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Results for “Adeno-Associated Virus (AAV)”

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Showing 20 of 0 results

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
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
Very early researchLooking for participantsNCT05454774
What this trial is testing

FVIII Gene Therapy for Hemophilia A

Who this might be right for
Hemophilia A
Institute of Hematology & Blood Diseases Hospital, China 8
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
Very early researchNot Yet RecruitingNCT07533591
What this trial is testing

Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus

Who this might be right for
Parkinson's Disease (PD)
Second Affiliated Hospital, Zhejiang University, School of Medicine 6
Testing effectiveness (Phase 2)Active Not RecruitingNCT05541627
What this trial is testing

Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

Who this might be right for
Huntington Disease
AskBio France, SAS, a subsidiary of AskBio Inc. 5
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
Testing effectiveness (Phase 2)Ended earlyNCT03328130
What this trial is testing

Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B Gene

Who this might be right for
Retinitis Pigmentosa
eyeDNA Therapeutics 19
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)Study completedNCT01054339
What this trial is testing

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

Who this might be right for
Alpha-1 Antitrypsin Deficiency
Beacon Therapeutics 9
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
Early research (Phase 1)Looking for participantsNCT06228924
What this trial is testing

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

Who this might be right for
Arrhythmogenic Right Ventricular Cardiomyopathy
Tenaya Therapeutics 15
Testing effectiveness (Phase 2)Study completedNCT03003533
What this trial is testing

A Gene Transfer Study for Hemophilia A

Who this might be right for
Hemophilia A
Spark Therapeutics, Inc. 25
Not applicableEnded earlyNCT02315638
What this trial is testing

Long-Term Follow-Up Safety Monitoring of Patients Dosed in the First-in-Man Phase I/II Study of TT-034.

Who this might be right for
Hepatitis C
Tacere Therapeutics, Inc. 9
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06275620
What this trial is testing

A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)

Who this might be right for
X-Linked Retinitis Pigmentosa
Beacon Therapeutics 24
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
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
Very early researchNot Yet RecruitingNCT06645197
What this trial is testing

An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis

Who this might be right for
Amyotrophic Lateral Sclerosis (ALS)
Peking University Third Hospital 7
Testing effectiveness (Phase 2)Active Not RecruitingNCT06100276
What this trial is testing

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

Who this might be right for
Amyotrophic Lateral Sclerosis
UniQure Biopharma B.V. 20
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
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