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Results for “Mucopolysaccharidoses”

307 trials

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Showing 20 of 307 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 applicableUnknownNCT02583152
What this trial is testing

New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis

Who this might be right for
Mucopolysaccharidoses
Manchester Royal Eye Hospital 50
Not applicableStudy completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
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 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
Large-scale testing (Phase 3)Active Not RecruitingNCT04348136
What this trial is testing

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 27
Not applicableLooking for participantsNCT05031507
What this trial is testing

Study of Skeletal Disorders

Who this might be right for
Skeletal Disorders
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 100
Not applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
Large-scale testing (Phase 3)Study completedNCT04759885
What this trial is testing

Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep®

Who this might be right for
Elective Colonoscopy
NTC srl 886
Not applicableStudy completedNCT00004738
What this trial is testing

Genetic Analysis of the Chiari I Malformation

Who this might be right for
SyringomyeliaType I Arnold Chiari Malformation
National Institute of Neurological Disorders and Stroke (NINDS) 152
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableUnknownNCT03017677
What this trial is testing

A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis

Who this might be right for
Mucopolysaccharidoses
Mackay Memorial Hospital 100
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
Not applicableStudy completedNCT06209112
What this trial is testing

Sevoflurane Wash in Method Using Conventional Flows Versus Low Flows of 0.5L

Who this might be right for
Inhalation; Gas
Royal Hospital, Oman 48
Not applicableUnknownNCT04320329
What this trial is testing

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Who this might be right for
Morquio B DiseaseGM1 Gangliosidosis Type III
University of British Columbia 30
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
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