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Results for “PAH Deficiency”

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Showing 20 of 0 results

Not applicableUnknownNCT05679544
What this trial is testing

Biological Signatures Resulting From Occupational Exposure to Complex Mixtures of PAHs

Who this might be right for
Metabolic DisturbanceProtein DeficiencyGenotoxicity
University Hospital, Grenoble 100
Testing effectiveness (Phase 2)Study completedNCT00355264
What this trial is testing

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

Who this might be right for
Tetrahydrobiopterin DeficienciesHyperphenylalaninemia, Non-Phenylketonuric
BioMarin Pharmaceutical 12
Not applicableApproved For MarketingNCT00484991
What this trial is testing

Sapropterin Expanded Access Program

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical
Testing effectiveness (Phase 2)Active Not RecruitingNCT04480567
What this trial is testing

AAV Gene Therapy Study for Subjects with PKU

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 100
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Post-approval studies (Phase 4)Active Not RecruitingNCT06901323
What this trial is testing

Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria

Who this might be right for
Phenylketonuria
Mansoura University 176
Early research (Phase 1)Active Not RecruitingNCT07255599
What this trial is testing

Evaluation of BH4 Responsiveness in Our PKU Patients

Who this might be right for
Phenylketonuria (PKU)
Sohag University 20
Not applicableLooking for participantsNCT07484945
What this trial is testing

Multiomics Approach in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
University Hospital, Tours 149
Not applicableLooking for participantsNCT07825883
What this trial is testing

The Impact of Frequency of Home Phenylalanine Measurements on Metabolic Control in a Population of Patients With Classic Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Michał Kania 36
Large-scale testing (Phase 3)Study completedNCT01114737
What this trial is testing

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 206
Not applicableStudy completedNCT01016392
What this trial is testing

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

Who this might be right for
Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
BioMarin Pharmaceutical 627
Not applicableStudy completedNCT04086511
What this trial is testing

PANDA: PKU Amino Acid Evaluation

Who this might be right for
Phenylketonuria (PKU)
Nutricia Research 37
Not applicableStudy completedNCT03858101
What this trial is testing

SNAP: Study Nutrients in Adult PKU

Who this might be right for
Phenylketonuria
Nutricia Research 71
Not applicableLooking for participantsNCT04404530
What this trial is testing

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

Who this might be right for
Phenylketonurias
Emory University 45
Not applicableStudy completedNCT04318509
What this trial is testing

Market Research - Acceptability Study for a New PKU Protein Substitute

Who this might be right for
PKUPhenylketonuriasInborn Errors of Metabolism+1 more
Dr. Schär AG / SPA 8
Testing effectiveness (Phase 2)Study completedNCT00325962
What this trial is testing

The Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

Who this might be right for
Hypertension
BioMarin Pharmaceutical 84
Not applicableEnded earlyNCT01541397
What this trial is testing

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

Who this might be right for
HyperphenylalaninemiaPhenylketonuria
The University of Texas Health Science Center, Houston 6
Not applicableStudy completedNCT03820804
What this trial is testing

Nutritional Status in Phenylketonuria

Who this might be right for
Phenylketonurias
Universidade do Porto 94
Large-scale testing (Phase 3)Study completedNCT00104247
What this trial is testing

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Who this might be right for
Phenylketonurias
BioMarin Pharmaceutical 89
Not applicableEnded earlyNCT05827536
What this trial is testing

Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.

Who this might be right for
Phenylketonurias
APR Applied Pharma Research s.a. 21
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