Search clinical trials for: adeno associated virus

Total 44835 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, United Kingdom, France, Italy
Tenaya Therapeutics
Johns Hopkins University; Mayo Clinic

Recruiting

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (RIDGE-1)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States
Zhejiang Cancer Hospital

Recruiting

The Use of Shengmai San to Prevent Anthracycline Sequential Trastuzumab Therapy Related Cardiac Toxicity (START)

Cardiotoxicity Induced by Drug Therapy for Breast Cancer

China
Grace Science, LLC

Recruiting

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

NGLY1 Deficiency

United States
West China Hospital

Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

China
Shanghai Belief-Delivery BioMed Co., Ltd
Shanghai MYGT Biopharmaceutical LLC

Recruiting

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Hemophilia A

China
UniQure Biopharma B.V.

Not yet recruiting

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

Amyotrophic Lateral Sclerosis
CSL Behring

Recruiting

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

Hemophilia B

United States, Canada, Israel
TScan Therapeutics, Inc.

Recruiting

A Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors

Melanoma | Cervical Cancer | Head and Neck Cancer | HPV-Related Malignancy | Ovarian Cancer | HPV-Related Carcinoma | HPV-Related Cervical Carcinoma | HPV Positive Oropharyngeal Squamous Cell Carcinoma | Non-small Cell Carcinoma | HPV - Anogenital Human Papilloma Virus Infection | HPV-Related Adenocarcinoma | HPV-Related Adenosquamous Carcinoma and other conditions

United States
Yu jiren

Not yet recruiting

Perioperative Chemotherapy Plus Toripalimab for Epstein-Barr Virus-associated Locally Advanced Gastric or Esophagogastric Junction Adenocarcinoma

Adenocarcinoma of the Stomach | Adenocarcinoma of Esophagogastric Junction | Epstein-Barr Virus-Associated Gastric Carcinoma

China
National Cancer Institute (NCI)

Recruiting

Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer

Metastatic Melanoma | Unresectable Melanoma | Clinical Stage III Cutaneous Melanoma AJCC v8 | Metastatic Merkel Cell Carcinoma | Metastatic Skin Squamous Cell Carcinoma | Clinical Stage IV Cutaneous Melanoma AJCC v8 | Metastatic Basal Cell Carcinoma | Unresectable Merkel Cell Carcinoma | Unresectable... and other conditions

United States
Tenaya Therapeutics

Recruiting

Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (MyPEAK-1)

Hypertrophic Cardiomyopathy

United States
Shanghai East Hospital
Shanghai Juvensis Therapeutics Biotechnology Company Limited

Recruiting

Myocardial Telomere Recapping Study for Dilated Cardiomyopathy (MERCURY-DCM)

Heart Failure | Dilated Cardiomyopathy

China
Xiangya Hospital of Central South University

Not yet recruiting

Safety and Efficacy Study of Parkinson's Disease Gene Therapy Drug (BBM-P002)

Parkinson's Disease

China
Regeneron Pharmaceuticals

Recruiting

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

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Spain, United Kingdom, United States
Christian Hinrichs

Recruiting

E7 TCR-T Cell Immunotherapy for Human Papillomavirus (HPV) Associated Cancers

Cervical Cancer | HPV-Related Malignancy | Metastatic Cancer | Anal Cancer | Oropharynx Cancer | Vaginal Cancer | HPV-Related Carcinoma | HPV-Related Cervical Carcinoma | HPV Positive Oropharyngeal Squamous Cell Carcinoma | Penile Cancer | HPV-Related Adenocarcinoma | HPV-Related Adenosquamous Carcinoma | HPV-Related... and other conditions

United States
Opus Genetics, Inc

Recruiting

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

LCA5

United States
BioMarin Pharmaceutical

Recruiting

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

Hemophilia A

Argentina
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
Seventh Medical Center of PLA General Hospital
GeneCradle Therapeutics, Inc

Recruiting

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

Infantile-onset Pompe Disease

China
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.

Hemophilia A

China
University of Texas Southwestern Medical Center
Cure SPG50

Recruiting

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

Microcephaly | Spasticity, Muscle | Growth Retardation | Intellectual Deficiency | SPG50 | Spastic Paraplegia

United States
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

A Study of FVIII Gene Therapy for Hemophilia A

Hemophilia A

China
ICM Co. Ltd.

Not yet recruiting

A Study Evaluating the Safety, Tolerability, and Activity of ICM-203 in Subjects With Knee Osteoarthritis.

Osteoarthritis, Knee
Jules Bordet Institute

Recruiting

Immunological Variables Associated to ICI Toxicity in Cancer Patients

Melanoma | Renal Cell Carcinoma | Breast Cancer | Head and Neck Cancer | Small Cell Lung Cancer | Hepatocellular Carcinoma | Non Small Cell Lung Cancer | Mesothelioma, Malignant | Bladder Cancer | Gastrointestinal Cancer | Merkel Cell Carcinoma | Non-melanoma Skin Cancer | MSI-H Colorectal Cancer

Belgium
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
ICM Biotech Australia Pty Ltd.

