Weekly shot may boost growth in kids with dwarfism

NCT ID NCT04085523

Completed Disease control Sponsor: Ascendis Pharma A/S Source: ClinicalTrials.gov ↗

First seen Jul 02, 2026 · Last updated Jul 02, 2026

Summary

This study tests a weekly injection called TransCon CNP in children aged 2 to 10 with achondroplasia, the most common form of dwarfism. The goal is to see if it can safely improve growth over one year. Half the children receive the drug and half receive a placebo, and all children have the option to receive the drug in an extension period.

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 (a long-acting form of C-type natriuretic peptide)
What this could lead to: If successful, this could provide a non-surgical treatment option to improve growth in children with achondroplasia.
What could go wrong: This is an early-phase trial with a small number of participants. The treatment may not improve growth significantly, and side effects are possible.

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

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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

Little Rock, Arkansas, 72211, United States
Ascendis Pharma Investigational Site

Aurora, Colorado, 80045, United States
Ascendis Pharma Investigational Site

Saint Paul, Minnesota, 55102, United States
Ascendis Pharma Investigational Site

Columbia, Missouri, 65212, United States
Ascendis Pharma Investigational Site

Buffalo, New York, 14203, United States
Ascendis Pharma Investigational Site

Houston, Texas, 77030, United States
Ascendis Pharma Investigational Site

Seattle, Washington, 98105, United States
Ascendis Pharma Investigational Site

Madison, Wisconsin, 53705, United States
Ascendis Pharma Investigational Site

Parkville, Victoria, 3052, Australia
Ascendis Pharma Investigational Site

Linz, 4020, Austria
Ascendis Pharma Investigational Site

Copenhagen, 2100, Denmark
Ascendis Pharma Investigational Site

Berlin, 13353, Germany
Ascendis Pharma Investigational Site

Dublin, D01 YC76, Ireland
Ascendis Pharma Investigational Site

Auckland, 1023, New Zealand
Ascendis Pharma Investigational Site

Coimbra, 3000-602, Portugal

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