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