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Results for “Fah Deficiency”

26 trials

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Showing 20 of 26 results

Testing effectiveness (Phase 2)Study completedNCT02931071
What this trial is testing

Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Who this might be right for
Fanconi Anemia
Hospital Universitari Vall d'Hebron Research Institute 13
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 applicableStudy completedNCT04761588
What this trial is testing

Evaluation of TYR Sphere in France

Who this might be right for
TyrosinemiaAKU
Vitaflo International, Ltd 14
Testing effectiveness (Phase 2)Study completedNCT03377556
What this trial is testing

Lung-MAP: Talazoparib in Treating Patients With HRRD Positive Recurrent Stage IV Squamous Cell Lung Cancer

Who this might be right for
ATM Gene MutationATR Gene MutationBARD1 Gene Mutation+18 more
SWOG Cancer Research Network 51
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
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
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
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
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 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
Early research (Phase 1)Study completedNCT02750709
What this trial is testing

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

Who this might be right for
Hereditary Tyrosinemia, Type I
Cycle Pharmaceuticals Ltd. 24
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
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 applicableActive Not RecruitingNCT04437771
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 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 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
Early research (Phase 1)Study completedNCT02750345
What this trial is testing

Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)

Who this might be right for
Hereditary Tyrosinemia, Type I
Cycle Pharmaceuticals Ltd. 24
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 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 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
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