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Results for “Amino Acid Metabolism, Inborn Errors”

502 trials

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Showing 20 of 502 results

Testing effectiveness (Phase 2)Study completedNCT00237185
What this trial is testing

The Efficacy and Safety of Imatinib Mesylate in Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumors Expressing C-kit Gene

Who this might be right for
Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
Novartis Pharmaceuticals 148
Testing effectiveness (Phase 2)Ended earlyNCT00467831
What this trial is testing

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Who this might be right for
Hermansky-Pudlak Syndrome (HPS)Pulmonary FibrosisOculocutaneous Albinism+2 more
National Human Genome Research Institute (NHGRI) 3
Not applicableApproved For MarketingNCT00484991
What this trial is testing

Sapropterin Expanded Access Program

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical
Not applicableStudy completedNCT02368340
What this trial is testing

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

Who this might be right for
Hermansky Pudlak Syndrome
Vanderbilt University 55
Testing effectiveness (Phase 2)Study completedNCT03378531
What this trial is testing

AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

Who this might be right for
Arginase I DeficiencyHyperargininemia
Aeglea Biotherapeutics 14
Large-scale testing (Phase 3)Study completedNCT02426775
What this trial is testing

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

Who this might be right for
Propionic AcidemiaMethylmalonic Acidemia
King Abdullah International Medical Research Center 33
Not applicableUnknownNCT00002301
What this trial is testing

Foscarnet in the Treatment of Cytomegalovirus (CMV) of the Eyes in Patients With AIDS Who Have Not Had Success With Ganciclovir

Who this might be right for
Cytomegalovirus RetinitisHIV Infections
Astra USA
Not applicableStudy completedNCT00744588
What this trial is testing

Stress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in Alcoholism

Who this might be right for
Alcohol Dependence
University of Texas Southwestern Medical Center 75
Not applicableStudy completedNCT01917344
What this trial is testing

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

Who this might be right for
Phenylketonuria
Boston Children's Hospital 10
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
Testing effectiveness (Phase 2)Study completedNCT00483314
What this trial is testing

Homocystinuria: Treatment With N-Acetylcysteine

Who this might be right for
Homocystinuria
McGill University Health Centre/Research Institute of the McGill University Health Centre 5
Early research (Phase 1)Not Yet RecruitingNCT07643844
What this trial is testing

AAVrh10-PCCA Gene Therapy for Propionic Acidemia

Who this might be right for
Propionic Acidemia
Mayo Clinic 9
Not applicableWithdrawnNCT01650909
What this trial is testing

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

Who this might be right for
Classical Phenylketonuria(PKU)
Stanford University
Testing effectiveness (Phase 2)Study completedNCT00001034
What this trial is testing

The Safety and Effectiveness of Ganciclovir in the Prevention of Cytomegalovirus (CMV) of the Eyes and Disease of the Stomach and Intestines in Patients With HIV

Who this might be right for
Cytomegalovirus RetinitisHIV InfectionsGastrointestinal Diseases
National Institute of Allergy and Infectious Diseases (NIAID) 850
Testing effectiveness (Phase 2)WithdrawnNCT01791192
What this trial is testing

Study of FTY720 in Patients With Uveitis

Who this might be right for
Acute Noninfectious Posterior, Intermediate, or Pan Uveitis
Novartis Pharmaceuticals
Post-approval studies (Phase 4)Study completedNCT02246218
What this trial is testing

The Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorder
Amgen 27
Not applicableLooking for participantsNCT00237315
What this trial is testing

Longitudinal Study of Urea Cycle Disorders

Who this might be right for
Brain Diseases, Metabolic, InbornAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders
Andrea Gropman 1,500
Not applicableStudy completedNCT05706714
What this trial is testing

Th1, Th2, Th17 Phenotype in Urea Cycle Disorders

Who this might be right for
Urea Cycle DisorderLysinuric Protein Intolerance
Istanbul University - Cerrahpasa 65
Testing effectiveness (Phase 2)Study completedNCT05781399
What this trial is testing

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

Who this might be right for
Phenylketonuria
Otsuka Pharmaceutical Development & Commercialization, Inc. 111
Large-scale testing (Phase 3)Study completedNCT01819727
What this trial is testing

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 261
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