Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
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This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.
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Layout table for eligibility information
Ages Eligible for Study:
30 Months to 10 Years (Child)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Children with achondroplasia
Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable)
Aged 2.5 to 10 years (inclusive) at study entry
Diagnosis of ACH
Ambulatory and able to stand without assistance
Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures
Have hypochondroplasia or short stature condition other than ACH (e.g trisomy 21, pseudoachondroplasia, psychosocial short stature)
In females, having had their menarche
Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening
Have a concurrent disease or condition that in the view of the Investigator and/or Sponsor, may impact growth or where the treatment is known to impact growth.
Significant abnormality in screening laboratory results.
Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time
Have received a C-type natriuretic peptide (CNP) analog or treatment targeting fibroblast growth factor receptor (FGFR) inhibition at any time
Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
Have used any other investigational product or investigational medical device for the treatment of ACH or short stature