RecruitingNCT06675097
Metabolic
Morquio Syndrome
Also known as MPS IV, mucopolysaccharidosis type IV, Morquio-Brailsford disease, Types A and B
Morquio syndrome (MPS IV) is characterized by severe skeletal dysplasia with normal intelligence, distinguishing it from other MPS disorders. MPS IVA (GALNS deficiency) is the most severe form, causing short stature, odontoid hypoplasia wit
ORPHA:309297 ↗Gene GALNS (A)Gene GLB1 (B)Prevalence 1-9 per 100,000 (Orphanet)Onset Infantile, ChildhoodAutosomal recessive genetic
18
studies recruiting now
as of 7 Sept 2026
101
studies registered in total
as of 7 Sept 2026
10
countries with a recruiting site
as of 7 Sept 2026
11 Sept 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT05031507
Study of Skeletal Disorders
RecruitingNCT06410976
Prospective Clinical Assessment Study in Children With Hypochondroplasia
RecruitingPhase 2NCT07169279
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
RecruitingNCT06749366
Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 18 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.
Keep watching
Get an email when a new Morquio Syndrome study opens.
One email a day at most. Unsubscribe with one click.
Used only for these alerts. Privacy.
Support
Patient organisations
Registry: MPS Society Patient Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Morquio Syndrome
Morquio syndrome (MPS IV) is characterized by severe skeletal dysplasia with normal intelligence, distinguishing it from other MPS disorders. MPS IVA (GALNS deficiency) is the most severe form, causing short stature, odontoid hypoplasia with cervical instability, joint laxity, and corneal clouding. MPS IVB is milder. Elosulfase alfa (Vimizim) is approved as ERT for MPS IVA, improving endurance and respiratory function.
Common clinical features
Severe short statureOdontoid hypoplasia and cervical instabilityJoint laxity and painPectus carinatumCorneal cloudingHearing lossRespiratory compromise
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Elosulfase Alfa (Bmn 110, rhgalns, n-acetylgalactosamine-6-sulfatase, chondroitinsulfatase, galactose-6-sulfate sulfatase, ec=3.1.6.4)
Before you apply
Things trial teams commonly ask about for Morquio Syndrome. Not eligibility rules; those are set by each study.
- Cervical spine MRI/CT is required before enrollment in any Morquio trial due to high risk of cervical myelopathy
- Elosulfase alfa (Vimizim) ERT is standard for MPS IVA — document prior ERT history and infusion-related reaction history
- Six-minute walk test (6MWT) and pulmonary function tests are standard eligibility and outcome measures
- MPS IVA versus MPS IVB must be confirmed by GALNS enzyme activity and/or GLB1 genotyping
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).