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Results for “Inborn Errors of Metabolism Disorders”

3,277 trials

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Showing 20 of 3,277 results

Testing effectiveness (Phase 2)Study completedNCT01603446
What this trial is testing

L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome

Who this might be right for
MELAS Syndrome
The Hospital for Sick Children 7
Not applicableLooking for participantsNCT07648303
What this trial is testing

Prevalence of Cardiac Thrombi in Cardiac Amyloidosis

Who this might be right for
Cardiac Amyloidosis
Centre Hospitalier Universitaire Dijon 200
Not applicableStudy completedNCT01194141
What this trial is testing

Exercise Training in Barth Syndrome

Who this might be right for
Barth Syndrome
Washington University School of Medicine 4
Not applicableUnknownNCT04808856
What this trial is testing

Treatment of Refractory Gout With ACTH or Methylprednisolone

Who this might be right for
Gout
Shanghai 10th People's Hospital 60
Early research (Phase 1)Study completedNCT01380431
What this trial is testing

A Comparative Bioavailability Study of Lisinopril Tablets, 40 mg - Effect of Food Study

Who this might be right for
To Determine Bioequivalence Under Fed Conditions
Par Pharmaceutical, Inc. 18
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07773246
What this trial is testing

Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD

Who this might be right for
Urea Cycle Disorders, InbornUrea Cycle DisordersOrnithine Transcarbamylase (OTC) Deficiency+8 more
Korro Bio, Inc. 30
Testing effectiveness (Phase 2)Enrolling By InvitationNCT07393373
What this trial is testing

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Who this might be right for
Hypochondroplasia
QED Therapeutics, a BridgeBio company 135
Testing effectiveness (Phase 2)Ended earlyNCT05116046
What this trial is testing

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Who this might be right for
Achondroplasia
Pfizer 35
Testing effectiveness (Phase 2)Looking for participantsNCT06518005
What this trial is testing

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

Who this might be right for
Crigler-Najjar Syndrome
Genethon 3
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableUnknownNCT01448577
What this trial is testing

Study to Re-assess and Re-confirm Data Previously Recorded About the Incidence and Severity of Acute Abdominal "Pancreatitis" Episodes in Lipoprotein Lipase Deficient (LPLD) Subjects Previously Enrolled on AMT Clinical Studies

Who this might be right for
Lipoprotein Lipase Deficiency
Amsterdam Molecular Therapeutics 22
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Large-scale testing (Phase 3)UnknownNCT02552251
What this trial is testing

COrticosteroid in Congenital Adrenal Hyperplasia

Who this might be right for
Congenital Adrenal Hyperplasia
University Hospital, Caen 40
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Testing effectiveness (Phase 2)Ended earlyNCT04273269
What this trial is testing

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 5
Not applicableStudy completedNCT00351156
What this trial is testing

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Amicus Therapeutics 50
Early research (Phase 1)Study completedNCT02422654
What this trial is testing

Taste Evaluation of Different Liquid Formulations With Eliglustat

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 8
Not applicableActive Not RecruitingNCT04869631
What this trial is testing

Screening for Amyloidosis Before Aortic Valve Elective Replacement

Who this might be right for
AmyloidosisAortic Valve Stenosis
Charite University, Berlin, Germany 1,000
Not applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
Testing effectiveness (Phase 2)Study completedNCT04165239
What this trial is testing

The KHENERGYZE Study

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more
Khondrion BV 27
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