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Showing 1–14 of 14 trials
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Recruiting
A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma
NCT07075185
- Conditions
- Blood Protein Disorders, Cardiovascular Diseases, Disease, +24 more
- Locations
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- City of Hope Duarte, California, United States
- Stanford University Palo Alto, California, United States
- UCSF San Francisco, California, United States
- Moffitt Cancer Center Tampa, Florida, United States
NCT03311503
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gene Therapy, Genetic Diseases, Inborn, +8 more
- Locations
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- Mattel Children's Hospital - UCLA Los Angeles, California, United States
- Emory University/Childrens Healthcare of Atlanta Atlanta, Georgia, United States
- Boston Childrens Hospital Boston, Massachusetts, United States
- Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
Recruiting
CIBMTR Research Database
NCT01166009
- Conditions
- Allogeneic Stem Cell Transplantation, Autologous Stem Cell Transplantation, Blood Cancers, +9 more
- Locations
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- Center for International Blood and Marrow Transplant Research Minneapolis, Minnesota, United States
- Medical College of Wisconsin Milwaukee, Wisconsin, United States
Recruiting
Gene Therapy for Chinese Hemophilia A
NCT04728841
- Conditions
- Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
- Locations
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- Chinese Academy of Medical Science and Blood Disease Hospital Tianjin, Tianjin Municipality, China
NCT07024524
- Conditions
- Congenital Hearing Loss, Gene Therapy, Rehabilitation, +2 more
- Locations
-
- Eye & ENT Hospital of Fudan University Shanghai, China
NCT05991336
- Conditions
- Gene Therapy, Transfusion-dependent Beta-Thalassemia
- Locations
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- Regenerative Medicine Center Tianjin, Tianjin Municipality, China
Enrolling by Invitation
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD
NCT05210803
- Conditions
- AMD, Gene Therapy, Neovascular Age-related Macular Degeneration (nAMD), +3 more
- Locations
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- Retinal Research Institute, LLC Phoenix, Arizona, United States
- California Retina Consultants Bakersfield, California, United States
- Retina-Vitreous Associates Medical Group Beverly Hills, California, United States
- Northern California Retina Vitreous Associates Medical Group Inc Mountain View, California, United States
Not Yet Recruiting
A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease
NCT07085195
- Conditions
- Basal Ganglia Diseases, Brain Diseases, Central Nervous System Diseases, +10 more
- Locations
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- Department of neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine Shanghai, China
Not Yet Recruiting
A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis
NCT06963008
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystic Fibrosis, Cystic Fibrosis (CF), +7 more
- Locations
-
Not specified
Not Yet Recruiting
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
NCT06948019
- Conditions
- AP4B1, Central Nervous System Diseases, Congenital Abnormalities, +31 more
- Locations
-
- Boston Children's Hospital Boston, Massachusetts, United States
Completed
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
NCT03306277
- Conditions
- Central Nervous System Diseases, Gene Therapy, Motor Neuron Disease, +6 more
- Locations
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- David Geffen School of Medicine at UCLA Los Angeles, California, United States
- Stanford University Stanford, California, United States
- Children's Hospital Colorado Aurora, Colorado, United States
- Nemours Children's Hospital Orlando, Florida, United States
NCT05044845
- Conditions
- Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +8 more
- Locations
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- St. Jude Children's Research Hospital Memphis, Tennessee, United States
- Civil Service Hospital Kathmandu, Nepal
- Hospital Dos De Mayo Lima, Peru
- National Hospital of Sri Lanka Colombo, Sri Lanka
NCT03734588
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, +18 more
- Locations
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- Orthopaedic Institute for Children Los Angeles, California, United States
- Illinois Bleeding and Clotting Disorders Institute Peoria, Illinois, United States
- University of Michigan Ann Arbor, Michigan, United States
- Mississippi Center for Advanced Medicine Madison, Mississippi, United States
NCT01024998
- Conditions
- Age-Related Maculopathies, Age-Related Maculopathy, Eye Diseases, +13 more
- Locations
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- investigational site number 03WilmerEy Baltimore, Maryland, United States
- investigational site number 02Ophthalm Boston, Massachusetts, United States
- investigational site number 01UMassMem Worcester, Massachusetts, United States
- investigational site number 05RetinaCo Slingerlands, New York, United States