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Results for “Inherited Metabolic Disorders”

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Showing 20 of 0 results

Very early researchStudy completedNCT00965666
What this trial is testing

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

Who this might be right for
Fanconi Anemia
Children's Hospital Medical Center, Cincinnati 3
Not applicableStudy completedNCT04649866
What this trial is testing

Coronary Artery Disease in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Pittsburgh 7
Not applicableStudy completedNCT01820702
What this trial is testing

Short Term Bed Rest Study: Evaluation of the Use of Artificial Gravity, Induced by Short-arm Centrifugation

Who this might be right for
Bone Diseases, MetabolicAcid-Base ImbalanceMuscular Atrophy+4 more
DLR German Aerospace Center 10
Not applicableStudy completedNCT02468570
What this trial is testing

Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 9
Early research (Phase 1)Study completedNCT02218294
What this trial is testing

Study to Determine How BCX4161 is Metabolized and Eliminated by the Body

Who this might be right for
Hereditary Angioedema
BioCryst Pharmaceuticals 7
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
Not applicableLooking for participantsNCT04613089
What this trial is testing

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more
Universitätsklinikum Hamburg-Eppendorf 500
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableEnded earlyNCT01806051
What this trial is testing

A Pilot Study on Diurnal Variation

Who this might be right for
Phenylketonuria (PKU)
Dr. Linda Randolph 6
Not applicableLooking for participantsNCT06546137
What this trial is testing

National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry

Who this might be right for
Cardiomyopathy, HypertrophicCardiomyopathy, DilatedCardiomyopathy Restrictive+11 more
Hospital do Coracao 1,211
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
Large-scale testing (Phase 3)Study completedNCT03862807
What this trial is testing

Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant

Who this might be right for
Amyloidosis, FamilialTransthyretin Amyloidosis
Alnylam Pharmaceuticals 24
Early research (Phase 1)Study completedNCT01563432
What this trial is testing

Clinical Study to Evaluate the Pharmacokinetic Characteristics Between TMX-67 and Feburic® in Healthy Volunteers

Who this might be right for
Gout
SK Chemicals Co., Ltd. 30
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Not applicableUnknownNCT06050057
What this trial is testing

Surgical Treatment of Adrenal Diseases- Laparoscopic vs. Robotic-assisted Adrenalectomy

Who this might be right for
Adrenal TumorAdrenal DiseaseAdrenal Mass+10 more
University Hospital Olomouc 100
Not applicableEnded earlyNCT04222101
What this trial is testing

Association of Insulin Resistance and FGF21 on Cardiac Function in Pediatric Dilated Cardiomyopathy

Who this might be right for
Cardiomyopathy, DilatedCardiomyopathy, HypertrophicInsulin Resistance
Le Bonheur Children's Hospital 9
Not applicableStudy completedNCT06904612
What this trial is testing

Using Iodized Salt to Improve Serum Folate, B12 and Iron Levels

Who this might be right for
Neural Tube DefectsFolic Acid DeficiencySpina Bifida+4 more
Hydrocephalus and Neuroscience Institute 200
Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
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