Search clinical trials for: AAV vector

Total 3136 results

Tenaya Therapeutics
Medical University of South Carolina; Johns Hopkins University; University of... and other collaborators

Recruiting

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) (RIDGE)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States, Italy, United Kingdom, France, Sweden, Germany
Grace Science, LLC

Recruiting

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

NGLY1 Deficiency

United States
Pfizer

Recruiting

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

Hemophilia A | Hemophilia B

Australia, United States, Turkey
SwanBio Therapeutics, Inc.

Recruiting

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN) (PROPEL)

AMN | AMN Gene Mutation | X-ALD

Netherlands, United States
Weill Medical College of Cornell University
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Phase IA Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Myocardial Fibrosis | Cardiomyopathies | Friedreich Ataxia | Cardiac Hypertrophy

United States
UniQure Biopharma B.V.

Recruiting

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

Huntington Disease

Poland, United Kingdom, Germany
BioMarin Pharmaceutical

Recruiting

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (GENEr8-INH)

Hemophilia A With Inhibitor | Hemophilia A With Anti Factor VIII

United States, United Kingdom, Taiwan, Israel, Korea, Republic of, South Africa, Brazil, Italy, Germany
Pfizer

Recruiting

A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.

Household Contacts

United States
UniQure Biopharma B.V.

Recruiting

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

Huntington's Disease

United States
National Human Genome Research Institute (NHGRI)
Sio Gene Therapies

Recruiting

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Gangliosidosis | Lysosomal Diseases | GM1

United States
GenSight Biologics

Recruiting

Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa (PIONEER)

Non-syndromic Retinitis Pigmentosa

United States, France, United Kingdom
CSL Behring

Active, not recruiting

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

Hemophilia B

United States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
National Institute of Neurological Disorders and...

Completed

AAV2-GDNF for Advanced Parkinson s Disease

Parkinson's Disease

United States
VegaVect, Inc.
National Eye Institute (NEI)

Active, not recruiting

Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

Retinoschisis | X-Linked

United States
Spark Therapeutics, Inc.
Children's Hospital of Philadelphia; University of Iowa

Active, not recruiting

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Leber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 Mutations

United States
Nanoscope Therapeutics Inc.

Completed

Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE] (RESTORE)

Eye Diseases | Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa | Retinitis | Eye Diseases, Hereditary | Retinal Dystrophies

United States, Puerto Rico
Spark Therapeutics

Completed

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

Hematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditions

United States
GenSight Biologics

Completed

RESCUE and REVERSE Long-term Follow-up (RESTORE)

Leber Hereditary Optic Neuropathy

United States, France, Italy, United Kingdom, Germany
Spark Therapeutics

Completed

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Choroideremia | CHM (Choroideremia) Gene Mutations

United States
Spark Therapeutics

Active, not recruiting

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

United States
Spark Therapeutics

Completed

A Gene Transfer Study for Hemophilia A

Hemophilia A

United States, Australia, Canada, Israel, Thailand
Adverum Biotechnologies, Inc.
Parexel

Active, not recruiting

Safety and Efficacy of ADVM-022 in Treatment-Experienced Patients With Neovascular Age-related Macular Degeneration [LUNA]

Neovascular Age-related Macular Degeneration

United States, France, United Kingdom
GenSight Biologics

No longer available

EAP_GS010_single Patient

Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Severe Haemophilia A

United Kingdom
Nanoscope Therapeutics Inc.

Enrolling by invitation

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study (REMAIN)

Eye Diseases | Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa | Retinitis | Eye Diseases, Hereditary | Retinal Dystrophies

United States, Puerto Rico
Byron Lam
National Eye Institute (NEI)

Active, not recruiting

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy

United States
CSL Behring

Completed

Dose Confirmation Trial of AAV5-hFIXco-Padua

Hemophilia B

United States
Adverum Biotechnologies, Inc.

