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Results for “Carbohydrate Metabolism, Inborn Errors”

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Showing 20 of 0 results

Not applicableStudy completedNCT04531033
What this trial is testing

Does Daily Supplementation of Lactobacillus Acidophilus MPH734, for One Week, Affect Acute (Immediate), Subacute (7 Days), and Post-treatment Discontinuation Lactose Metabolism, Gastrointestinal Symptoms, and Clinical Markers of Inflammation and Safety Compared to a Placebo

Who this might be right for
Lactose Intolerance
Lemuel W. Taylor IV 50
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Post-approval studies (Phase 4)Study completedNCT00486889
What this trial is testing

Growth and Development Study of Alglucosidase Alfa

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease
Genzyme, a Sanofi Company 12
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 completedNCT02434952
What this trial is testing

Safety and Tolerability of Low Dose Primaquine

Who this might be right for
Malaria, FalciparumG6PD Deficiency
Malaria Consortium 109
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
Testing effectiveness (Phase 2)Study completedNCT04117711
What this trial is testing

Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

Who this might be right for
Classic Galactosemia
Applied Therapeutics, Inc. 114
Not applicableUnknownNCT02761421
What this trial is testing

Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 15
Not applicableUnknownNCT02708784
What this trial is testing

An MRI Study on Muscular Diseases -Pompe Disease and Dystrophia Myotonica-

Who this might be right for
Glycogen Storage Disease Type 2Dystrophia Myotonica
University of Aarhus 60
Post-approval studies (Phase 4)Ended earlyNCT01597596
What this trial is testing

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Who this might be right for
Pompe Disease (Infantile-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenosis 2+1 more
Genzyme, a Sanofi Company 4
Not applicableUnknownNCT01482494
What this trial is testing

Detection of Pompe Disease in Adult Patients With Myopathies of Uncertain Origin or With Asymptomatic Hyper-CK-emia

Who this might be right for
Pompe Disease
Jordi Perez Lopez 50
Not applicableLooking for participantsNCT02026388
What this trial is testing

Rare Kidney Stone Consortium Biobank

Who this might be right for
Primary HyperoxaluriaDent DiseaseAPRT Deficiency+1 more
Mayo Clinic 2,000
Early research (Phase 1)Study completedNCT04073953
What this trial is testing

Primaquine Enantiomers in G6PD Deficient Human Volunteers

Who this might be right for
G6PD Deficiency
University of Mississippi, Oxford 18
Not applicableStudy completedNCT04349566
What this trial is testing

Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases

Who this might be right for
Becker Muscular DystrophyMcArdle DiseaseLimb-Girdle Muscular Dystrophy Type 2
Mads Peter Godtfeldt Stemmerik 36
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
Not applicableStudy completedNCT00663546
What this trial is testing

Malaria Studies in Cambodia

Who this might be right for
Malaria
National Institute of Allergy and Infectious Diseases (NIAID) 1,978
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