CompletedPhase 2/3

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia

United States, Australia, Canada84 participantsStarted 2023-03-03

Plain-language summary

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly SC doses of 100 µg CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period is followed by an Open Label Extension (OLE) period of a 52-week duration.

Who can participate

Age range

2 Years – 11 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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). * Male or female, between 2 and 11 years of age (inclusive) at the time of Screening. * Clinical diagnosis of ACH with documented genetic confirmation available. * Able to stand without assistance. * Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and to follow the protocol. * At least six months of growth and disease history from ACHieve (TCC-NHS-01) trial or comparable growth and disease history available from medical records (pending confirmation by Medical Monitor). * Considered eligible based on the medical history, physical examination, and the results of vital signs, ECG and clinical laboratory tests performed during the Screening period Exclusion Criteria: * Participation (i.e., signed informed consent) in any interventional clinical trial before within 3 months prior to screening. * Closed epiphysis. * Known or suspected hypersensitivity to the IMP or related products (trehalose, tris\[hydroxymethyl\]aminomethane, succinate, and mPEG). * Have a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoa…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Annualized Growth Velocity

Timeframe: 52 weeks

Trial details

NCT IDNCT05598320

SponsorAscendis Pharma Growth Disorders A/S

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08-09

View on ClinicalTrials.gov More Achondroplasia trials