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Results for “Fah Deficiency”

26 trials

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Enrolling by Invitation

Showing 20 of 26 results

Not applicableEnrolling By InvitationNCT04437771
What this trial is testing

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Who this might be right for
Fanconi Anemia Complementation Group AFanconi Anemia
Rocket Pharmaceuticals Inc. 9
Not applicableStudy completedNCT04196959
What this trial is testing

Evaluation of TYR Sphere

Who this might be right for
TyrosinemiasAlkaptonuriaTyrosinemia, Type I+2 more
Vitaflo International, Ltd 18
Not applicableWithdrawnNCT06227429
What this trial is testing

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum
Large-scale testing (Phase 3)Study completedNCT02323529
What this trial is testing

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum 18
Not applicableUnknownNCT03351868
What this trial is testing

FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector

Who this might be right for
Fanconi Anemia
Shenzhen Geno-Immune Medical Institute 10
Not applicableStudy completedNCT00004443
What this trial is testing

Study of NTBC for Tyrosinemia I

Who this might be right for
Tyrosinemia I
University of Washington 20
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
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 applicableUnknownNCT04113772
What this trial is testing

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine

Who this might be right for
Hereditary Tyrosinemia, Type I
Sutphin Drugs 4
Not applicableActive Not RecruitingNCT05051657
What this trial is testing

Evaluation of the Express Plus Range

Who this might be right for
PhenylketonuriasPKUHomocystinuria+3 more
Vitaflo International, Ltd 28
Not applicableStudy completedNCT05062226
What this trial is testing

GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR

Who this might be right for
PKUTyrosinemias
Nutricia UK Ltd 45
Early research (Phase 1)Study completedNCT01734889
What this trial is testing

Taste and Palatability of Orfadin Suspension

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum 18
Not applicableStudy completedNCT04761588
What this trial is testing

Evaluation of TYR Sphere in France

Who this might be right for
TyrosinemiaAKU
Vitaflo International, Ltd 14
Early research (Phase 1)Ended earlyNCT01331018
What this trial is testing

Gene Therapy for Fanconi Anemia

Who this might be right for
Fanconi Anemia
Fred Hutchinson Cancer Center 3
Not applicableStudy completedNCT02320084
What this trial is testing

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum 315
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
Early research (Phase 1)Study completedNCT02750332
What this trial is testing

Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)

Who this might be right for
Hereditary Tyrosinemia, Type I
Cycle Pharmaceuticals Ltd. 20
Not applicableUnknownNCT03446586
What this trial is testing

Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)

Who this might be right for
Hereditary Tyrosinemia, Type I
Yassin Abdelghaffar Charity Center for Liver Disease and Research 50
Not applicableLooking for participantsNCT06941532
What this trial is testing

GMP Powdered Substitutes in PKU and TYR

Who this might be right for
PhenylketonuriaTyrosinemia
Nutricia UK Ltd 40
Testing effectiveness (Phase 2)Study completedNCT00004333
What this trial is testing

Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Who this might be right for
Tyrosinemia, Type I
National Center for Research Resources (NCRR)
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