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Results for “MPS VI”

37 trials

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Showing 20 of 37 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT05845749
What this trial is testing

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

Who this might be right for
MPS IVAMPS VI
University of California, San Francisco 6
Not applicableStudy completedNCT01586871
What this trial is testing

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Who this might be right for
Mucopolysaccharidoses
University of Minnesota 1,147
Not applicableStudy completedNCT01521429
What this trial is testing

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Who this might be right for
Mucopolysaccharidoses
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 55
Large-scale testing (Phase 3)Study completedNCT00104234
What this trial is testing

Study of rhASB in Patients With Mucopolysaccharidosis VI

Who this might be right for
Mucopolysaccharidosis VI
BioMarin Pharmaceutical 39
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
Early research (Phase 1)Study completedNCT00048620
What this trial is testing

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Who this might be right for
Mucopolysaccharidosis VI
BioMarin Pharmaceutical
Large-scale testing (Phase 3)Ended earlyNCT00748969
What this trial is testing

Clinical Trial of Growth Hormone in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
Testing effectiveness (Phase 2)Study completedNCT00048711
What this trial is testing

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Who this might be right for
Mucopolysaccharidosis VI
BioMarin Pharmaceutical
Not applicableLooking for participantsNCT06036693
What this trial is testing

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis III+5 more
Institut National de la Santé Et de la Recherche Médicale, France 1,000
Not applicableStudy completedNCT01387854
What this trial is testing

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

Who this might be right for
MPS VI
BioMarin Pharmaceutical 59
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
Not applicableEnded earlyNCT01675674
What this trial is testing

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

Who this might be right for
MucopolysaccharidosesMucopolysaccharidosis IMucopolysaccharidosis II+2 more
National MPS Society 3,000
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
Not applicableEnded earlyNCT02156674
What this trial is testing

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Who this might be right for
Maroteaux-Lamy Syndrome
Masonic Cancer Center, University of Minnesota 1
Not applicableWithdrawnNCT01458613
What this trial is testing

Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)

Who this might be right for
Lysosomal Storage DiseaseLung DiseasesObstructive Sleep Apnoea+2 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)UnknownNCT03632213
What this trial is testing

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Who this might be right for
Mucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidoses+6 more
Hospital de Clinicas de Porto Alegre 10
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
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