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Results for “X-Linked Genetic Diseases”

1,754 trials

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Showing 20 of 1,754 results

Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Not applicableLooking for participantsNCT06375239
What this trial is testing

Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration

Who this might be right for
Retinitis PigmentosaChoroideremiaStargardt Macular Dystrophy+4 more
Ray Therapeutics, Inc. 120
Not applicableWithdrawnNCT02429453
What this trial is testing

FFP Versus PCC in Intracranial Hemorrhage

Who this might be right for
Intracranial Hemorrhage, TraumaticIntracranial Hemorrhage, Spontaneous
University of Utah
Early research (Phase 1)Study completedNCT00092937
What this trial is testing

Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant

Who this might be right for
Granulomatous DiseaseChronic
National Institute of Allergy and Infectious Diseases (NIAID) 2
Large-scale testing (Phase 3)WithdrawnNCT00935753
What this trial is testing

Trial of Kuvan in Lesch-Nyhan Disease

Who this might be right for
Behavioral Manifestations of Lesch-Nyhan Disease
University of California, San Diego
Not applicableStudy completedNCT03002298
What this trial is testing

Virtual Reality in Individuals With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
University of Sao Paulo 64
Large-scale testing (Phase 3)Active Not RecruitingNCT03614234
What this trial is testing

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 29
Not applicableActive Not RecruitingNCT04946409
What this trial is testing

Burden of Disease and Functional Impairment in XLH

Who this might be right for
X Linked Hypophosphatemia
Wuerzburg University Hospital 60
Not applicableStudy completedNCT01645098
What this trial is testing

Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Nationwide Children's Hospital 53
Not applicableLooking for participantsNCT06147414
What this trial is testing

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Who this might be right for
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis+12 more
Assistance Publique - Hôpitaux de Paris 550
Post-approval studies (Phase 4)Study completedNCT00638001
What this trial is testing

Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy

Who this might be right for
Haemophilia AHaemophilia B
Cliniques universitaires Saint-Luc- Université Catholique de Louvain 70
Not applicableStudy completedNCT00244114
What this trial is testing

Study Evaluating Allergic Reactions To Benefix In Hemophilia B Patients

Who this might be right for
Hemophilia B
Wyeth is now a wholly owned subsidiary of Pfizer 20
Large-scale testing (Phase 3)Active Not RecruitingNCT02616484
What this trial is testing

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Saol Therapeutics Inc 34
Testing effectiveness (Phase 2)Study completedNCT00084084
What this trial is testing

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Not applicableUnknownNCT02061033
What this trial is testing

Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease

Who this might be right for
Hemophilia AHemophilia BVon Willebrand's Disease
Karolinska University Hospital 180
Early research (Phase 1)Study completedNCT01562587
What this trial is testing

Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State

Who this might be right for
Congenital Bleeding DisorderHaemophilia AHaemophilia B
Novo Nordisk A/S 18
Early research (Phase 1)UnknownNCT00473187
What this trial is testing

Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets

Who this might be right for
Hypophosphatemic RicketsGrowth DisordersSomatropin Therapy
University of Rostock
Not applicableStudy completedNCT03161171
What this trial is testing

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type III+2 more
University Hospital Heidelberg 34
Not applicableActive Not RecruitingNCT07096206
What this trial is testing

Characteristics and Impacts of X-linked Hypohidrotic Ectodermal Dysplasia (XLHED) in Boys: An Observational International Study

Who this might be right for
X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)
Pierre Fabre Medicament 27
Not applicableUnknownNCT00427180
What this trial is testing

IRIS PILOT - Extended Pilot Study With a Retinal Implant System

Who this might be right for
Retinitis PigmentosaCone-Rod DystrophyChoroideremia
Intelligent Medical Implants GmbH 20
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