A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
TransCon CNP: TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.
Study summary
TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.
Eligibility
Sex
ALL
Min age
3 Years
Max age
15 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Safety and Tolerability — Through trial completion, an average of 10 years Incidence of Treatment-Emergent Adverse Events
Height Z-scores — Through trial completion, an average of 10 years Number of standard deviations
Trial sites (19)
Facility
City
Region
Status
Ascendis Pharma Investigational Site
Little Rock
Arkansas
Ascendis Investigational Site
Aurora
Colorado
Ascendis Investigational Site
Wilmington
Delaware
Ascendis Investigational Site
Saint Paul
Minnesota
Ascendis Pharma Investigational Site
Columbia Falls
Montana
Ascendis Pharma Investigational Site
Buffalo
New York
Ascendis Investigational Site
Houston
Texas
Ascendis Pharma Investigational Site
Madison
Wisconsin
Ascendis Pharma Investigational Site
Parkville
Victoria
Ascendis Investigational Site
Linz
Austria
Ascendis Investigational Site
Montreal
Canada
Ascendis Pharma Investigational Site
Copenhagen
Denmark
Ascendis Pharma Investigational Site
Paris
France
Ascendis Investigational Site
Berlin
Germany
Ascendis Pharma Investigational Site
Dublin
Ireland
Ascendis Investigational Site
Auckland
New Zealand
Ascendis Investigational Site
Coimbra
Portugal
Ascendis Investigational Site
Vitoria-Gasteiz
Spain
Ascendis Investigational Site
London
United Kingdom
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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