Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Study Overview
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Brief Summary
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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.

Official Title
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An International, Prospective Registry Investigating the Natural History of Participants With Achondroplasia

Conditions
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Achondroplasia

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out The child has been diagnosed as having achondroplasia documented by clinical diagnosis The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent The investigator has considered the family and prospective participating child being able to comply with the study procedures Exclusion Criteria: The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital [SEDC], pseudoachondroplasia, trisomy 21) The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus [SLE], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature Has had bone-related surgery impacting assessment of anthropometric measurements or is expected to have it during the study period. Children with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the date of consent/assent and healing is complete without sequelae as determined by the investigator Has any condition that in the view of the investigator places the child at high risk of poor compliance with the visit schedule or of not completing the study. Any concurrent disease or condition that in the view of the investigator would interfere with study participation

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 15 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Collection of Natural History of Achondroplasia Characteristics in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia | To quantify the number & type of Achondroplasia Characteristics by review of medical records | Up to 5 Years | 
| Collection of Natural History of Achondroplasia Symptoms in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia | To quantify the number & type of Achondroplasia Symptoms by review of medical records | Up to 5 Years | 
| Collection of Natural History of Achondroplasia related Tests & Treatments in a cohort of Children aged 0-10 years old diagnosed with Achondroplasia | To quantify the number & type of Achondroplasia related Tests & Treatments by review of medical records | Up to 5 Years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measurement of biomarkers for bone growth | changes from Baseline in blood samples of collagen fragments | Baseline, Month 12, Month 24, Month 36, Month 48, Month 60 |