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Results for “Mucopolysaccharidoses”

313 trials

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Showing 20 of 313 results

Post-approval studies (Phase 4)Study completedNCT05134571
What this trial is testing

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Who this might be right for
Mucopolysaccharidosis I
Genzyme, a Sanofi Company 12
Not applicableStudy completedNCT01961518
What this trial is testing

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

Who this might be right for
Morquio Syndrome AMaroteaux Lamy SyndromeMPS IVA+1 more
Greenwood Genetic Center 17
Large-scale testing (Phase 3)Not Yet RecruitingNCT07579910
What this trial is testing

Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)

Who this might be right for
MPS IIIB
Spruce Biosciences 14
Testing effectiveness (Phase 2)Active Not RecruitingNCT04201405
What this trial is testing

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Who this might be right for
Mucopolysaccharidosis Type IIIA
University of Manchester 5
Early research (Phase 1)Looking for participantsNCT05682144
What this trial is testing

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

Who this might be right for
Mucopolysaccharidosis IH/SMucopolysaccharidosis IS
Immusoft of CA, Inc. 11
Not applicableEnded earlyNCT00787995
What this trial is testing

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

Who this might be right for
MPS IV AMucopolysaccharidosis IVAMorquio A Syndrome
BioMarin Pharmaceutical 353
Not applicableEnded earlyNCT02232477
What this trial is testing

Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I

Who this might be right for
Mucopolysaccharidosis ICognitive Decline
agnes chen 6
Testing effectiveness (Phase 2)Study completedNCT01301898
What this trial is testing

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Who this might be right for
Mucopolysaccharidosis II
Green Cross Corporation 31
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
Testing effectiveness (Phase 2)Looking for participantsNCT07169279
What this trial is testing

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 77
Not applicableStudy completedNCT03582449
What this trial is testing

Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)

Who this might be right for
Hunter Syndrome
Shire 5
Post-approval studies (Phase 4)Study completedNCT00779493
What this trial is testing

Curcumin (Tumeric) in the Treatment of Irritable Bowel Syndrome: A Randomized-Controlled Trial

Who this might be right for
Irritable Bowel Syndrome
Kaiser Permanente 17
Early research (Phase 1)Study completedNCT04539340
What this trial is testing

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Who this might be right for
Mucopolysaccharidosis Type IIMetabolic Diseases
AO GENERIUM 20
Not applicableStudy completedNCT05648851
What this trial is testing

A Natural History Study of Sanfilippo Syndrome Type D

Who this might be right for
Sanfilippo Syndrome Type D
Phoenix Nest 10
Testing effectiveness (Phase 2)Study completedNCT02437253
What this trial is testing

Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 2
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 completedNCT00852358
What this trial is testing

Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Who this might be right for
Cognitive DeclineMucopolysaccharidosis IHurler-Scheie Syndrome+2 more
Patricia I. Dickson, M.D. 9
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Large-scale testing (Phase 3)Study completedNCT00069641
What this trial is testing

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Who this might be right for
Mucopolysaccharidosis II
Shire 96
Not applicableEnded earlyNCT02293408
What this trial is testing

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

Who this might be right for
MPS IIIB (Sanfilippo B Syndrome)
Alexion Pharmaceuticals, Inc. 30
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