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Results for “Enzyme Deficiency”

73 trials

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Showing 20 of 73 results

Post-approval studies (Phase 4)Ended earlyNCT00230607
What this trial is testing

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Who this might be right for
Fabry DiseaseAlpha Galactosidase A Deficiency
Genzyme, a Sanofi Company 7
Large-scale testing (Phase 3)Looking for participantsNCT07555483
What this trial is testing

A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)

Who this might be right for
Alpha 1 Antitrypsin Deficiency
Grifols Therapeutics LLC 40
Not applicableActive Not RecruitingNCT04098211
What this trial is testing

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

Who this might be right for
Neuronal Ceroid-LipofuscinosesNeuronal Ceroid Lipofuscinosis CLN2Spinocerebellar Ataxia, Autosomal Recessive 7
Children's Hospital of Orange County 5
Not applicableStudy completedNCT04403932
What this trial is testing

Increased Risk of Severe Coronavirus Disease 2019 in Patients With Vitamin D Deficiency

Who this might be right for
Coronavirus Disease 2019 (COVID-19)
Hospital San Carlos, Madrid 300
Large-scale testing (Phase 3)Study completedNCT01757184
What this trial is testing

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 66
Early research (Phase 1)Study completedNCT01289756
What this trial is testing

Influence of Pharmacogenetic Factors, Paroxetine and Clarithromycin on Pharmacokinetics of Clomiphene

Who this might be right for
AnovulationDisorder Due Cytochrome P450 CYP2D6 VariantCytochrome P450 CYP3A Enzyme Deficiency
Robert Bosch Gesellschaft für Medizinische Forschung mbH (RBMF) 20
Testing effectiveness (Phase 2)Looking for participantsNCT06887777
What this trial is testing

Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI)

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency Disease
Assistance Publique - Hôpitaux de Paris 15
Not applicableStudy completedNCT02276014
What this trial is testing

Effect of SNPs in the BCMO1 Enzyme

Who this might be right for
Beta-carotene BioavailabilityVitamin A Deficiency
Newcastle-upon-Tyne Hospitals NHS Trust 85
Not applicableStudy completedNCT03188601
What this trial is testing

Α1-antitrypsin (AAT) Levels and Functions in Allogeneic Bone Marrow Transplantation and Throughout Progression Into GVHD

Who this might be right for
GVHDBone Marrow Transplant FailureAlpha 1-Antitrypsin
Rambam Health Care Campus 27
Testing effectiveness (Phase 2)UnknownNCT04556071
What this trial is testing

Efficacy and Safety of Niraparib Combined With Bevacizumab in Platinum Refractory/Resistant Recurrent Ovarian Cancer

Who this might be right for
Ovarian NeoplasmsFallopian Tube NeoplasmsEndocrine Gland Neoplasms+21 more
Xiaoxiang Chen 32
Not applicableStudy completedNCT02656095
What this trial is testing

Lipoprotein Lipase Enzyme Activity Assay Validation and Clinical Assessment

Who this might be right for
Lipoprotein Lipase Deficiency
University of Colorado, Denver 12
Testing effectiveness (Phase 2)Study completedNCT00786422
What this trial is testing

Deep Vein Thrombosis Treatment With the Oral Direct Factor Xa Inhibitor Rivaroxaban in Patients Using a Strong CYP 3A4 Inducer

Who this might be right for
Venous ThrombosisDeep Vein Thrombosis
Bayer 25
Testing effectiveness (Phase 2)Study completedNCT02112994
What this trial is testing

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 31
Not applicableStudy completedNCT01884220
What this trial is testing

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Who this might be right for
Wolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2
Children's Hospital Medical Center, Cincinnati 4
Not applicableLooking for participantsNCT01665326
What this trial is testing

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Who this might be right for
Pompe Disease
Duke University 400
Early research (Phase 1)Study completedNCT01881984
What this trial is testing

Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

Who this might be right for
Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
University of Pittsburgh 4
Not applicableStudy completedNCT02191358
What this trial is testing

YouScript IMPACT Registry

Who this might be right for
Adverse Drug EventsAdverse Drug ReactionsDrug Interaction Potentiation+10 more
Genelex Corporation 800
Testing effectiveness (Phase 2)Study completedNCT07148804
What this trial is testing

A Double-Blind, Placebo-Controlled Study of Fermented Deglycyrrhizinated Licorice Extract

Who this might be right for
Diabetic Polyneuropathy
Helwan University 83
Testing effectiveness (Phase 2)Study completedNCT00947960
What this trial is testing

Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Who this might be right for
Adult Polyglucosan Body DiseaseGlycogen Brancher Enzyme DeficiencyGlycogen Storage Disease Type IV
Baylor Research Institute 23
Early research (Phase 1)Looking for participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
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