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Results for “Mucopolysaccharidosis Disorders”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableActive Not RecruitingNCT05991609
What this trial is testing

Extreme Morphology and Metabolic Health

Who this might be right for
Skeletal Dysplasia
University of Bath 102
Testing effectiveness (Phase 2)Ended earlyNCT02350816
What this trial is testing

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Who this might be right for
Sanfilippo SyndromeMucopolysaccharidosis (MPS)
Shire 17
Large-scale testing (Phase 3)Active Not RecruitingNCT04573023
What this trial is testing

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Who this might be right for
Mucopolysaccharidosis II
JCR Pharmaceuticals Co., Ltd. 86
Post-approval studies (Phase 4)Study completedNCT00144781
What this trial is testing

A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

Who this might be right for
Mucopolysaccharidosis IHurler's SyndromeHurler-Scheie Syndrome+1 more
Genzyme, a Sanofi Company 34
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319
What this trial is testing

Study to Evaluate the Safety and Efficacy of Adalimumab 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 14
Testing effectiveness (Phase 2)Study completedNCT04571970
What this trial is testing

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Who this might be right for
Mucopolysaccharidosis Type II (MPS II)
REGENXBIO Inc. 6
Not applicableLooking for participantsNCT05247645
What this trial is testing

Data Collection of Patients With Rare Bone Diseases

Who this might be right for
Skeletal Dysplasia
Luca Sangiorgi 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
Not applicableStudy completedNCT05006222
What this trial is testing

The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis

Who this might be right for
Mucopolysaccharidosis
Hasan Kalyoncu University 12
Large-scale testing (Phase 3)UnknownNCT01238328
What this trial is testing

Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Who this might be right for
Mucopolysaccharidosis
Tehran University of Medical Sciences 10
Not applicableWithdrawnNCT00286689
What this trial is testing

Effects of Growth Hormone in Chronically Ill Children

Who this might be right for
Hurler SyndromeCerebral PalsyJuvenile Rheumatoid Arthritis+2 more
University of Texas Southwestern Medical Center
Not applicableUnknownNCT01695161
What this trial is testing

Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.

Who this might be right for
MucopolysaccharidosisFabry Disease
Johannes Gutenberg University Mainz 75
Testing effectiveness (Phase 2)Study completedNCT03423186
What this trial is testing

Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Who this might be right for
Sanfilippo Syndrome Type A (MPS IIIA)
Swedish Orphan Biovitrum 6
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT06022380
What this trial is testing

Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II

Who this might be right for
Mucopolysaccharidosis Type II
Manchester University NHS Foundation Trust 46
Large-scale testing (Phase 3)Study completedNCT06164951
What this trial is testing

Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 114
Not applicableStudy completedNCT04112602
What this trial is testing

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis

Who this might be right for
Mucopolysaccharidoses
University Hospital, Tours 27
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 applicableStudy completedNCT01602601
What this trial is testing

Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

Who this might be right for
Mucopolysaccharidosis II
GlaxoSmithKline 10
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