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Results for “Carbohydrate Metabolism, Inborn Errors”

740 trials

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Showing 20 of 740 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
Large-scale testing (Phase 3)Active Not RecruitingNCT04910776
What this trial is testing

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Who this might be right for
Glycogen Storage Disease Type II
Sanofi 17
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
Not applicableStudy completedNCT04081272
What this trial is testing

Effect of G6PD Deficiency on Red Blood Cell Storage

Who this might be right for
G6PD Deficiency
Columbia University 40
Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
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
Post-approval studies (Phase 4)Study completedNCT00243542
What this trial is testing

Phase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD Deficiency

Who this might be right for
Acne Vulgaris
Allergan 64
Not applicableStudy completedNCT03173495
What this trial is testing

Effects of Exercise on Fructose-induced Postprandial Lipemia

Who this might be right for
Sedentary LifestyleDyslipidemiasLipid Metabolism Disorders+1 more
Federal University of Rio Grande do Sul 12
Not applicableStudy completedNCT04198987
What this trial is testing

Dietary Monosaccharide Supplementation in Patients With Congenital Disorders of Glycosylation

Who this might be right for
Congenital Disorder of Glycosylation
Mayo Clinic 20
Post-approval studies (Phase 4)UnknownNCT02525172
What this trial is testing

Immune Modulation Therapy for Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 8
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 applicableUnknownNCT02503267
What this trial is testing

"Incidence and Consequences of Disorders of Glycosylation in Patients With Conotruncal and Septal Heart Defects"

Who this might be right for
Congenital Heart DiseasesConotruncal DefectsCongenital Disorder of Glycosylation+1 more
Hospital Universitari Vall d'Hebron Research Institute 300
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
Very early researchStudy completedNCT03665636
What this trial is testing

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Who this might be right for
Glycogen Storage Disease Type I
Areeg El-Gharbawy 4
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
Not applicableNot Yet RecruitingNCT07337707
What this trial is testing

New Microbiota-endocrine Axis in Fructose Malabsorption-caused Visceral Hypersensitivity in Irritable Bowel Syndrome.

Who this might be right for
Irritable Bowel Syndrome (IBS)
University Hospital, Rouen 60
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 applicableWithdrawnNCT01457443
What this trial is testing

Biomarker for Pompe Disease (BioPompe)

Who this might be right for
Cardiac DiseasesMuscular WeaknessHepato-splenomegaly+4 more
CENTOGENE GmbH Rostock
Early research (Phase 1)Ended earlyNCT02795325
What this trial is testing

DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

Who this might be right for
Primary Hyperoxaluria Type 1
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company 41
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