Ascendis starts an observational study for achondroplasia
The ACHieve is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. 1
Measuring height velocity in children with achondroplasia has been central in drug research and development but is extremely important to consider the reduction of occurrence and recurrence of complications that tend to happen in children with achondroplasia, especially at very young age, as for example, foramen magnum stenosis. Ascendis is also going to study these comorbidities, that are the additional conditions that co-occur and are concomitant with achondroplasia.
This is a natural history study and no study medication will be administered1. Nevertheless, only children that participate in the ACHieve study will be invited after to join the clinical trial with TransCon CNP.
This is a natural history study and no study medication will be administered1. Nevertheless, only children that participate in the ACHieve study will be invited after to join the clinical trial with TransCon CNP.
- 200 children will be recruited from diverse clinical centers, with ages up to 8 years old.
- The study will collect data for the annualized height velocity (centimeters/year) in children with achondroplasia with measurements on Day 1 and then every 6 months over the study period with height velocity reported in cm/year.
- It also will collect natural history symptoms in children with achondroplasia as a prospective study.
- Is expected to be complete in 5 years, around 2024.
A prospective study watches for outcomes, such as the development of a disease, during the study period and relates this to other factors such as suspected risk or protection factor(s). The study usually involves taking a cohort of subjects and watching them over a long period.2
Inclusion Criteria1:
- A legally authorized representative is willing and able to provide written, signed informed consent (with a written assent from the child when appropriate per local requirements)
- Willing and able to comply with study protocol per investigator judgment
- Clinical diagnosis of achondroplasia (confirmed by the investigator)
- Age between 0 to 8 years old at enrollment
- Able to stand without assistance (if the child is 24 months or older)
How to enroll a child in this study?
Contact the study coordinator of the clinical center of your selection and request information about the study. The clinical centers will start to open and enroll gradually.
Locations
United States, Arkansas | |
Little Rock, Arkansas, United States, 72211 | Recruiting |
United States, California | |
Oakland, California, United States, 94609 | Not yet recruiting |
United States, Delaware | |
Wilmington, Delaware, United States, 19803 | Not yet recruiting |
United States, Georgia | |
Atlanta, Georgia, United States, 30322 | Not yet recruiting |
United States, Missouri | |
Columbia, Missouri, United States, 65212 | Not yet recruiting |
United States, Washington | |
Seattle, Washington, United States, 98105 | Not yet recruiting |
Australia, Victoria | |
Parkville, Victoria, Australia, 3052 | Not yet recruiting |
Canada | |
Montréal, Canada, H3T 1C5 | Not yet recruiting |
Germany | |
Berlin, Germany, 13353 | Not yet recruiting |
United Kingdom | |
Liverpool, United Kingdom, LA12 2AP | Not yet recruiting |
Any queries and questions contact: This email address is being protected from spambots. You need JavaScript enabled to view it.
1. clinicaltrials.gov
2. Stats Direct