Completed

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency (ADVANCE)

Alpha 1-Antitrypsin Deficiency

United States
Spark Therapeutics

Active, not recruiting

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Leber Congenital Amaurosis

United States
MeiraGTx, LLC
National Institute of Dental and Craniofacial Research (NIDCR)

Active, not recruiting

Safety of a Single Administration of AAV2hAQP1, an Adeno-Associated Viral Vector Encoding Human Aquaporin-1 to One Parotid Salivary Gland in People With Irradiation-Induced Parotid Salivary Hypofunction

Squamous Cell Head and Neck Cancer | Radiation Induced Xerostomia | Salivary Hypofunction

United States
Adverum Biotechnologies, Inc.

Completed

ADVM-022 Intravitreal Gene Therapy for DME (INFINITY)

Diabetic Retinopathy | Diabetic Macular Edema

United States, Puerto Rico
Adverum Biotechnologies, Inc.

Completed

ADVM-022 Intravitreal Gene Therapy for Wet AMD (OPTIC)

Neovascular Age-related Macular Degeneration | Wet Age-related Macular Degeneration

United States
Nanoscope Therapeutics Inc.

Enrolling by invitation

A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection (EXTEND)

Retinal Degeneration | Retinitis Pigmentosa | Retinal Disease

India
Brainvectis, a subsidiary of Asklepios BioPharmaceutical...

Active, not recruiting

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

Huntington Disease

France
Baxalta now part of Shire
Takeda Development Center Americas, Inc.; Baxalta Innovations GmbH, now part...

Active, not recruiting

A Study of BAX 888 in Male Adults With Severe Hemophilia A

Hemophilia A

United States, Spain, Hungary, Italy, Germany, Austria, France
Adverum Biotechnologies, Inc.

Active, not recruiting

Long-term Study of ADVM-022 in Neovascular (Wet) AMD [OPTIC-EXT]

Neovascular Age-related Macular Degeneration | Wet Age-related Macular Degeneration

United States
National Taiwan University Hospital
PTC Therapeutics

Completed

A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC (AADC)

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Taiwan
Biocad

Terminated

Clinical Study of the Safety and Immunogenicity of a Recombinant Viral Vector AAV5 (Adeno-Associated Virus Type 5 )-RBD (Receptor Binding Domain)-S Vaccine for the Prevention of Coronavirus Infection (COVID-19) (COVER)

COVID-19 | Coronavirus Infection

Russian Federation
MeiraGTx UK II Ltd
Janssen Research & Development, LLC

Completed

Gene Therapy for Achromatopsia (CNGA3) (CNGA3)

Achromatopsia

United Kingdom, United States
Adverum Biotechnologies, Inc.

Enrolling by invitation

Long-Term Follow-up Study of ADVM-022 in DME (INFINITY-EXT)

Diabetic Retinopathy | Diabetic Macular Edema

United States, Puerto Rico
GenSight Biologics

Active, not recruiting

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT)

Leber Hereditary Optic Neuropathy

Belgium, Spain, United States, France, United Kingdom, Taiwan, Italy
GenSight Biologics

Completed

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, France, Germany, Italy
Baxalta now part of Shire

Terminated

A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

Hemophilia B

Turkey, Spain
Adverum Biotechnologies, Inc.

Completed

Long-Term Follow-up Study of ADVM-043

Alpha-1 Antitrypsin Deficiency

United States
Neurocrine Biosciences
Voyager Therapeutics

Completed

Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease

Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Parkinson's Disease | Parkinsonian Disorders | Movement Disorders | Neurodegenerative Diseases | Idiopathic Parkinson's Disease | Basal Ganglia Disease

United States
Institut National de la Santé Et de la Recherche...
Assistance Publique - Hôpitaux de Paris; European Leukodystrophy Association

Active, not recruiting

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy (TG-MLD)

Metachromatic Leukodystrophy

France
Nanoscope Therapeutics Inc.

Completed

Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa

Retinal Degeneration | Retinal Diseases | Retinitis Pigmentosa

India
GenSight Biologics

Completed

REALITY LHON Registry (REALITY)

Leber Hereditary Optic Neuropathy

United States, France, Italy, Spain, United Kingdom
Weill Medical College of Cornell University
National Institutes of Health (NIH)

Completed

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Batten Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis

United States
Weill Medical College of Cornell University
Nathan's Battle Foundation

Completed

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

Batten Disease | Late Infantile Neuronal Ceroid Lipofuscinosis

United States

Search clinical trials for: AAV vector

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