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Results for “Adeno-Associated Virus (AAV)”

79 trials

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Showing 20 of 79 results

Large-scale testing (Phase 3)Active Not RecruitingNCT00999609
What this trial is testing

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Inherited Retinal Dystrophy Due to RPE65 MutationsLeber Congenital Amaurosis
Spark Therapeutics, Inc. 31
Testing effectiveness (Phase 2)Looking for participantsNCT07443826
What this trial is testing

CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy

Who this might be right for
Age-related Muscle Decline
Unlimited Biotechnology LLC 12
Testing effectiveness (Phase 2)Study completedNCT00888446
What this trial is testing

Safety and Immunogenicity Study of tgAAC09, an HIV Vaccine in an Adeno-associated Virus (AAV) Capsid

Who this might be right for
Human Immunodeficiency Virus InfectionsHIV Infections
International AIDS Vaccine Initiative 91
Large-scale testing (Phase 3)Looking for participantsNCT07160634
What this trial is testing

SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

Who this might be right for
Duchenne Muscular Dystrophy
Solid Biosciences Inc. 80
Not applicableStudy completedNCT05101902
What this trial is testing

Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

Who this might be right for
Congenital Adrenal Hyperplasia
Adrenas Therapeutics Inc 65
Not applicableLooking for participantsNCT03466463
What this trial is testing

Gene Therapy for Severe Crigler Najjar Syndrome

Who this might be right for
Crigler-Najjar Syndrome
Genethon 17
Testing effectiveness (Phase 2)Active Not RecruitingNCT04480567
What this trial is testing

AAV Gene Therapy Study for Subjects with PKU

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 100
Testing effectiveness (Phase 2)Looking for participantsNCT05788536
What this trial is testing

DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations

Who this might be right for
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Regeneron Pharmaceuticals 30
Not applicableStudy completedNCT02971969
What this trial is testing

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Who this might be right for
Hemophilia B
Ultragenyx Pharmaceutical Inc 6
Testing effectiveness (Phase 2)Ended earlyNCT02418598
What this trial is testing

AADC Gene Therapy for Parkinson's Disease

Who this might be right for
Parkinson Disease
Jichi Medical University 2
Not applicableStudy completedNCT04560933
What this trial is testing

Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 186
Testing effectiveness (Phase 2)Active Not RecruitingNCT03588299
What this trial is testing

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Who this might be right for
Hemophilia A
Bayer 11
Testing effectiveness (Phase 2)Active Not RecruitingNCT04783181
What this trial is testing

Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

Who this might be right for
Congenital Adrenal Hyperplasia
Adrenas Therapeutics Inc 8
Early research (Phase 1)Study completedNCT00482027
What this trial is testing

Safety and Immunogenicity Study of tgAAC09, a Gag-PR-RT AAV HIV Vaccine

Who this might be right for
HIV Infection
International AIDS Vaccine Initiative 80
Testing effectiveness (Phase 2)Active Not RecruitingNCT05243017
What this trial is testing

Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease

Who this might be right for
Huntington Disease
UniQure Biopharma B.V. 14
Not applicableEnded earlyNCT05580692
What this trial is testing

A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 321
Testing effectiveness (Phase 2)Study completedNCT02053064
What this trial is testing

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

Who this might be right for
Mucopolysaccharidosis Type III ASanfilippo Disease Type A
LYSOGENE 4
Early research (Phase 1)Study completedNCT00195143
What this trial is testing

Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease

Who this might be right for
Parkinson's Disease
Neurologix, Inc. 12
Testing effectiveness (Phase 2)Ended earlyNCT00643890
What this trial is testing

Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease

Who this might be right for
Parkinson's Disease
Neurologix, Inc. 44
Testing effectiveness (Phase 2)Study completedNCT03734588
What this trial is testing

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Who this might be right for
Adeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, Inherited+14 more
Spark Therapeutics, Inc. 4
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