Mucopolysaccharidosis Type IVA (DBCOND0060256)

Identifiers

Synonyms

Mucopolysaccharidosis Type IV A / MPS IVA / Morquio A Disease / Morquio A Syndrome / Morquio Syndrome A / Mucopolysaccharidosis IVA / Mucopolysaccharidosis IVA (Morquio A Syndrome) / Mucopolysaccharidosis IV Type A / Mucopolysaccharidosis type IVA / Morquio A mucopolysaccharidoses / Mucopolysaccharidosis, MPS-IV-A (disorder) / Deficiency of N-acetylgalactosamine-6-sulfatase (finding)

Associated Data

Indicated Drugs and Targets

Drug	Description	Targets
Elosulfase alfa	A lysosomal glycosaminoglycan (GAG)-specific enzyme indicated as an enzyme replacement therapy for Mucopolysaccharidosis type IV A.	No drug targets

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT03655223	Early Check: Expanded Screening in Newborns	No drug interventions	Not Available	Not Available	enrolling_by_invitation
NCT01858103	BMN 110 US Expanded Access Program	Elosulfase alfa	Not Available	Not Available	approved_for_marketing
NCT02294877	A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)	Elosulfase alfa	Not Available	Not Available	completed
NCT04532047	In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases	Alglucosidase alfa Elosulfase alfa Galsulfase Idursulfase Laronidase Sebelipase alfa Vestronidase alfa	treatment	1	recruiting
NCT01920828	Gait Analysis in MPS IVA	No drug interventions	Not Available	Not Available	completed
NCT05284006	Non-invasive Functional Assessment and Pathogenesis of Morquio A	No drug interventions	Not Available	Not Available	recruiting
NCT00884949	A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA	Elosulfase alfa	treatment	1 / 2	completed
NCT01961518	Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome	No drug interventions	Not Available	Not Available	completed
NCT01415427	Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Elosulfase alfa Galactose	treatment	3	completed
NCT01242111	A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Elosulfase alfa	treatment	1 / 2	terminated
NCT01515956	Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Elosulfase alfa Galactose	treatment	2	completed
NCT05845749	Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI	Vosoritide	other	1 / 2	recruiting
NCT03632213	Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI	Losartan	treatment	2	active_not_recruiting
NCT01697319	Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation	Elosulfase alfa Galactose	treatment	2	terminated
NCT01275066	A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Elosulfase alfa	treatment	3	completed
NCT01609062	Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome	Elosulfase alfa Galactose	treatment	2	terminated
NCT00787995	A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)	No drug interventions	Not Available	Not Available	terminated
NCT01733615	Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA	No drug interventions	Not Available	Not Available	terminated
NCT02153255	Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa	No drug interventions	Not Available	Not Available	withdrawn
NCT02208661	Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)	No drug interventions	Not Available	Not Available	completed
NCT01457456	Biomarker for Morquio Disease (BioMorquio)	No drug interventions	Not Available	Not Available	withdrawn
NCT05368038	ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program	No drug interventions	Not Available	Not Available	enrolling_by_invitation
NCT01707433	Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease	No drug interventions	Not Available	Not Available	completed
NCT03204370	Natural History of Atypical Morquio A Disease	Elosulfase alfa	Not Available	Not Available	recruiting
NCT05619900	Registry of Patients Diagnosed With Lysosomal Storage Diseases	No drug interventions	Not Available	Not Available	recruiting
NCT05687474	Baby Detect : Genomic Newborn Screening	No drug interventions	Not Available	Not Available	recruiting
NCT01966029	BMN 110 Phase 3B in Australian Patients	Elosulfase alfa	treatment	3	completed