Mucopolysaccharidosis IVA

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Mucopolysaccharidosis IVA trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 4 trials

RecruitingNot Applicable

NCT05619900

Registry of Patients Diagnosed With Lysosomal Storage Diseases

This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the dise…

United States250 participantsStarted 2022-05-31

Treatment: There is no intervention

RecruitingPhase 1

NCT04532047

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and…

United States10 participantsStarted 2021-07-01

Treatment: Aldurazyme (laronidase)

RecruitingNot Applicable

NCT07361536

Cardiac Structure and Function in MPS

The purpose of this study is to better understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS). The investigators are…

United States240 participantsStarted 2025-09-01

RecruitingNot Applicable

NCT05284006

Non-invasive Functional Assessment and Pathogenesis of Morquio A

Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establi…

United States60 participantsStarted 2021-05-01

Treatment: Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.

All recent trials (23 shown)

Active — Not RecruitingPhase 1/2

NCT05845749

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI.…

United States6 participantsStarted 2023-09-25

Treatment: Vosoritide Injection [Voxzogo]

CompletedNot Applicable

NCT05687474

Baby Detect : Genomic Newborn Screening

Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…

Belgium6,824 participantsStarted 2022-09-01

Mucopolysaccharidosis IVA

Trial phase breakdown

Trial status

Actively recruiting — 4 trials

Registry of Patients Diagnosed With Lysosomal Storage Diseases

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Cardiac Structure and Function in MPS

Non-invasive Functional Assessment and Pathogenesis of Morquio A

All recent trials (23 shown)

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Baby Detect : Genomic Newborn Screening

Trial phase breakdown

Trial status

Actively recruiting — 4 trials

Registry of Patients Diagnosed With Lysosomal Storage Diseases

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Cardiac Structure and Function in MPS

Non-invasive Functional Assessment and Pathogenesis of Morquio A

All recent trials (23 shown)

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

Baby Detect : Genomic Newborn Screening

Early Check: Expanded Screening in Newborns

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Gait Analysis in MPS IVA

BMN 110 Phase 3B in Australian Patients

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A S…

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio…

A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

BMN 110 US Expanded Access Program

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics