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Results for “X-LINKED”

1,824 trials

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Showing 20 of 1,824 results

Not applicableEnded earlyNCT01357408
What this trial is testing

Heart Failure and Risk of Re-Admissions Determined by Abnormal REVEAL Parameters

Who this might be right for
Heart Failure
Thomas Jefferson University 40
Not applicableStudy completedNCT06054971
What this trial is testing

NSAA NON-Interventional Study Protocol

Who this might be right for
Duchenne Muscular Dystrophy
Red Nucleus Enterprise Solutions, LLC 31
Testing effectiveness (Phase 2)Study completedNCT01462292
What this trial is testing

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

Who this might be right for
Muscular Dystrophies
GlaxoSmithKline 51
Not applicableStudy completedNCT02168114
What this trial is testing

Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle

Who this might be right for
Duchenne Muscular Dystrophy
University of Florida 17
Early research (Phase 1)Study completedNCT02262338
What this trial is testing

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
ArmaGen, Inc 6
Large-scale testing (Phase 3)Study completedNCT01963143
What this trial is testing

Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Gammaplex® 10 and Gammaplex® 5% in Primary Immunodeficiency Diseases

Who this might be right for
Primary Immune Deficiency DisordersCommon Variable ImmunodeficiencyX-linked Agammaglobulinaemia+1 more
Bio Products Laboratory 48
Not applicableWithdrawnNCT06634836
What this trial is testing

Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B

Who this might be right for
Hemophilia AHemophilia B
Pfizer
Not applicableStudy completedNCT00728364
What this trial is testing

Anderson-Fabry Disease in Chronic Kidney Disease Patients Not on Renal Replacement Therapy

Who this might be right for
Focus of Study: Prevalence of Fabry Disease in CKD Population
Klinikum Wels-Grieskirchen 4,353
Not applicableStudy completedNCT00207532
What this trial is testing

Dosage Effects of Folic Acid on Blood Folates of Honduran Women

Who this might be right for
Spina Bifida and Anencephaly
Centers for Disease Control and Prevention 100
Not applicableStudy completedNCT03292887
What this trial is testing

Hunter Outcome Survey (HOS)

Who this might be right for
Hunter Syndrome
Shire 1,443
Not applicableStudy completedNCT01959555
What this trial is testing

Project to Update the Study of Congenital Haemophilia in Spain

Who this might be right for
Congenital Bleeding DisorderHaemophilia AHaemophilia B
Novo Nordisk A/S 1,157
Not applicableStudy completedNCT01625663
What this trial is testing

Heart and Muscle Metabolism in Barth Syndrome

Who this might be right for
Barth Syndrome
Duke University 64
Testing effectiveness (Phase 2)Study completedNCT01725152
What this trial is testing

Ganaxolone Treatment in Children With Fragile X Syndrome

Who this might be right for
Fragile x Syndrome
Marinus Pharmaceuticals 59
Testing effectiveness (Phase 2)Study completedNCT02952534
What this trial is testing

Rucaparib in Patients With Metastatic Castration-resistant Prostate Cancer and Homologous Recombination Gene Deficiency

Who this might be right for
Metastatic Castration Resistant Prostate Cancer
pharmaand GmbH 277
Testing effectiveness (Phase 2)Study completedNCT06304064
What this trial is testing

Halt cardiomyOPathy progrEssion in Duchenne (HOPE-OLE)

Who this might be right for
Duchenne Muscular Dystrophy
Capricor Inc. 8
Post-approval studies (Phase 4)Study completedNCT00001317
What this trial is testing

A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood

Who this might be right for
Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID) 100
Large-scale testing (Phase 3)Study completedNCT03566017
What this trial is testing

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 97
Testing effectiveness (Phase 2)Study completedNCT04188964
What this trial is testing

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

Who this might be right for
X-linked Hypophosphatemia (XLH)
Kyowa Kirin Pharmaceutical Development Ltd 16
Post-approval studies (Phase 4)Ended earlyNCT04708314
What this trial is testing

An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular Dystrophy
Rare Disease Research, LLC 2
Large-scale testing (Phase 3)Looking for participantsNCT07236606
What this trial is testing

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Who this might be right for
MPS IIHunter Syndrome (MPS II)
REGENXBIO Inc. 2
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