Search clinical trials for: Genetic Vectors

Total 18754 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
Ain Shams University

Recruiting

Effect of Captopril on GLS in Duchenne Myodystrophy

Effect of Drug

Egypt
Royal Free Hospital NHS Foundation Trust
Brunel University; Sanius Health

Not yet recruiting

A Real-world Wearables Study in Fabry Disease.

Fabry Disease

United Kingdom
Astellas Gene Therapies

Not yet recruiting

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia

Friedreich Ataxia | Cardiomyopathy

United States
NMD Pharma A/S

Not yet recruiting

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (SYNAPSE-CMT)

Charcot-Marie-Tooth Disease
Institute of Hematology & Blood Diseases Hospital...

Not yet recruiting

Efficacy and Safety of Chemotherapy Combined With CAR-T Cells in Newly Diagnosed Adult Patients With Ph- B-ALL

Acute Lymphoblastic Leukemia, Adult | Philadelphia Chromosome Negative ALL

China
Bristol-Myers Squibb

Not yet recruiting

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

Healthy Volunteers | Anemia, Sickle Cell

United States
Connecta Therapeutics, S.L.
Adknoma Health Research; Ministerio de Ciencia e Innovación, Spain; Hospital...

Recruiting

CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction (FIH-CTH120)

Fragile X Syndrome

Spain
Children's Hospital of Fudan University

Recruiting

A Long-term Follow-up Study in Participants Who Received CS-101

Beta-Thalassemia

China
University of Exeter
Royal Devon University Healthcare NHS Foundation Trust

Not yet recruiting

Understanding Beta Cell Disorders Through the Study of Rare Genotypes (ENDURE) (ENDURE)

Diabetes Mellitus | Hyperinsulinism | Monogenic Diabetes

United Kingdom
Shanghai 6th People's Hospital

Recruiting

Calcium Folinate Treatment of Spastic Paraplegia 56 (CFT-SPG56)

Hereditary Spastic Paraplegia

China
Simcere Pharmaceutical Co., Ltd

Not yet recruiting

A Study to Evaluate the Efficacy and Safety of SIM0718 in Adult and Adolescent Patients With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis
Enterprise Therapeutics Ltd

Recruiting

A Two-Part Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of Repeat Doses of Inhaled ETD001 in People With Cystic Fibrosis

Cystic Fibrosis

Germany, France, United Kingdom
St. Jude Children's Research Hospital

Recruiting

Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

Sickle Cell Disease

United States
Children's Hospital of Fudan University

Not yet recruiting

Effects of an Emollient Containing Postbiotic Saccharomyces and Lactobacillus on Pediatric Atopic Dermatitis

Eczema | Atopic Dermatitis | Emollient
Huntington Study Group
Neurotargeting LLC; University of Rochester Center for Health + Technology; Modality...

Not yet recruiting

Longitudinal Endpoint Assessment of Disease Burden in HD (LEAD-HD)

Huntington Disease
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset (FOETEPISIGN)

Congenital Malformation | Rare Fetal Genetic Diseases

France
Luye Pharma Group Ltd.

Not yet recruiting

Effect of Food on the Pharmacokinetics of LPM3770164 Sustained-release Tablets

Tardive Dyskinesia | Huntington Disease

China
University Hospital Tuebingen

Recruiting

Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)

Spinocerebellar Ataxia Type 27B

Germany, Spain
Amur State Medical Academy

Recruiting

The Endovascular Plus GENe Therapy Versus Only EndoVascular Therapy for Severe Limb ischaemiA Trial

Severe Lower Limb Ischemia

Russian Federation
Medibiofarma S.L.

Recruiting

Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients

Huntington Disease

Spain
Ikaria Bioscience Pty Ltd

Not yet recruiting

Study of RN0361 in Adult Subjects

Hypertriglyceridemia | Familial Chylomicronemia

Australia
University of Cyprus

Recruiting

Unraveling the Genetic Basis of Nicotine Addiction for Novel Therapeutic Strategies (NicoGen)

Smoking Cessation | Epigenetics | Nicotine Addiction | Smoking Cessation Intervention | Molecular Biology | Genetic Epidemiology

Cyprus
Royal Marsden NHS Foundation Trust
Institute of Cancer Research, United Kingdom

Not yet recruiting

Prophylactic Prostatectomy for Prostate Cancer (PPPC) (PPPC)

Genetic Predisposition to Disease | Prostate Cancer | BRCA Mutation
Concerto Biosciences

Not yet recruiting

Topical ENS-002 for Atopic Dermatitis in Adults (EnSync)

Atopic Dermatitis | Atopic Dermatitis Eczema

United States
Kors van der Ent
European Union

Recruiting

Clinical Trial to Evaluate the Efficacy and Safety of Dirocaftor/Posenacaftor/Nesolicaftor in Adults With CF (CHOICES)

Cystic Fibrosis

Netherlands, Spain, France, Germany, Italy, United Kingdom, Portugal, Belgium, Sweden
Ascidian Therapeutics, Inc

Recruiting

Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR) (STELLAR)

Stargardt Disease | Stargardt Disease 1 | Juvenile Macular Degeneration | Cone Rod Dystrophy

United States
Guangdong Hengrui Pharmaceutical Co., Ltd

Not yet recruiting

Efficacy and Safety Study of SHR-1819 Injection in Adult Patients With Severe Atopic Dermatitis

Moderate to Severe Atopic Dermatitis

China
Peter Timoney
Inflammasome Therapeutics

Not yet recruiting

Evaluation of K9 in Subjects With Thyroid Eye Disease (TED)

Thyroid Eye Disease

United States
Ahram Canadian University

Recruiting

Inspiratory Muscle Training in Children With Beta Thalasemia

Beta Thalassemia Major

Egypt

Search clinical trials for: Genetic Vectors

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