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Results for “Inborn Errors of Metabolism Disorders”

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Showing 20 of 0 results

Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
Early research (Phase 1)Study completedNCT01554800
What this trial is testing

Effect of ACP-501 on Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Subjects With Coronary Artery Disease

Who this might be right for
Coronary Artery DiseaseAtherosclerosis
MedImmune LLC 16
Large-scale testing (Phase 3)Study completedNCT04031742
What this trial is testing

Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia

Who this might be right for
Homozygous Familial Hypercholesterolemia
Innovent Biologics (Suzhou) Co. Ltd. 18
Not applicableUnknownNCT05719337
What this trial is testing

Multi-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy Adults

Who this might be right for
Left Ventricular Hypertrophy
First Hospital of China Medical University 660
Post-approval studies (Phase 4)Not Yet RecruitingNCT07824895
What this trial is testing

Mazdutide for Weight Management and Gout in Adults With Obesity

Who this might be right for
GoutObesity (BMI>30)Hyperuricemia+1 more
Hongwei Jiang 30
Large-scale testing (Phase 3)Study completedNCT02174900
What this trial is testing

Safety of Primaquine + Artemether-lumefantrine in G6PD Deficient Males With an Asymptomatic Malaria Infection (SAFEPRIM)

Who this might be right for
Malaria
London School of Hygiene and Tropical Medicine 70
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
Large-scale testing (Phase 3)Study completedNCT05529992
What this trial is testing

Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 20
Not applicableStudy completedNCT02069236
What this trial is testing

Comparing G6PD Tests Using Capillary Blood Versus Venous Blood

Who this might be right for
Glucose-6 Phosphate Dehydrogenase Deficiency
PATH 150
Not applicableUnknownNCT02114554
What this trial is testing

Mitochondrial nt3243 A>G Mutation in Taiwan

Who this might be right for
MELAS SyndromeNoninsulin-dependent Diabetes Mellitus With Deafness
National Taiwan University Hospital 40
Testing effectiveness (Phase 2)Active Not RecruitingNCT07187375
What this trial is testing

Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old

Who this might be right for
Congenital Adrenal Hyperplasia
Neurocrine Switzerland GmbH 7
Not applicableWithdrawnNCT00018694
What this trial is testing

Cholestanol in Humans

Who this might be right for
Cerebrotendinous Xanthomatosis
US Department of Veterans Affairs
Testing effectiveness (Phase 2)Study completedNCT02767531
What this trial is testing

Orlistat for the Treatment of Type I Hyperlipoproteinemia

Who this might be right for
Hyperlipoproteinemia Type IHypertriglyceridemia
University of Texas Southwestern Medical Center 2
Not applicableStudy completedNCT00002257
What this trial is testing

Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS

Who this might be right for
Cytomegalovirus RetinitisHIV Infections
Hoffmann-La Roche 150
Not applicableActive Not RecruitingNCT04101149
What this trial is testing

Genetic Causes of Familial Hypercholesterolemia

Who this might be right for
Familial Hypercholesterolemia
Region Örebro County 150
Testing effectiveness (Phase 2)Study completedNCT03395704
What this trial is testing

LJPC-401 for the Treatment of Iron Overload in Adult Patients With Hereditary Hemochromatosis

Who this might be right for
Hereditary Hemochromatosis
La Jolla Pharmaceutical Company 70
Testing effectiveness (Phase 2)UnknownNCT06270225
What this trial is testing

SSGJ-613 in Gout Subjects Initiating Urate-Lowering Treatment.

Who this might be right for
Gout Arthritis
Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. 157
Testing effectiveness (Phase 2)Study completedNCT00104260
What this trial is testing

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

Who this might be right for
Phenylketonurias
BioMarin Pharmaceutical 700
Testing effectiveness (Phase 2)Study completedNCT03406962
What this trial is testing

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 8
Early research (Phase 1)Study completedNCT03211403
What this trial is testing

Multiple Doses Study of SHR4640 in Male Subjects With High Serum Uric Acid Level

Who this might be right for
GoutHyperuricemia
Atridia Pty Ltd. 24
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