Search clinical trials for: Genetic Vectors

Total 18631 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
National Cancer Institute (NCI)

Enrolling by invitation

Gene Therapy Follow up Protocol for Subjects Previously Enrolled in NCI Center for Immuno-Oncology Studies

Cervical Intraepithelial Neoplasia | Oropharyngeal Neoplasms | Squamous Intraepithelial Lesions of the Cervix | Cervical Neoplasms | Hematologic Malignacies

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
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
National Institute of Allergy and Infectious Diseases...

Completed

Safety and Effectiveness of Anti-HIV Vaccines in HIV-Negative Adults

HIV Infections

United States
National Institute of Allergy and Infectious Diseases...

Completed

A Study of the Effectiveness of an HIV Vaccine (ALVAC vCP205) to Boost Immune Functions in HIV-Negative Volunteers Who Have Already Received an HIV Vaccine

HIV Infections | HIV Seronegativity

United States
National Institute of Allergy and Infectious Diseases...

Completed

A Randomized Phase I Safety and Immunogenicity Trial of Live Recombinant Canarypox ALVAC-HIV vCP205 Delivered by Alternate Mucosal Routes in HIV-1 Uninfected Adult Volunteers

HIV Infections

United States
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
National Human Genome Research Institute (NHGRI)

Completed

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

Severe Combined Immunodeficiency Syndrome

United States
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
National Human Genome Research Institute (NHGRI)
National Institutes of Health Clinical Center (CC); National Cancer Institute... and other collaborators

Completed

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

ADA-SCID | Adenosine Deaminase Deficiency

United States
National Institute of Allergy and Infectious Diseases...
New York Presbyterian Hospital

Completed

Immune Therapies and Anti-HIV Therapy Withdrawal in Controlling Viral Load

HIV Infections

United States
Ribozyome

Completed

Gene Therapy in HIV-Positive Patients With Non-Hodgkin's Lymphoma

HIV Infections | Lymphoma, Non-Hodgkin

United States
SpliSense Ltd.

Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

Cystic Fibrosis

United States
University of North Carolina, Chapel Hill
North Carolina Translational and Clinical Sciences Institute

Not yet recruiting

PeLear CCC: Proyecto Latino Contra Cancer Colorectal (PeLear CCC)

Colorectal Cancer | Rectal Cancer | Rectal Neoplasms | Colon Cancer | Colon Adenocarcinoma | Colon Rectal Cancer | Hereditary Nonpolyposis Colon Cancer

United States
University of Cologne
The Clinical Trials Centre Cologne; ESCRS (European Society of Cataract and...

Not yet recruiting

Trial Comparing Cataract Surgery With Triple-DMEK in Patients With Cataract and Fuchs Endothelial Corneal Dystrophy

Cataract Surgery | Cataract and Fuchs Endothelial Corneal Dystrophy

Denmark, Germany, Netherlands, Spain
University of Mons

Recruiting

Quantitative Assessment of Orofacial Muscle Function in FSHD

Facioscapulohumeral Muscular Dystrophy

Belgium
Amryt Pharma
Chiesi Farmaceutici S.p.A.

Not yet recruiting

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez (FOSteR)

Epidermolysis Bullosa, Junctional | Epidermolysis Bullosa, Dystrophic
Takeda

Recruiting

A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine

Dravet Syndrome (DS) | Lennox-Gastaut Syndrome (LGS)

United Kingdom, Denmark
University of Southern California
National Cancer Institute (NCI)

Not yet recruiting

Access to Genetic Testing in Underserved Patients With Cancer

Malignant Solid Neoplasm | Breast Carcinoma | Metastatic Prostate Carcinoma | Ovarian Carcinoma | Triple-Negative Breast Carcinoma | Male Breast Carcinoma | Pancreatic Exocrine Neoplasm | Stage IVB Prostate Cancer American Joint Committee on Cancer v8

United States
Rocket Pharmaceuticals Inc.

