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Weekly shot may boost growth in kids with dwarfism

NCT ID NCT04085523

Disease control Sponsor: Ascendis Pharma 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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

Study facts

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

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

57 people

The number who actually took part.

Started

Jun 2020

Finished

Oct 2024

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

2 to 10 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: 1. Clinical diagnosis of ACH with genetic confirmation 2. Age between 2 to 10 years old (inclusive) at Screening Visit 3. Prepubertal (Stage 1 breasts for girls or testicular volume \< 4ml for boys) at Screening Visit 4. Able to stand without assistance 5. Caregiver willing and able to administer subcutaneous injections of study drug Exclusion Criteria: 1. 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 2. Have received treatment (\>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time 3. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit 4. Have received any study drug or device intended to affect stature or body proportionality at any time 5. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones

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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

More trials for these conditions

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