Search clinical trials for: AAV directed hLDLR gene therapy

Total 449 results

REGENXBIO Inc.
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Italy, Netherlands
Neuracle Genetics, Inc
ORA, Inc.

Recruiting

NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration

Age-Related Macular Degeneration

Canada
Biogen

Completed

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111 (GEMINI)

Choroideremia

United States, Germany, France
The Methodist Hospital Research Institute

Recruiting

HSV-tk and XRT and Chemotherapy for Newly Diagnosed GBM

Glioblastoma | Anaplastic Astrocytoma

United States
REGENXBIO Inc.

Active, not recruiting

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

Homozygous Familial Hypercholesterolemia (HoFH)

United States, Canada, Netherlands
David Baskin MD
Center for Cell and Gene Therapy, Baylor College of Medicine

Recruiting

HSV-tk + Valacyclovir + SBRT + Chemotherapy for Recurrent GBM

Glioblastoma Multiforme | Astrocytoma, Grade III

United States
University of Oxford
University College, London; Moorfields Eye Hospital NHS Foundation Trust

Completed

REP1 Gene Replacement Therapy for Choroideremia (REGENERATE)

Choroideremia

United Kingdom
REGENXBIO Inc.

Active, not recruiting

A First-in-Human Study in Pediatric Patients With Ocular CLN2 Disease

Neuronal Ceroid Lipofuscinosis Type 2

United Kingdom
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
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, 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
Brain Neurotherapy Bio, Inc.

Recruiting

GDNF Gene Therapy for Multiple System Atrophy

Multiple System Atrophy

United States
Genethon

Completed

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Limb Girdle Muscular Dystrophy Type 2C | Gamma-sarcoglycanopathy

France
Mark Tuszynski
Ohio State University

Recruiting

A Clinical Trial of AAV2-BDNF Gene Therapy in Early Alzheimer's Disease and Mild Cognitive Impairment

Mild Cognitive Impairment | Alzheimer's Disease

United States
Pfizer

Active, not recruiting

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE)

Hemophilia A

Spain, Taiwan, Australia, United States, Japan, France, Turkey, Germany, Korea, Republic of, United Kingdom, Saudi Arabia, Canada, Greece, Sweden, Brazil, Italy
Arthrogen
Centre for Human Drug Research (CHDR)

Unknown

A Single Dose Clinical Trial to Study the Safety of ART-I02 in Patients With Arthritis

Arthritis, Rheumatoid | Osteo Arthritis

Netherlands
Beijing Chao Yang Hospital
Tongji University; The No.180 Hospital of People's Liberation Army

Completed

Liver Transplantation With ADV-TK Gene Therapy Improves Survival in Patients With Advanced Hepatocellular Carcinoma

Liver Transplantation | Hepatocellular Carcinoma

China
Arthrogen

Unknown

ART-I02 in Patients With Rheumatoid Arthritis With Inflamed Wrists

Rheumatoid Arthritis

Canada
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
CSL Behring

Completed

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Hemophilia B

Germany, Denmark, Netherlands
Leiden University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development; Horizon...

Recruiting

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID (RAG1-SCID)

Severe Combined Immunodeficiency Due to RAG1 Deficiency

Spain, Netherlands, Italy, Poland, Australia, Turkey, United Kingdom
World Federation of Hemophilia

Not yet recruiting

The World Federation of Hemophilia Gene Therapy Registry (WFH GTR)

Hemophilia
Megan Waldrop
Alcyone Therapeutics

Enrolling by invitation

Gene Therapy for IGHMBP2-Related Diseases

SMARD1 | CMT2S

United States
National Taiwan University Hospital

Completed

Neurological Effects of Goal-directed Fluid Therapy in Beach Chair Position Shoulder Surgery

Postoperative Cognitive Dysfunction | Anesthesia

Taiwan
Shenzhen SiBiono GeneTech Co.,Ltd

Unknown

p53 Gene Therapy in Treatment of Diabetes Concurrent With Hepatocellular Carcinoma

HCC | Diabetes

China
University College, London
Medical Research Council

Not yet recruiting

Lentiviral Gene Therapy for Epilepsy

Drug Resistant Epilepsy
American Gene Technologies International Inc.

