This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, \& treatments Children's information will be collected in the registry for a maximum of 5 years.
Study Type
OBSERVATIONAL
Enrollment
315
Change From Baseline in Standing Height
Standing height (for participants aged ≥2 years) is an assessment of maximum vertical size. Standing height was measured using a stadiometer with a fixed vertical backboard and an adjustable head piece. Height in supine position (or length for participants aged \<2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (\<2 years and ≥2 years) and gender of the participants.
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Sitting Height
Sitting height (for participants aged ≥2 years) is a measure of the trunk of the body from the buttocks to the top of the head when the participant is sitting upright. Sitting height was measured using a stadiometer and sitting height table. Crown-rump length (for participants aged \<2 years) was measured using measuring board. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (\<2 years and ≥2 years) and gender of the participants.
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Knee Height
Knee height means lower segment length (lower segment=standing height - sitting height). Knee height was measured using knee height caliper. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (\<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Head Circumference
Head circumference is a measurement of the circumference of the child's head at its largest area, above the eyebrows and ears and around the back of the head. Head circumfernce was measured using measuring tape. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (\<2 years, 2-10 years, and ≥10 years) and gender of the participants.
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Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre
Los Angeles, California, United States
Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States
Alfred I. duPont Hospital for Children - Wilmington
Wilmington, Delaware, United States
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
The John Hopkins
Baltimore, Maryland, United States
The Johns Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Boston Childrens Hospital
Boston, Massachusetts, United States
Univesity of Missouri - Columbia
Columbia, Missouri, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
...and 47 more locations
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Change From Baseline in Arm Span
Arm span measurement is the distance between fingertips when the arms are outstretched. All participants should be measured supine lying on the measurement grid. Three valid measurements were performed at each visit and the average of the 3 valid measurements was reported and used in descriptive summaries. Data is reported according to age group (\<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Time frame: Baseline, 1 year, 2 years, 3 years, 4 years
Number of Participants With Achondroplasia-Related Treatments
Achondroplasia-related treatments were collected in a prospective and standardized fashion to allow characterization of the natural history of the disease course in the youngest participants in relation to the level of actual disease burden over time due to treatments, as well as data to support identification of possible risk factors. Data is reported according to the gender of the participants.
Time frame: Baseline to end of study visit (up to 55 months)
Baseline and Post-baseline Measurements of Serum Collagen X Marker
Collagen X Marker (CXM), is a degradation by-product of endochondral ossification that is released into the circulation in proportion to overall growth plate activity. This marker corresponds to the rate of linear bone growth at time of measurement as recently demonstrated in children of normal average growth. Data is reported according to age group (\<2 years, 2-10 years, and ≥10 years) and gender of the participants.
Time frame: Baseline, 1 year, 2 years, 3 years