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Results for “Adeno-Associated Virus (AAV)”

77 trials

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Showing 20 of 77 results

Early research (Phase 1)Study completedNCT00494195
What this trial is testing

Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)

Who this might be right for
Muscular Dystrophies
Nationwide Children's Hospital 6
Not applicableLooking for participantsNCT06311708
What this trial is testing

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

Who this might be right for
Arrhythmogenic Right Ventricular Cardiomyopathy
Tenaya Therapeutics 200
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 applicableStudy completedNCT02302690
What this trial is testing

Immunity Against AAV in Crigler Najjar Patient

Who this might be right for
Crigler Najjar Syndrome
Genethon 49
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
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
Testing effectiveness (Phase 2)Looking for participantsNCT06641154
What this trial is testing

Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)

Who this might be right for
Crigler-Najjar Syndrome Type I
Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare 5
Not applicableEnded earlyNCT04909346
What this trial is testing

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Who this might be right for
Ornithine Transcarbamylase DeficiencyWilson DiseaseGlycogen Storage Disease Type IA
Ultragenyx Pharmaceutical Inc 51
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
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
Not applicableStudy completedNCT04135300
What this trial is testing

Gene Therapy for Chinese Hemophilia B

Who this might be right for
Hemophilia B
Institute of Hematology & Blood Diseases Hospital, China 10
Early research (Phase 1)Study completedNCT00516477
What this trial is testing

Safety Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Leber Congenital Amaurosis
Spark Therapeutics, Inc. 12
Not applicableActive Not RecruitingNCT03636438
What this trial is testing

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc 11
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)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
Not applicableUnknownNCT05567627
What this trial is testing

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

Who this might be right for
Infantile-onset Pompe Disease
Seventh Medical Center of PLA General Hospital 6
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
Large-scale testing (Phase 3)Looking for participantsNCT06971094
What this trial is testing

Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III

Who this might be right for
SMA - Spinal Muscular Atrophy
GeneCradle Inc 50
Large-scale testing (Phase 3)Active Not RecruitingNCT05139316
What this trial is testing

Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Who this might be right for
Glycogen Storage Disease Type IA
Ultragenyx Pharmaceutical Inc 49
Large-scale testing (Phase 3)Study completedNCT03569891
What this trial is testing

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Who this might be right for
Hemophilia B
CSL Behring 67
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