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Results for “X-linked Genetic Diseases”

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Showing 20 of 0 results

Not applicableUnknownNCT04710186
What this trial is testing

AR, IGF-IR, IR and Peripheral Artery Disease

Who this might be right for
Androgen Receptor AbnormalInsulin-Like Growth Factor I DeficiencyInsulin Receptor, Defect in
University of Catanzaro 30
Not applicableStudy completedNCT00004773
What this trial is testing

Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome

Who this might be right for
Rett Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 95
Not applicableStudy completedNCT05409846
What this trial is testing

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

Who this might be right for
Fabry Disease
Universidade do Porto 409
Testing effectiveness (Phase 2)Study completedNCT04046224
What this trial is testing

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Who this might be right for
Fabry Disease
Sangamo Therapeutics 36
Testing effectiveness (Phase 2)Study completedNCT03307980
What this trial is testing

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

Who this might be right for
Hemophilia B
Pfizer 21
Large-scale testing (Phase 3)Study completedNCT00864851
What this trial is testing

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

Who this might be right for
Fabry Disease
Shire 44
Not applicableStudy completedNCT04007536
What this trial is testing

Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Who this might be right for
Mucopolysaccharidosis II
Denali Therapeutics Inc. 18
Not applicableStudy completedNCT05044845
What this trial is testing

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Who this might be right for
Gene TherapyHemophilia B
St. Jude Children's Research Hospital 150
Not applicableLooking for participantsNCT05630651
What this trial is testing

The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.

Who this might be right for
Hemophilia B
Institute of Hematology & Blood Diseases Hospital, China 6
Not applicableUnknownNCT02082353
What this trial is testing

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

Who this might be right for
Granulomatous Disease, Chronic
National Institute of Allergy and Infectious Diseases (NIAID) 1,480
Not applicableUnknownNCT05719337
What this trial is testing

Multi-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy Adults

Who this might be right for
Left Ventricular Hypertrophy
First Hospital of China Medical University 660
Not applicableStudy completedNCT00359411
What this trial is testing

Genetic Studies of X-linked Lymphoproliferative Disease

Who this might be right for
X-Linked Lymphoproliferative DiseaseLymphoproliferative DiseaseGenetic Diseases, X-Linked
National Institute of Allergy and Infectious Diseases (NIAID) 12
Not applicableApproved For MarketingNCT04337112
What this trial is testing

The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

Who this might be right for
Muscular Dystrophy, DuchenneDMD
NS Pharma, Inc.
Not applicableNot Yet RecruitingNCT07730112
What this trial is testing

Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

Who this might be right for
Rett SyndromeFamily CaregiversSocial Support+1 more
Colorado State University 60
Testing effectiveness (Phase 2)Active Not RecruitingNCT03153319
What this trial is testing

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis VI
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center 14
Early research (Phase 1)Study completedNCT00001765
What this trial is testing

Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease

Who this might be right for
Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID) 60
Not applicableStudy completedNCT01386775
What this trial is testing

Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia: Intrafamilial Variation

Who this might be right for
Hypohidrotic Ectodermal Dysplasia
Edimer Pharmaceuticals 64
Very early researchActive Not RecruitingNCT03910452
What this trial is testing

Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

Who this might be right for
Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID) 4
Not applicableStudy completedNCT02090608
What this trial is testing

Paricalcitol in Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
Federico II University 14
Not applicableLooking for participantsNCT06982417
What this trial is testing

InsightRP2 Registry

Who this might be right for
RP2-associated Retinitis PigmentosaX-Linked Retinitis Pigmentosa (XLRP)Retinitis Pigmentosa 2
University of Göttingen 200
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