Search clinical trials for: AAV vectors

Total 357 results

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
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
GenSight Biologics

Completed

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

Leber Hereditary Optic Neuropathy

United States, France, Italy, United Kingdom, Germany
GenSight Biologics

No longer available

EAP_GS010_single Patient

Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
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
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
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
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
GenSight Biologics

Completed

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE) (REVERSE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, Germany, Italy, France
Spark Therapeutics
Children's Hospital of Philadelphia; University of Pittsburgh; St. James's Hospital... and other collaborators

Terminated

Hemophilia B Gene Therapy With AAV8 Vector

Hemophilia B

United States, Australia
Tacere Therapeutics, Inc.

Terminated

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

Hepatitis C

United States
Tacere Therapeutics, Inc.

Completed

Safety and Efficacy Study of Single Doses of TT-034 in Patients With Chronic Hepatitis C

Hepatitis C

United States
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R09b in Severe AADC Deficiency

Aromatic L-amino Acid Decarboxylase Deficiency
Second Affiliated Hospital, School of Medicine,...

Recruiting

A Novel Viscoelastic Test Based on Ultrasonic Guided Wave for Identifying Hyperfibrinolysis Rapidly

Hyperfibrinolysis

China
NGGT (Suzhou) Biotechnology Co., Ltd.

Not yet recruiting

AAV Gene Therapy Clinical Study in Adult Classic PKU

Phenylketonurias
Amgen

Not yet recruiting

Study to Evaluate Avacopan in Combination With a Rituximab or Cyclophosphamide-containing Regimen, in Children From 6 Years to < 18 Years of Age With AAV.

Vasculitis
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
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
Zhongmou Therapeutics

Not yet recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients (ZM-02)

Retinitis Pigmentosa

China
ViGeneron GmbH

Recruiting

Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

Retinitis Pigmentosa

Germany
Lantu Biopharma

Not yet recruiting

An Open Label Study of Gene Therapy Product in Spinal Muscular Atrophy Patients

Spinal Muscular Atrophy
Asklepios Biopharmaceutical, Inc.
Bayer

Not yet recruiting

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (REGENERATE-PD)

Parkinson Disease
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (UK Only)

Dravet Syndrome

United Kingdom
Xinhua Hospital, Shanghai Jiao Tong University...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Type II Gaucher Disease

China
Alimentiv Inc.
Takeda

Not yet recruiting

VECTORS - A Study to Evaluate Transmural Healing as a Treatment Target in Crohn's Disease (VECTORS)

Crohn Disease | Moderately to Severely Active Crohn's Disease | Disease Crohn
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
NovelMed Therapeutics

Not yet recruiting

Study of NM8074 in Patients With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis (AAV)

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Sardocor Corp.

Not yet recruiting

Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD)

DMD-Associated Dilated Cardiomyopathy
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

Glutaric Acidemia Type I | Glutaric Aciduria Type I

China
Grace Science, LLC

Recruiting

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

NGLY1 Deficiency

United States
Mitsubishi Tanabe Pharma Corporation

Recruiting

An Exploratory Study of MT-2990 in Patients With AAV

Antineutrophil Cytoplasmic Antibody (ANCA) -Associated Vasculitis (AAV)

Japan
Lori Gawron
Planned Parenthood Association of Utah

Recruiting

Etonogestrel (ENG) Implant Insertion for Emergency Contraception With Oral Levonorgestrel (LNG) vs Placebo

Emergency Contraception

United States
Astellas Gene Therapies

Recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Pompe Disease (Late-onset)

United States, Spain, France, Taiwan, Italy
University of Notre Dame
Infectious Diseases Research Collaboration, Uganda; SC Johnson, A Family Company and other collaborators

Not yet recruiting

Spatial Repellents for Malaria Control (AEGIS Uganda)

Malaria

Uganda
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)

Dravet Syndrome

Australia
UniQure Biopharma B.V.

Not yet recruiting

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

Amyotrophic Lateral Sclerosis

United States
Weill Medical College of Cornell University
Massachusetts General Hospital

Recruiting

Prospective Study of Sensation and Satisfaction in Cancer and Transgender Mastectomy Patients

Pain, Postoperative | Sensation Disorders | Pain, Chronic | Sensory Disorder | Phantom Pain | Phantom Sensation | Sensation, Phantom | Sensory Defect | Numbness

United States
Amgen

Recruiting

A Study to Evaluate Avacopan in Participants With ANCA-associated Vasculitis

Antineutrophil Cytoplasmic Antibody-associated Vasculitis

United States
Apurano Pharmaceuticals GmbH

Recruiting

Efficacy and Tolerability of AP707 in Patients With Chronic Pain Due to Central Neuropathy of Any Genesis

Stroke | Pain | Multiple Sclerosis | Chronic Pain | Traumatic Brain Injury | Neuropathic Pain | Pain, Chronic | Chronic Pain Syndrome | Paraplegia | Phantom Pain | Pain Syndrome | Central Neuropathic Pain | Phantom Limb Syndrome With Pain

Germany
Apurano Pharmaceuticals GmbH

Recruiting

Long Term Efficacy and Tolerability of AP707 in Patients With Chronic Pain Due to Central Neuropathy of Any Genesis

Stroke | Pain | Multiple Sclerosis | Chronic Pain | Traumatic Brain Injury | Neuropathic Pain | Pain, Chronic | Chronic Pain Syndrome | Paraplegia | Phantom Pain | Pain Syndrome | Central Neuropathic Pain | Phantom Limb Syndrome With Pain

Germany
Virion Therapeutics

Recruiting

Dose-Escalation Prime/Boost Therapeutic Vaccination Study Of 2 Chimp Adenoviral Vectors in Adults With Chronic HBV On Nucleos(t)Ide Therapy

Chronic Hepatitis B

Hong Kong, New Zealand
Zhongmou Therapeutics

Recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

X-linked Retinoschisis

China
uniQure France SAS

Recruiting

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

Mesial Temporal Lobe Epilepsy

United States
AviadoBio Ltd

Recruiting

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN) (ASPIRE-FTD)

Frontotemporal Dementia | FTD | FTD-GRN | Dementia, Frontotemporal

Netherlands, Spain, Poland, United States
Sardocor Corp.

Recruiting

Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction (MUSIC-HFpEF)

Heart Failure, Diastolic | Heart Failure With Preserved Ejection Fraction

United States
Zuyderland Medisch Centrum

Recruiting

Combining CEM and Magnetic Seed Localization of Non-Palpable Breast Tumors (CEMMAG)

Breast Cancer

Netherlands
RenJi Hospital

Recruiting

Exploratory Research of PCSK9 Inhibitor on Patency of aAVF After PTA With PCB

Hemodialysis Access Failure

China

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