Status:
COMPLETED
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia
Lead Sponsor:
Ascendis Pharma A/S
Conditions:
Achondroplasia
Eligibility:
All Genders
2-10 years
Phase:
PHASE2
Brief Summary
The trial is a multicenter, double-blind, randomized, placebo-controlled, dose escalation trial of weekly TransCon CNP administered subcutaneously in prepubertal children 2 to 10 years old, inclusive,...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 24 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2024
Estimated Enrollment :
57 Patients enrolled
Trial Details
Trial ID
NCT04085523
Start Date
June 24 2020
End Date
October 1 2024
Last Update
May 22 2025
Active Locations (15)
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1
Ascendis Pharma Investigational Site
Little Rock, Arkansas, United States, 72211
2
Ascendis Pharma Investigational Site
Aurora, Colorado, United States, 80045
3
Ascendis Pharma Investigational Site
Saint Paul, Minnesota, United States, 55102
4
Ascendis Pharma Investigational Site
Columbia, Missouri, United States, 65212