Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Study Overview
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Brief Summary
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This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

Official Title
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Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

Conditions
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Achondroplasia

Participation Criteria
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Eligibility Criteria
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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 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.

Ages Eligible for Study
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Minimum Age: 30 Months
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in height Z score |  | Up to 2 years | 
| Change from baseline in upper to lower body segment ratio |  | Up to 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Skeletal dysplasia, Endochondral ossification, Achondroplasia (ACH), Shortened proximal limbs, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate dwarfism, Quality of life in achondroplasia, Dwarfism, Bone diseases, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, Inborn, Functional abilities, Average growth velocity, Average height velocity, growth