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Results for “Inherited Metabolic Disease”

5,130 trials

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Showing 20 of 5,130 results

Early research (Phase 1)Looking for participantsNCT06298071
What this trial is testing

A Phase Ib Clinical Trial of Peguricase for Injection With Methotrexate in Patients With Uncontrolled Gout.

Who this might be right for
Gout
Shanghai Institute Of Biological Products 36
Not applicableStudy completedNCT01632111
What this trial is testing

Pulmonary Involvement in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University of Zurich 110
Not applicableStudy completedNCT00966407
What this trial is testing

Assessing Inherited Markers of Metabolic Syndrome in the Young

Who this might be right for
Metabolic SyndromeDiabetes Mellitus, Type 2Obesity
Eric Hoffman 700
Large-scale testing (Phase 3)Enrolling By InvitationNCT07102511
What this trial is testing

Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH

Who this might be right for
Familial Hypercholesterolemia - Heterozygous
LIB Therapeutics LLC 150
Not applicableLooking for participantsNCT04982744
What this trial is testing

Registry of Li Fraumeni and Li Fraumeni Like Syndromes

Who this might be right for
Li-Fraumeni SyndromeLi-Fraumeni-Like Syndrome
Istituto Ortopedico Rizzoli 200
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
Not applicableStudy completedNCT01416467
What this trial is testing

Characterization of the Patient Population With Galactosialidosis

Who this might be right for
Galactosialidosis
St. Jude Children's Research Hospital 3
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
Large-scale testing (Phase 3)Study completedNCT06431113
What this trial is testing

Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma

Who this might be right for
GlaucomaNeuroprotection
Fondazione IRCCS Policlinico San Matteo di Pavia 40
Very early researchLooking for participantsNCT06458010
What this trial is testing

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Who this might be right for
Familial Hypercholesterolemia
RenJi Hospital 20
Post-approval studies (Phase 4)Study completedNCT00097890
What this trial is testing

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableLooking for participantsNCT05007990
What this trial is testing

Caregiving Networks Across Disease Context and the Life Course

Who this might be right for
Inherited Metabolic DisordersUndiagnosed DiseasesBatten's Disease+2 more
National Human Genome Research Institute (NHGRI) 2,800
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
Not applicableStudy completedNCT05024110
What this trial is testing

Effect of Exercise Intensity on Vitamin D

Who this might be right for
Down SyndromeVitamin D Deficiency
Taif University 44
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 applicableLooking for participantsNCT07649031
What this trial is testing

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

Who this might be right for
Fanconi Anemia
University of Minnesota 80
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Testing effectiveness (Phase 2)Study completedNCT00000766
What this trial is testing

CMV Retinitis Retreatment Trial

Who this might be right for
Cytomegalovirus RetinitisHIV Infections
National Institute of Allergy and Infectious Diseases (NIAID) 300
Not applicableStudy completedNCT07090629
What this trial is testing

The Benefits of the Measureof Epicardial Adipose Tissue During Coronary Calcium Assessment of the Refine Cardiovascular Risk Assessment at Reunion Island.

Who this might be right for
High CholesterolLipodystrophy, Familial PartialDiabetes
Centre Hospitalier Universitaire de la Réunion 126
Not applicableStudy completedNCT02483702
What this trial is testing

Irradiated Blood Versus Non Irradiated Blood Transfusions in Craniosynostosis Repair

Who this might be right for
Graft Versus Host DiseaseHyperkalemiaCraniectomy+3 more
Valley Anesthesiology Consultants 20
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