A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Childre… (NCT05598320) | Clinical Trial Compass
CompletedPhase 2/3
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
United States, Australia84 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 range2 Years – 11 Years
SexALL
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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…