Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

 Achondroplasia / Posted 3 years ago

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.

  • 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 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
  • 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 less than−2 or more than +2 standard deviations for age and sex based on reference tables on growth in children with ACH
    • Annualized height growth velocity less than 1.5 cm/year over a period more than 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 (more than 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 (more than 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
    • Have had previous limb-lengthening surgery
  • Study start date : 12/07/2019
  • Study end date : 01/06/2026
  • Wales-Based Study Contact : please speak to your clinician
Contact details

Birmingham Children's Hospital, Bristol Royal Hospital for Children, Bristol, United Kingdom, Queen Elizabeth University Hospital, Glasgow, United Kingdom, Guy's and Saint Thomas' NHS Foundation Trust, Manchester University Children's Hospital, Sheffield Children's Hospital. England

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