Recruiting

A Study Evaluating the Safety, Tolerability, and Range of Biologically Active Doses of ICM-203 in Mild to Moderate Knee Osteoarthritis

Osteoarthritis, Knee

Australia
Beacon Therapeutics

Recruiting

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

X-Linked Retinitis Pigmentosa

United States
Myrtelle Inc.

Recruiting

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease (CAN-GT)

Canavan Disease

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
Taproot Health

Recruiting

The Registry of Oncology Outcomes Associated With Testing and Treatment (ROOT)

Melanoma | Sarcoma | Kidney Cancer | Adenocarcinoma | Renal Cell Carcinoma | Cervical Cancer | Acute Myeloid Leukemia | Stomach Cancer | Breast Cancer | Head and Neck Neoplasms | Nasopharyngeal Carcinoma | Glioblastoma | Astrocytoma | Multiple Myeloma | Colorectal Cancer | Pancreatic Cancer | Esophageal Cancer | Small Bowel Cancer and other conditions

United States
Eutilex

Recruiting

A Multi-center, Single-arm, Open, Phase I/IIa Clinical Trial to Evaluate the Efficacy and Safety of EBViNT Cell (EBV Specific Autologous CD8+ T Cell) in Patients With Treatment Failed Epstein Barr Virus (EBV)-Positive Malignancies

EBV Associated Extranodal NK/T-cell Lymphoma | EBV-Associated GastricCarcinoma or Esophageal AdenoCarcinoma

Korea, Republic of
Genethon

Recruiting

Gene Therapy for Severe Crigler Najjar Syndrome (CareCN)

Crigler-Najjar Syndrome

Italy, France, Netherlands
eyeDNA Therapeutics

Recruiting

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

Retinitis Pigmentosa

France
Genascence Corporation
California Institute for Regenerative Medicine (CIRM)

Active, not recruiting

Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis (DONATELLO)

Knee Osteoarthritis | Osteoarthritis, Knee | Osteo Arthritis Knee

United States
National Cancer Institute (NCI)

Active, not recruiting

Nivolumab and Ipilimumab in Treating Patients With Rare Tumors

Malignant Solid Neoplasm | Nasopharyngeal Carcinoma | Adenoid Cystic Carcinoma | Transitional Cell Carcinoma | Cholangiocarcinoma | Intrahepatic Cholangiocarcinoma | Chordoma | Gestational Trophoblastic Tumor | Pseudomyxoma Peritonei | Cervical Adenocarcinoma | Vaginal Adenocarcinoma | Vaginal Squamous Cell... and other conditions

United States, Guam
BioMarin Pharmaceutical

Completed

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

Hemophilia A

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
Claire Verschraegen

Completed

Nivolumab and Radiation Therapy or Ipilimumab as Adjuvant Therapy in Treating Patients With Merkel Cell Cancer

Merkel Cell Carcinoma | Pathologic Stage IIIA Merkel Cell Carcinoma AJCC v8 | Pathologic Stage IIIB Merkel Cell Carcinoma AJCC v8

United States
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
Beacon Therapeutics

Active, not recruiting

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

X-Linked Retinitis Pigmentosa

United States
Ultragenyx Pharmaceutical Inc

Active, not recruiting

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

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
National Cancer Institute (NCI)

Active, not recruiting

Tacrolimus, Nivolumab, and Ipilimumab in Treating Kidney Transplant Recipients With Selected Unresectable or Metastatic Cancers

Metastatic Melanoma | Unresectable Melanoma | Clinical Stage III Cutaneous Melanoma AJCC v8 | Pathologic Stage IIIB Cutaneous Melanoma AJCC v8 | Pathologic Stage IIIC Cutaneous Melanoma AJCC v8 | Pathologic Stage IIID Cutaneous Melanoma AJCC v8 | Metastatic Merkel Cell Carcinoma | Metastatic Skin... and other conditions

United States
Beacon Therapeutics

Enrolling by invitation

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

X-Linked Retinitis Pigmentosa

United States
Sarepta Therapeutics, Inc.
Hansa Biopharma AB

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Duchenne Muscular Dystrophy

Spain
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
Beacon Therapeutics

Active, not recruiting

Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (HORIZON)

X-Linked Retinitis Pigmentosa

United States
Sarepta Therapeutics, Inc.

Active, not recruiting

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Limb-Girdle Muscular Dystrophy, Type 2E

United States
Baxalta now part of Shire

Active, not recruiting

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B

Hemophilia B

United States

Search clinical trials for: adeno associated virus

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