ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period
TransCon CNP
+ Placebo for TransCon CNP
Achondroplasia+6
+ Bone Diseases
+ Bone Diseases, Developmental
Treatment Study
Summary
Study start date: June 24, 2020
Actual date on which the first participant was enrolled.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, with Achondroplasia.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.57 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 2 to 10 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Inclusion Criteria: 1. Clinical diagnosis of ACH with genetic confirmation 2. Age between 2 to 10 years old (inclusive) at Screening Visit 3. Prepubertal (Stage 1 breasts for girls or testicular volume \< 4ml for boys) at Screening Visit 4. Able to stand without assistance 5. Caregiver willing and able to administer subcutaneous injections of study drug Exclusion Criteria: 1. 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 2. Have received treatment (\>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time 3. Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit 4. Have received any study drug or device intended to affect stature or body proportionality at any time 5. History or presence of injury or disease of the growth plate(s), other than Achondroplasia, that affects growth potential of long bones
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.6 intervention groups are designated in this study
16.667% chance of being blinded to the placebo group
Treatment Groups
Group I
ExperimentalGroup II
ExperimentalGroup III
ExperimentalGroup IV
ExperimentalGroup 5
ExperimentalGroup 6
PlaceboStudy Objectives
Primary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 15 locations
Ascendis Pharma Investigational Site
Little Rock, United StatesOpen Ascendis Pharma Investigational Site in Google MapsAscendis Pharma Investigational Site
Aurora, United StatesAscendis Pharma Investigational Site
Saint Paul, United StatesAscendis Pharma Investigational Site
Columbia, United States