Clinical trial NCT05929807: A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
Official title: A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children and Adolescents With Achondroplasia
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
PHASE3
Conditions: Achondroplasia
Keywords: Achondroplasia
Dwarfism
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2023-07-03
Enrollment: 140
Sex: ALL
Minimum age (years): 3
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Patients will roll over from previous TransCON CNP clinical trials
Primary purpose: TREATMENT

Brief 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 criteria: 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 outcomes:
measure: Safety and Tolerability
timeFrame: Through trial completion, an average of 10 years
measure: Height Z-scores
timeFrame: Through trial completion, an average of 10 years
Secondary outcomes:
measure: Annualized Growth Velocity
timeFrame: Through trial completion, an average of 10 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Ascendis Pharma Investigational Site
state: Arkansas
city: Aurora
country: United States
facility: Ascendis Investigational Site
state: Colorado
city: Wilmington
country: United States
facility: Ascendis Investigational Site
state: Delaware
city: Saint Paul
country: United States
facility: Ascendis Investigational Site
state: Minnesota
city: Columbia Falls
country: United States
facility: Ascendis Pharma Investigational Site
state: Montana
city: Buffalo
country: United States
facility: Ascendis Pharma Investigational Site
state: New York
city: Houston
country: United States
facility: Ascendis Investigational Site
state: Texas
city: Madison
country: United States
facility: Ascendis Pharma Investigational Site
state: Wisconsin
city: Parkville
country: Australia
facility: Ascendis Pharma Investigational Site
state: Victoria
city: Linz
country: Austria
facility: Ascendis Investigational Site
city: Copenhagen
country: Denmark
facility: Ascendis Pharma Investigational Site
city: Berlin
country: Germany
facility: Ascendis Investigational Site
city: Dublin
country: Ireland
facility: Ascendis Pharma Investigational Site
city: Auckland
country: New Zealand
facility: Ascendis Investigational Site
city: Coimbra
country: Portugal
facility: Ascendis Investigational Site
Study officials:
affiliation: Ascendis Pharma
name: Vibeke Breinholt
role: STUDY_DIRECTOR
References: