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Weekly shot shows promise for helping kids with dwarfism grow

NCT ID NCT05929807

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 18, 2026 · Updated 4 times

Summary

This study tests a weekly injection called TransCon CNP in children and teens with achondroplasia, the most common form of dwarfism. The goal is to see if it is safe and helps improve growth over several years. Participants who completed a previous TransCon CNP trial can join this long-term extension, receiving the same dose until their bones stop growing.

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 weekly injection of C-type natriuretic peptide)
What this could lead to
If successful, this treatment could help children with achondroplasia grow taller and improve bone development, potentially reducing some complications of the condition.
What could go wrong
This is an early-to-mid stage trial with no placebo group, so results may be less reliable. Possible side effects include injection site reactions and unknown long-term risks.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 140 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Mar 2039

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 to 15 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent. * Participants with achondroplasia who have completed a clinical trial with TransCon CNP. * Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of TransCon CNP and to follow the protocol. * Considered eligible based on the safety evaluations performed for evaluating stopping/holding rule criteria during the prior TransCon CNP clinical trial. Exclusion Criteria: * Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris\[hydroxymethyl\]aminomethane, succinate, and methoxy polyethylene glycol \[mPEG\]). * Have received any dose of prescription medications, investigational medicinal product (other than TransCon CNP). * Sexually active female participants and female partners of male participants of childbearing potential not using a highly effective form of contraceptive (including oral, injectable, or implantable contraception, or intrauterine device (IUD)) for the entire trial period and for 90 days post end of the trial. * Participants with serum 25-hydroxy-vitamin D (25OHD) levels of \<50 nmol/L (\<20 ng/mL) at Visit 1 must be on treatment regimen of Vitamin D supplementation. * Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    19 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ascendis Investigational Site

    Aurora, Colorado, 80045, United States

  • Ascendis Investigational Site

    Wilmington, Delaware, 19803, United States

  • Ascendis Investigational Site

    Saint Paul, Minnesota, 55102, United States

  • Ascendis Investigational Site

    Houston, Texas, 77030, United States

  • Ascendis Investigational Site

    Linz, 4020, Austria

  • Ascendis Investigational Site

    Montreal, H3T 1C5, Canada

  • Ascendis Investigational Site

    Berlin, 13353, Germany

  • Ascendis Investigational Site

    Auckland, 1023, New Zealand

  • Ascendis Investigational Site

    Coimbra, 3000-602, Portugal

  • Ascendis Investigational Site

    Vitoria-Gasteiz, 1008, Spain

  • Ascendis Investigational Site

    London, SE1 7EH, United Kingdom

  • Ascendis Pharma Investigational Site

    Little Rock, Arkansas, 72202, United States

  • Ascendis Pharma Investigational Site

    Columbia Falls, Montana, 65212, United States

  • Ascendis Pharma Investigational Site

    Buffalo, New York, 14203, United States

  • Ascendis Pharma Investigational Site

    Madison, Wisconsin, 53705, United States

  • Ascendis Pharma Investigational Site

    Parkville, Victoria, 3052, Australia

  • Ascendis Pharma Investigational Site

    Copenhagen, 2100, Denmark

  • Ascendis Pharma Investigational Site

    Paris, 75743, France

  • Ascendis Pharma Investigational Site

    Dublin, D01 YC76, Ireland

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