ACHieve: A Multicenter, Longitudinal, Observational Study of Children With Achondroplasia

Trial Name

ACHieve: A Multicenter, Longitudinal, Observational Study of Children With Achondroplasia

What is the trial about?

The purpose of this study is to observe and study the growth velocity, body proportionality, and complications (comorbidities) in children with achondroplasia.

Who can participate?

Children less than 9 years old who have been diagnosed with achondroplasia (a skeletal growth disorder). Achondroplasia (ACH) is a disorder of bone growth that prevents the changing of cartilage (particularly in the long bones of the arms and legs) into bone. It is caused by a gene mutation. Physical features of ACH include short stature with particular shortening of the upper arm and upper leg, macrocephaly (large head) and a prominent forehead. Complications of ACH include (but not limited to) repeat ear infections, deformities of the spinal column, joint pains and restricted range of motion of the extremities.

What is involved?

Procedures and Activities: There will be no study medication or study treatment involved. Below is a list of all the things that happen during the study.

• Collect medical history
• Review of medications your child may be taking
• Collect demographic information
• Collection of information about your child’s developmental milestones such as lifting head when lying on stomach, roll over, sitting, standing, walking, using single words, short sentences, cup drinking and feeding self
• Measurement of vital signs including heart rate and blood pressure
• Collection of age your child was diagnosed with Achondroplasia
• Body Measurements including: Standing and sitting height, head circumference, chest circumference, body weight, arm span, upper and lower leg length, upper and lower arm length, hand length and foot length
• All assessments and collecting of information will be repeated every 6 months.