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Results for “Mucinoses”

327 trials

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Showing 20 of 327 results

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
Post-approval studies (Phase 4)Study completedNCT00144768
What this trial is testing

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 25
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Large-scale testing (Phase 3)Study completedNCT01275066
What this trial is testing

Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 177
Early research (Phase 1)Looking for participantsNCT06567769
What this trial is testing

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Who this might be right for
Sanfilippo Syndrome Type A
GC Biopharma Corp 9
Testing effectiveness (Phase 2)Study completedNCT03370653
What this trial is testing

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil

Who this might be right for
Mucopolysaccharidosis VI
Inventiva Pharma 20
Not applicableActive Not RecruitingNCT05634512
What this trial is testing

Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.

Who this might be right for
Hematopoietic Cell TransplantationMucopolysaccharidosis Type I
Masonic Cancer Center, University of Minnesota 13
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 applicableEnrolling By InvitationNCT06103487
What this trial is testing

Long Term Follow-Up for RGX-111

Who this might be right for
Mucopolysaccharidosis I
REGENXBIO Inc. 21
Not applicableStudy completedNCT04035811
What this trial is testing

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 271
Not applicableUnknownNCT04149093
What this trial is testing

The Association Between Calretinin and the Function of Ganglion Cells in Hirschsprung Disease

Who this might be right for
Hirschsprung DiseaseGanglionHirschsprung Disease, Long-Segment+1 more
St. Justine's Hospital 50
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Not applicableStudy completedNCT01509768
What this trial is testing

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

Who this might be right for
Sanfilippo Syndrome Type B
Shire 19
Not applicableLooking for participantsNCT05858606
What this trial is testing

Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients

Who this might be right for
Idiopathic Short Stature
University Hospital, Montpellier 200
Not applicableStudy completedNCT07230821
What this trial is testing

Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes

Who this might be right for
Gas and BloatingDiarrheaConstipation
Radicle Science 500
Large-scale testing (Phase 3)Active Not RecruitingNCT06149403
What this trial is testing

Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Who this might be right for
MPS-IH (Hurler Syndrome)
Orchard Therapeutics 41
Not applicableStudy completedNCT02293382
What this trial is testing

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB (Sanfilippo Syndrome)
Alexion Pharmaceuticals, Inc. 30
Not applicableStudy completedNCT03872713
What this trial is testing

Establishment of Human Cellular Disease Models for Morquio Disease

Who this might be right for
Morquio Disease
CENTOGENE GmbH Rostock 40
Not applicableNot Yet RecruitingNCT06493591
What this trial is testing

Atrial Fibrillation Ablation and Autonomic Modulation Via Thoracoscopic Surgery, 10 Year Follow up.

Who this might be right for
Atrial Fibrillation
Amsterdam UMC 200
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