational Site — Aurora, Colorado, United States
• Ascendis Pharma Investigational Site — Saint Paul, Minnesota, United States
• Ascendis Pharma Investigational Site — Columbia, Missouri, United States
• Ascendis Pharma Investigational Site — Buffalo, New York, United States
• Ascendis Pharma Investigational Site — Houston, Texas, United States
• Ascendis Pharma Investigational Site — Seattle, Washington, United States
• Ascendis Pharma Investigational Site — Madison, Wisconsin, United States
• Ascendis Pharma Investigational Site — Parkville, Victoria, Australia
• Ascendis Pharma Investigational Site — Linz, Austria
• Ascendis Pharma Investigational Site — Copenhagen, Denmark
• Ascendis Pharma Investigational Site — Berlin, Germany
• Ascendis Pharma Investigational Site — Dublin, Ireland
• Ascendis Pharma Investigational Site — Auckland, New Zealand
• Ascendis Pharma Investigational Site — Coimbra, Portugal

Full record on ClinicalTrials.gov

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