Not yet recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
AbbVie

Not yet recruiting

An Observational Study to Assess Real-World Use of Upadacitinib Tablets in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis in China (REACH-AD)

Atopic Dermatitis
Leiden University Medical Center
The Dutch Brain Foundation (funding)

Not yet recruiting

Stimulating Amyloid Clearance in Cerebral Amyloid Angiopathy (Clear-Brain)

Cerebral Amyloid Angiopathy
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
Gattaca Genomics

Recruiting

Embryo Assessment Utilizing Timelapse Imaging in Conjunction With Preimplantation Genetic Testing for Aneuploidy With Next Generation Sequencing

Infertility | Aneuploidy | Infertility, Female | Infertility, Male | IVF

United States
Assistance Publique - Hôpitaux de Paris
Institut Pasteur

Recruiting

Physiology and Pathologies Linked to Human Splenic Function : Direct and Ex-vivo Perfusion Explorations (SPLEENVIVO)

Splenectomy

France
University of Haifa
Bnai Zion Medical Center; Clalit Health Services, Haifa and West Galilee

Not yet recruiting

Risk and Clinical Consequences of Low Count Monoclonal B-cell Lymphocytosis (LC MBL) (MBL RiskConseq)

Cardiovascular Diseases | Infections | Autoimmune Diseases | Parkinson Disease | Dementia | Alzheimer Disease | Chronic Lymphocytic Leukemia | Monoclonal B-Cell Lymphocytosis | Cancers
Instituto Nacional de Investigacao em Saude, Angola
FUNDAÇAO CALOUSTE GULBENKIAN; FUNDACIÓN BANCARIA CAIXA D'ESTALVIS I PENSIONS... and other collaborators

Recruiting

LEARNER- Low dosE AspiRiN prEterm tRial (Angola) (LEARNER)

Pregnancy Complications | Pregnancy Related | Sickle Cell Disease | Pre-Eclampsia

Angola
Sanofi
Regeneron Pharmaceuticals

Not yet recruiting

Study in Paediatric Patients With Atopic Dermatitis Treated With Dupilumab in Spain (PROADAP)

Atopic Dermatitis
Ospedale San Raffaele

Recruiting

Genetics in the Progression of Nephropathies

Hypertension | AKI | CKD | Hematologic Malignancy | Ckd-Mbd | ADPKD | Bone Marrow Transplant Complications | CKD (Chronic Kidney Disease) Stage 5D

Italy
AstraZeneca

Recruiting

Hereditary Transthyretin Amyloidosis Polyneuropathy in Patients With Carpal Tunnel Syndrome in Russia (LOCUS)

Carpal Tunnel Syndrome | Amyloidosis | Polyneuropathy | Hereditary Transthyretin Amyloidosis

Russian Federation
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Poitiers University Hospital

Recruiting

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD) (MUSIC-HD)

Huntington Disease

France
Jiangsu HengRui Medicine Co., Ltd.

Not yet recruiting

Clinical Trial of HR091506 Tablets in Treatment of Gout With Hyperuricemia in Adults

Gout With Hyperuricemia in Adults
Spinogenix
Avance Clinical Pty Ltd.

Not yet recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
Children's Hospital Los Angeles

Not yet recruiting

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Cystic Fibrosis | Constipation Chronic Idiopathic

United States
IRCCS Ospedale San Raffaele

Not yet recruiting

Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies

Rare Diseases | Corneal Neovascularization | Limbal Stem Cell Deficiency | Aniridia | Neurotrophic Keratopathy | Ocular Cicatricial Pemphigoid | Ocular Graft-versus-host Disease | EEC Syndrome
Ospedale San Raffaele
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Recruiting

Identification of New Biological Markers for the Progression of Mycobacterium Abscessus-induced Lung Disease in Cystic Fibrosis (BioMol-MA)

Non-Tuberculous Mycobacterial Pneumonia | Cystic Fibrosis Lung

Italy
All India Institute of Medical Sciences, Bhubaneswar

Not yet recruiting

Study on Efficacy of Add on Selenium in Mild-to-moderate Graves Ophthalmopathy

Thyroid Eye Disease | Graves Ophthalmopathy

India
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Avidity Biosciences, Inc.

Not yet recruiting

Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

Myotonic Dystrophy | Myotonic Disorders | Myotonia | Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy 1 | Myotonic Muscular Dystrophy | DM1 | Dystrophy Myotonic | Steinert Disease | Steinert
QED Therapeutics, Inc.

Recruiting

Prospective Clinical Assessment Study in Children With Hypochondroplasia (HCH)

Hypochondroplasia

United States, Australia, Norway, Portugal, Singapore, Spain

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