Prospective Clinical Assessment Study in Children with Achondroplasia (ACH)

Participation Requirements

• Sex:

  ALL

• Eligible Ages:

  3 to 17

Participation Criteria

Key Inclusion Criteria:

* 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 \<17 years at study entry
* Diagnosis of ACH
* Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures

Key Exclusion Criteria:

* 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
* Current evidence of corneal or retinal disorder/keratopathy
* Current evidence of endocrine alterations of calcium/phosphorus homeostasis
* 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 had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
* Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.