Weekly shot may boost growth in kids with dwarfism
NCT ID NCT04085523
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ACHONDROPLASIA are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Achondroplasia Drug Trial for Children (NCT06067425)
- PROPEL Trial: Observing Growth in Children with NCT04035811
- BMN 333 vs vosoritide for achondroplasia in chil NCT07441876
- Bone Disorder Registry Study (NCT02597881)
- Long-Term Study of BMN 111 for Achondroplasia in NCT03989947
- Achondroplasia Growth Study Extension (NCT03424018)
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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