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Results for “Inherited Disorders of Metabolism”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00519363
What this trial is testing

Statins and Lupus: Effects of Statins on Clinical Lupus Parameters, Serological Markers and Toll-like Receptors

Who this might be right for
Systemic Lupus Erythematosus
The Center for Rheumatic Disease, Allergy, & Immunology 15
Not applicableEnded earlyNCT02345421
What this trial is testing

Identify and Characterize LAL-D Patients in High-risk Populations

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 640
Not applicableStudy completedNCT00001721
What this trial is testing

Study of Smith-Lemli-Opitz Syndrome

Who this might be right for
Smith-Lemli-Opitz Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 130
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04240821
What this trial is testing

Theophylline for Treatment of Pseudohypoparathyroidism

Who this might be right for
PseudohypoparathyroidismPseudohypoparathyroidism Type 1aAlbright Hereditary Osteodystrophy
Jaclyn Tamaroff 34
Not applicableStudy completedNCT05181839
What this trial is testing

Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

Who this might be right for
X-Linked Hypophosphatemia
Kyowa Kirin Pharmaceutical Development Ltd 25
Not applicableEnded earlyNCT00827762
What this trial is testing

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

Who this might be right for
Phenylketonuria
Washington University School of Medicine 2
Not applicableLooking for participantsNCT07716488
What this trial is testing

Screening for Disorders of Lipid Metabolism and Familial Hyperlipidemia in Children and Adolescents With Increased Cardiovascular Risk Due to Chronic Diseases Such as Congenital Heart Disease, Chronic Inflammatory Bowel Disease, Celiac Disease and Rheumatic Disorders

Who this might be right for
Lipid Metabolism DisordersLipid Metabolism, Inborn ErrorsCongenital Heart Disease+4 more
Alexandra Miriam Kiess 600
Testing effectiveness (Phase 2)Study completedNCT01965327
What this trial is testing

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

Who this might be right for
Friedreich Ataxia
Children's Hospital of Philadelphia 12
Post-approval studies (Phase 4)Active Not RecruitingNCT07248761
What this trial is testing

Early Hydrocortisone Versus Regular Treatment in Shock in Extremely Preterm Neonates - an Open Randomized Controlled Trial

Who this might be right for
ShockPreterm Birth ComplicationPreterm Intraventricular Hemorrhage+8 more
Coordinación de Investigación en Salud, Mexico 76
Testing effectiveness (Phase 2)Study completedNCT02595983
What this trial is testing

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

Who this might be right for
Transthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR Amyloidosis+1 more
Alnylam Pharmaceuticals 12
Not applicableLooking for participantsNCT06439654
What this trial is testing

Atlantic Lipid Lowering Treatment Optimization Program

Who this might be right for
Familial HypercholesterolemiaLipoprotein Types--Lp System Lp(A) HyperlipoproteinemiaApolipoprotein B 100, Familial Defective+2 more
Atlantic Health System 250
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
Large-scale testing (Phase 3)Study completedNCT01347073
What this trial is testing

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

Who this might be right for
Urea Cycle Disorders
Amgen 23
Not applicableStudy completedNCT01565005
What this trial is testing

Microcephaly Genetic Deficiency in Neural Progenitors

Who this might be right for
Microcephaly
Assistance Publique - Hôpitaux de Paris 98
Early research (Phase 1)Study completedNCT02336594
What this trial is testing

RDEA3170 Bioavailability Study

Who this might be right for
Gout
Ardea Biosciences, Inc. 15
Not applicableLooking for participantsNCT06767605
What this trial is testing

Spanish Registry of Autoimmune and Cholestatic Liver Diseases (ColHai)

Who this might be right for
Hepatitis Autoimmune DiseasePrimary Biliary Cholangitis (PBC)Primary Sclerosing Cholangitis (PSC)+1 more
Asociación Española para el Estudio del Hígado 5,000
Not applicableLooking for participantsNCT07154394
What this trial is testing

Cardiac Amyloid Deposits and Heart Dysfunction in Alzheimer's Disease

Who this might be right for
Dementia, Alzheimer TypeMild Cognitive ImpairmentCardiac Amyloidosis
University Hospital, Essen 15
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
Early research (Phase 1)Study completedNCT01603186
What this trial is testing

Bioequivalence Study of Quetiapine Fumarate Tablets 25 mg Under Fed Condition

Who this might be right for
Fed
IPCA Laboratories Ltd. 54
Not applicableUnknownNCT00951886
What this trial is testing

The Validity of Forced Expiratory Maneuvers in Ataxia Telangiectasia Studied Longitudinally

Who this might be right for
Ataxia Telangiectasia
Sheba Medical Center 28
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