RecruitingNCT05619900
Metabolic
Maroteaux-Lamy Syndrome
Also known as MPS VI, mucopolysaccharidosis type VI, ARSB deficiency, arylsulfatase B deficiency
Maroteaux-Lamy syndrome (MPS VI) is caused by deficiency of arylsulfatase B (ARSB), resulting in accumulation of dermatan sulfate in lysosomes throughout the body. Unlike MPS I (Hurler), intelligence is generally preserved.
3
studies recruiting now
as of 7 Sept 2026
36
studies registered in total
as of 7 Sept 2026
2
countries with a recruiting site
as of 7 Sept 2026
14 Sept 2023
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 1NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
RecruitingNCT06036693
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Showing the 3 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
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About Maroteaux-Lamy Syndrome
Maroteaux-Lamy syndrome (MPS VI) is caused by deficiency of arylsulfatase B (ARSB), resulting in accumulation of dermatan sulfate in lysosomes throughout the body. Unlike MPS I (Hurler), intelligence is generally preserved. The disease causes severe skeletal dysplasia, cardiac valve disease, corneal clouding, obstructive airway disease, and hepatosplenomegaly. Galsulfase (Naglazyme), the approved enzyme replacement therapy, significantly improves endurance and respiratory function.
Common clinical features
Skeletal dysplasia and short statureCardiac valve diseaseCorneal cloudingJoint stiffnessObstructive sleep apneaHepatosplenomegalyHearing loss
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
1 approved treatment and 2 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Galsulfase (Aryplase)
Phase 2/3Somatropin (Genotropin)
Phase 2Odiparcil
Before you apply
Things trial teams commonly ask about for Maroteaux-Lamy Syndrome. Not eligibility rules; those are set by each study.
- Galsulfase (Naglazyme) ERT is standard — document infusion history, antibody status, and any infusion reactions
- Urinary dermatan sulfate and ARSB enzyme activity are required biomarkers for eligibility documentation
- Six-minute walk test and pulmonary function tests are standard outcome measures — baseline values are eligibility determinants
- Cardiac valve assessment (echocardiogram) is required — severe cardiac disease may affect anesthesia eligibility for trial procedures
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).