Can a weekly shot help children with achondroplasia grow taller?

NCT ID NCT05246033

Completed Disease control Sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This phase 2 trial is testing an experimental drug called TransCon CNP in children with achondroplasia, a common form of dwarfism. The study aims to see if a once-weekly injection under the skin can safely improve growth, measured by height velocity over a year. Participants will be randomly assigned to receive either the drug or a placebo, and neither they nor their doctors will know which they get. After the blinded year, all may receive the active drug in an open-label phase.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: TransCon CNP, an experimental drug designed to provide sustained exposure to C-type natriuretic peptide (CNP) via weekly subcutaneous injection
What this could lead to: If successful, this could lead to a new treatment option to improve growth and reduce complications in children with achondroplasia.
What could go wrong: This is an early-phase trial with a small number of participants, so results may not be conclusive or generalizable. The drug is experimental and may have side effects or fail to show significant benefit.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ACHONDROPLASIA are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

achondroplasia Dwarfism

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ACHONDROPLASIA

Contacts and locations

Locations

Ascendis Pharma Investigational Site

Beijing, 100045, China
Ascendis Pharma Investigational Site

Guangzhou, 510080, China
Ascendis Pharma Investigational Site

Hangzhou, 310053, China
Ascendis Pharma Investigational Site

Shanghai, 20082, China
Ascendis Pharma Investigational Site

Wuhan, 430030, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.