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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"
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.
ACHieve in numbers 1

  • 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:

  1. 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)
  2. Willing and able to comply with study protocol per investigator judgment
  3. Clinical diagnosis of achondroplasia (confirmed by the investigator)
  4. Age between 0 to 8 years old at enrollment
  5. 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.

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
Montréal, Canada, H3T 1C5 Not yet recruiting
Berlin, Germany, 13353 Not yet recruiting
United Kingdom
Liverpool, United Kingdom, LA12 2AP Not yet recruiting

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2. Stats Direct
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