Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
QED Therapeutics, a BridgeBio company
Age
2–17
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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.
Clareo Health | Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
