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Results for “Amino Acid Deficiency”

242 trials

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Enrolling by Invitation

Showing 20 of 242 results

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
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
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04903288
What this trial is testing

SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

Who this might be right for
AADC Deficiency
PTC Therapeutics 13
Not applicableStudy completedNCT01647529
What this trial is testing

Topical Administration of 0.15% Ganciclovir Gel for CMV Anterior Uveitis / Endotheliitis

Who this might be right for
Cytomegalovirus Anterior Segment InfectionAnterior UveitisEndotheliitis
Singapore National Eye Centre 29
Testing effectiveness (Phase 2)WithdrawnNCT03767270
What this trial is testing

Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Translate Bio, Inc.
Large-scale testing (Phase 3)Study completedNCT00006145
What this trial is testing

Preventing Cytomegalovirus (CMV) Organ Damage With Valganciclovir in People With HIV

Who this might be right for
Cytomegalovirus InfectionsHIV Infections
National Institute of Allergy and Infectious Diseases (NIAID) 350
Large-scale testing (Phase 3)Study completedNCT06582524
What this trial is testing

Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency

Who this might be right for
Arginase 1 Deficiency
Immedica Pharma AB 3
Not applicableEnrolling By InvitationNCT06330324
What this trial is testing

Reproductive Options in Inherited Skin Diseases

Who this might be right for
IchthyosisPalmoplantar KeratosesEpidermolysis Bullosa+7 more
Maastricht University Medical Center 650
Not applicableEnrolling By InvitationNCT03655223
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 completedNCT00000995
What this trial is testing

AZT Plus Ganciclovir in Patients With AIDS and Cytomegalovirus (CMV) Infection

Who this might be right for
Cytomegalovirus InfectionsHIV Infections
National Institute of Allergy and Infectious Diseases (NIAID) 60
Not applicableStudy completedNCT00000989
What this trial is testing

The Safety and Effectiveness of Ganciclovir Used Alone or in Combination With Granulocyte-Macrophage Colony Stimulating Factor in the Treatment of Cytomegalovirus (CMV) of the Eye in Patients With AIDS

Who this might be right for
Cytomegalovirus RetinitisHIV Infections
National Institute of Allergy and Infectious Diseases (NIAID) 50
Testing effectiveness (Phase 2)Ended earlyNCT03952156
What this trial is testing

Gene Therapy Clinical Study in Adult PKU

Who this might be right for
PhenylketonuriasPAH Deficiency
Homology Medicines, Inc 10
Testing effectiveness (Phase 2)Study completedNCT03269045
What this trial is testing

Study of ORL-1B in Patients With Biotinidase Deficiency

Who this might be right for
Biotinidase Deficiency
Orpha Labs 20
Large-scale testing (Phase 3)UnknownNCT01824030
What this trial is testing

FFR or OCT Guidance to RevasculariZe Intermediate Coronary Stenosis Using Angioplasty

Who this might be right for
Ischemic Heart Disease
Catholic University of the Sacred Heart 400
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