RecruitingPhase 2NCT06842355
Connective Tissue
Achondroplasia
Also known as short-limbed dwarfism, FGFR3 gain-of-function, classic achondroplasia
Achondroplasia is the most common form of disproportionate short stature, caused by a recurrent gain-of-function variant (p.
ORPHA:15 ↗Gene FGFR3Prevalence 1 in 15,000–40,000Onset CongenitalGenetic — autosomal dominant (80% de novo)
11
studies recruiting now
as of 7 Sept 2026
47
studies registered in total
as of 7 Sept 2026
17
countries with a recruiting site
as of 7 Sept 2026
2 Mar 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2 / Phase 3NCT07441876
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
RecruitingPhase 2NCT07169279
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
RecruitingNCT06168201
VIrtual STudy in Achondroplasia for the US (VISTA)
RecruitingPhase 2NCT06732895
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 11 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: CLARITY (Achondroplasia International Registry) · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Achondroplasia
Achondroplasia is the most common form of disproportionate short stature, caused by a recurrent gain-of-function variant (p.Gly380Arg) in FGFR3, which encodes fibroblast growth factor receptor 3, resulting in constitutive inhibition of endochondral bone ossification. Clinical features include rhizomelic shortening of limbs, macrocephaly with midface hypoplasia, trident hand configuration, and lumbar hyperlordosis, with intelligence and life expectancy generally normal in the absence of complications. Neurological complications from foramen magnum stenosis and spinal canal narrowing represent the most serious potential morbidities.
Common clinical features
Disproportionate short stature with rhizomelic (proximal) limb shorteningMacrocephaly and frontal bossingMidface hypoplasia and depressed nasal bridgeTrident hand configurationLumbar hyperlordosis and thoracolumbar kyphosis in infancyForamen magnum stenosis with risk of cervicomedullary compressionSpinal stenosis causing neurogenic claudication in adults
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment and 5 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Vosoritide (Voxzogo)
Phase 3Infigratinib
Phase 2/3Navepegritide
Phase 2Recifercept
Phase 2Lonapegsomatropin (Skytrofa)
Phase 1Sodium Chloride (Aqsia (balanced salt soln))
Before you apply
Things trial teams commonly ask about for Achondroplasia. Not eligibility rules; those are set by each study.
- Height, growth velocity, and limb-length measurements are primary endpoints in most trials — ensure current anthropometric data (within 3 months) is available using condition-specific growth charts.
- Most approved and investigational therapies (vosoritide, CNP analogues, FGFR3 inhibitors) have specific age and weight cutoffs; confirm current age, weight, and growth plate status before applying.
- Prior or current growth hormone therapy, limb-lengthening surgery, or foramen magnum decompression must be disclosed as these significantly affect trial eligibility and stratification.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).