Enrolling by invitation

Long-term Follow-up of Study Participant Treated With Lentiviral-Based Genetically Modified Autologous Cell Product ,AGT103-T

HIV

United States
Eunice Kennedy Shriver National Institute of Child...
University of Minnesota

Completed

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

Mucopolysaccharidosis II

United States
Shenzhen SiBiono GeneTech Co.,Ltd

Unknown

rAd-p53 Gene Therapy for Advanced Malignant Thyroid Tumors

Advanced Malignant Thyroid Tumors

China
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
Taipei Medical University WanFang Hospital

Withdrawn

Comparing the Prognosis of Lactate-directed and Goal-directed Therapy in Hyperthermic Intraperitoneal Chemotherapy.

Organ Failure, Multiple
Children's Hospital of Chongqing Medical University

Recruiting

Gene Therapy for X Linked Severe Combined Immunodeficiency

Gene Therapy

China
Nanoscope Therapeutics Inc.

Completed

Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease (STARLIGHT)

Stargardt Disease

United States
Genethon

Active, not recruiting

Gene Therapy for X-linked Chronic Granulomatous Disease

X-Linked Chronic Granulomatous Disease

France
Hubert Serve, Prof., MD

Unknown

Gene Therapy for X-CGD

X-linked Chronic Granulomatous Disease

Germany
University of Calgary
University of Alberta

Not yet recruiting

Goal-Directed Therapy Following Cardiac Surgery

Surgery
Medical University of South Carolina

Terminated

Does Perioperative Goal Directed Therapy Using Flotrac Improve Outcomes in Esophagectomy Patients

Esophageal Cancer

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
Genethon

Active, not recruiting

Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) (CGD)

X-Linked Chronic Granulomatous Disease

United Kingdom
Shenzhen Geno-Immune Medical Institute

Unknown

Lentiviral Gene Therapy for X-ALD

X-linked Adrenoleukodystrophy

China
Nanfang Hospital of Southern Medical University
Peking University People's Hospital; Guangzhou First People's Hospital; Sun Yat-Sen... and other collaborators

Completed

MRD-directed Therapy for Low-risk and Intermediate-risk AML.

Acute Myeloid Leukemia | Minimal Residual Disease

China
Eastern Cooperative Oncology Group
National Cancer Institute (NCI)

Completed

Gene Therapy Plus Radiation Therapy in Treating Patients With Non-Small Cell Lung Cancer

Lung Cancer

United States
University of California, Irvine

Completed

Goal Directed Fluid Management Based on Non-invasive Monitoring

Postoperative Complication

United States
ResMed

Terminated

Adherence to ASV Therapy in Heart Failure With Preserved Ejection Fraction Feasibility Study (CAT-PEF)

Sleep Apnea | Heart Failure With Normal Ejection Fraction

United States, Germany
Nanoscope Therapeutics Inc.

Enrolling by invitation

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study (SUSTAIN)

Stargardt Disease

United States
Wuhan Union Hospital, China
Wu Jieping Medical Foundation

Terminated

Peripheral Oxygen Saturation (SpO2) Directed Oxygen Therapy (POSDOT)

Critical Illness

China
Brno University Hospital

Completed

Goal-directed Therapy in Neurosurgery.

Surgery--Complications

Czechia
Erasmus Medical Center
Albert Schweitzer Hospital; Sint Franciscus Gasthuis; Medical Center Rijnmond... and other collaborators

Completed

Early Lactate-Directed Therapy in the Intensive Care Unit (ICU)

Hyperlactatemia | Tissue Hypoxia

Netherlands
University of Maryland, Baltimore

Not yet recruiting

Early Biomarker Kidney Injury Assessment After Acumen Directed Fluid Management in Cardiac Surgery (BE-KIND)

Acute Kidney Injury | Renal Injury
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

Search clinical trials for: AAV directed hLDLR gene therapy

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