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Results for “Tetrahydrobiopterin Deficiency”

14 trials

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Showing 14 of 14 results

Testing effectiveness (Phase 2)Study completedNCT03519711
What this trial is testing

PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

Who this might be right for
BH4 DeficiencyHyperphenylalaninemia
PTC Therapeutics 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 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
Testing effectiveness (Phase 2)Study completedNCT01425528
What this trial is testing

Study of Kuvan Treatment in Adults With GTPCH Deficiency

Who this might be right for
GTP Cyclohydrolase Deficiency
University of Utah 12
Not applicableStudy completedNCT03864029
What this trial is testing

Retrospective Observational Safety Effectiveness With Kuvan in hpA

Who this might be right for
Tetrahydrobiopterin Deficiency
BioMarin Pharmaceutical 26
Not applicableApproved For MarketingNCT00484991
What this trial is testing

Sapropterin Expanded Access Program

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical
Post-approval studies (Phase 4)UnknownNCT04227080
What this trial is testing

BH4 Responsiveness in PAH Deficiency PKU Patients

Who this might be right for
Pku PhenylketonuriaPAH Deficiency
Taipei Veterans General Hospital, Taiwan 40
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
Testing effectiveness (Phase 2)Study completedNCT00532844
What this trial is testing

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Who this might be right for
Endothelial Dysfunction
BioMarin Pharmaceutical 52
Large-scale testing (Phase 3)Ended earlyNCT00432822
What this trial is testing

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety

Who this might be right for
Phenylalanine Hydroxylase Deficiencies
Orphanetics Pharma Entwicklungs GmbH 50
Early research (Phase 1)WithdrawnNCT04014712
What this trial is testing

O2 Transport and Utilization in Health and Lung Disease

Who this might be right for
COPDTetrahydrobiopterin DeficiencyOxidative Stress
University of Massachusetts, Amherst
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT04375592
What this trial is testing

Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria

Who this might be right for
PhenylketonuriasHyperphenylalaninaemiaTetrahydrobiopterin Deficiency
metaX Institut fuer Diatetik GmbH 10
Not applicableLooking for participantsNCT04896281
What this trial is testing

Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU

Who this might be right for
Non-Phenylketonuric Hyperphenylalaninemia
Chang Gung Memorial Hospital 100