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Results for “X-linked Genetic Diseases”

1,731 trials

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Showing 20 of 1,731 results

Not applicableStudy completedNCT05564962
What this trial is testing

Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)

Who this might be right for
Duchenne Muscular DystrophyGait Disorders in Children
Hacettepe University 69
Very early researchWithdrawnNCT03921515
What this trial is testing

Skin Immunity Sample Collection Involving Blisters and Biopsies

Who this might be right for
Hyper-Immunoglobulin E. Syndrome (HEIS)Chronic Granulomatous Disease (CGD)
National Institute of Allergy and Infectious Diseases (NIAID)
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
Not applicableStudy completedNCT01826422
What this trial is testing

Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients

Who this might be right for
Muscular Dystrophy, Duchenne
Coordinación de Investigación en Salud, Mexico 40
Large-scale testing (Phase 3)Study completedNCT01254019
What this trial is testing

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophies
GlaxoSmithKline 186
Large-scale testing (Phase 3)Ended earlyNCT02790034
What this trial is testing

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms

Who this might be right for
Rett Syndrome
Newron Pharmaceuticals SPA 129
Testing effectiveness (Phase 2)Study completedNCT01540604
What this trial is testing

CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers

Who this might be right for
Duchenne Muscular DystrophyBecker Muscular Dystrophy
RSPR Pharma AB
Early research (Phase 1)Study completedNCT00744692
What this trial is testing

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Who this might be right for
Non Malignant DisordersImmunodeficienciesCongenital Marrow Failures+5 more
Duke University 22
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
Early research (Phase 1)Ended earlyNCT01620801
What this trial is testing

Hemophilia B Gene Therapy With AAV8 Vector

Who this might be right for
Hemophilia B
Spark Therapeutics, Inc. 4
Not applicableStudy completedNCT03323307
What this trial is testing

Low Cost OCT for Point of Care

Who this might be right for
Optical Coherence Tomography (OCT) of the Retina
Duke University 70
Post-approval studies (Phase 4)Study completedNCT01742507
What this trial is testing

A Study Comparing Two Stent on the Degree of Early Stent Healing and Late Lumen Loss.The OCT-ORION Study

Who this might be right for
Coronary Artery Disease
The University of Hong Kong 60
Not applicableUnknownNCT02843334
What this trial is testing

Study of the Prevalence of Fabry Disease in French Dialysis Patients

Who this might be right for
Fabry DiseaseEnd Stage Renal DiseaseRenal Dialysis
Hospices Civils de Lyon 6,000
Not applicableStudy completedNCT03292887
What this trial is testing

Hunter Outcome Survey (HOS)

Who this might be right for
Hunter Syndrome
Shire 1,443
Not applicableLooking for participantsNCT07290114
What this trial is testing

Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape

Who this might be right for
Stress Urinary Incontinence (SUI)
Mehmet Incebıyik 147
Not applicableStudy completedNCT03490214
What this trial is testing

Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography

Who this might be right for
Duchenne Muscular DystrophyMuscular DystrophiesMuscular Dystrophy, Duchenne
University of Erlangen-Nürnberg Medical School 20
Testing effectiveness (Phase 2)Study completedNCT00176865
What this trial is testing

Stem Cell Transplant for Immunologic or Histiocytic Disorders

Who this might be right for
Hemophagocytic LymphohistiocytosisX-Linked Lymphoproliferative DisordersChediak-Higashi Syndrome+3 more
Masonic Cancer Center, University of Minnesota 19
Not applicableUnknownNCT02854956
What this trial is testing

Clinical Phenotyping and Characterization of Neural Networks and Cognitive Processes Involved in Mental Retardation X-linked

Who this might be right for
X-linked Mental Retardation
Hospices Civils de Lyon 573
Large-scale testing (Phase 3)Study completedNCT03938792
What this trial is testing

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Who this might be right for
Hemophilia AHemophilia B
Pfizer 189
Not applicableStudy completedNCT03589612
What this trial is testing

Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy

Who this might be right for
Duchenne Muscular DystrophyPerformanceFlexibility
Hacettepe University 60
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