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Results for “Mucopolysaccharidosis (MPS)”

159 trials

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Showing 20 of 159 results

Early research (Phase 1)Study completedNCT01173016
What this trial is testing

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Who this might be right for
Hurler Syndrome
Masonic Cancer Center, University of Minnesota 11
Not applicableWithdrawnNCT02298699
What this trial is testing

Biomarker for Sly Disease (MPS VII) (BioSly)

Who this might be right for
Developmental DelaySkeletal AbnormalitiesHepatomegaly+1 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Ended earlyNCT01697319
What this trial is testing

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

Who this might be right for
Mucopolysaccharidosis IVAMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 16
Post-approval studies (Phase 4)Study completedNCT02455622
What this trial is testing

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Who this might be right for
Hunter Syndrome
Shire 21
Testing effectiveness (Phase 2)Study completedNCT04018755
What this trial is testing

Open-label Study of Anakinra in MPS III

Who this might be right for
Mucopolysaccharidosis III
Lynda E Polgreen 24
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 participantsNCT07361536
What this trial is testing

Cardiac Structure and Function in MPS

Who this might be right for
MPS IMPS IV AMPS - Mucopolysaccharidosis+1 more
Children's Hospital of Orange County 240
Large-scale testing (Phase 3)Enrolling By InvitationNCT05594992
What this trial is testing

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 80
Large-scale testing (Phase 3)Study completedNCT00146770
What this trial is testing

Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 45
Not applicableUnknownNCT03227042
What this trial is testing

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Who this might be right for
Mucopolysaccharidosis Type IIIB
Allievex Corporation 44
Not applicableWithdrawnNCT04591834
What this trial is testing

Mucopolysaccharidosis Type II Observational

Who this might be right for
Mucopolysaccharidosis II
REGENXBIO Inc.
Not applicableLooking for participantsNCT03604835
What this trial is testing

Mucopolysaccharidosis VII Disease Monitoring Program

Who this might be right for
Mucopolysaccharidosis VIIMPS VIIMPS 7+1 more
Ultragenyx Pharmaceutical Inc 50
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
Large-scale testing (Phase 3)UnknownNCT03612869
What this trial is testing

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

Who this might be right for
Mucopolysaccharidosis Type IIIA
LYSOGENE 20
Early research (Phase 1)Study completedNCT02371226
What this trial is testing

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Who this might be right for
Mucopolysaccharidosis I
ArmaGen, Inc 3
Large-scale testing (Phase 3)Study completedNCT03920540
What this trial is testing

GC1111 in Hunter Syndrom Patients

Who this might be right for
Hunter Syndrome
Green Cross Corporation 32
Large-scale testing (Phase 3)Study completedNCT02230566
What this trial is testing

UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
MPS 7Sly SyndromeMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Not applicableStudy completedNCT05073783
What this trial is testing

Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting

Who this might be right for
Pompe DiseaseMucopolysaccharidosis Type I (MPS I)
Sanofi 57
Testing effectiveness (Phase 2)Ended earlyNCT01299727
What this trial is testing

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome
Shire 12
Testing effectiveness (Phase 2)Study completedNCT02060526
What this trial is testing

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Who this might be right for
Sanfilippo Syndrome
Shire 21
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