A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
Achondroplasia
About this trial
This is an interventional treatment trial for Achondroplasia focused on measuring Achondroplasia, Dwarfism
Eligibility Criteria
Inclusion Criteria:
- Clinical diagnosis of ACH with genetic confirmation
- Age between 2 to 10 years old (inclusive) at Screening Visit
- Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit
- Able to stand without assistance
- Caregiver willing and able to administer subcutaneous injections of study drug
Exclusion Criteria:
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
- Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
- Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
- Have received any study drug or device intended to affect stature or body proportionality at any time
- History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones
Sites / Locations
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
- Ascendis Pharma Investigational Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Other
Other
Other
Other
Other
TransCon CNP 6 mcg
TransCon CNP 20 mcg
TransCon CNP 50 mcg
TransCon CNP 100 mcg
TransCon CNP >100 mcg
TransCon CNP 6 mcg CNP/kg or placebo mimicking TransCon CNP 6 mcg delivered once weekly by subcutaneous injection
TransCon CNP 20 mcg CNP/kg or placebo mimicking TransCon CNP 20 mcg delivered once weekly by subcutaneous injection
TransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
TransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
TransCon CNP >100 mcg CNP/kg delivered once weekly by subcutaneous injection (to be determined after completion of 100 mcg cohort)