Search clinical trials for: Genetic Therapy

Total 68528 results

eyeDNA Therapeutics

Recruiting

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

Retinitis Pigmentosa

France
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
University College, London

Recruiting

Targeted Stem Cells Expressing TRAIL as a Therapy for Lung Cancer (TACTICAL)

Adenocarcinoma of Lung

United Kingdom
City of Hope Medical Center

Recruiting

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

Sickle Cell Disease

United States
Crystal Mackall, MD

Recruiting

Phase I Dose Escalation Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory B Cell Malignancies

Recurrent Adult Acute Lymphoblastic Leukemia | Recurrent Childhood Acute Lymphoblastic Leukemia | B Acute Lymphoblastic Leukemia | Philadelphia Chromosome Positive | Refractory Acute Lymphoblastic Leukemia | Minimal Residual Disease | CD19 Positive

United States
Cambridge University Hospitals NHS Foundation Trust
Medical Research Council; National Institute for Health Research, United Kingdom

Recruiting

Gaucherite - A Study to Stratify Gaucher Disease (Gaucherite)

Gaucher Disease, Type III | Gaucher Disease, Type I

United Kingdom
Yonsei University

Recruiting

Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy

Fabry Disease

Korea, Republic of
National Cancer Institute (NCI)

Recruiting

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

Malignant Glioma | Rhabdoid Tumor | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditions

United States, Puerto Rico, Australia, Canada
LoneStar Heart, Inc.

Not yet recruiting

A Pivotal Trial to Establish the Efficacy and Safety of Algisyl in Patients With Moderate to Severe Heart Failure (AUGMENT-HFII)

Heart Failure | Dilated Cardiomyopathy | Heart Failure With Reduced Ejection Fraction
IRCCS San Raffaele
Fondazione Telethon

Recruiting

Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD (XCGD-MOBI)

Chronic Granulomatous Disease X-linked (X-CGD)

Italy
Children's Oncology Group
National Cancer Institute (NCI); EsPhALL Network/ BFM Study Group

Recruiting

Imatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia | B Acute Lymphoblastic Leukemia | T Acute Lymphoblastic Leukemia | Mixed Phenotype Acute Leukemia

United States, Canada, Puerto Rico, Australia, Finland, New Zealand, Saudi Arabia, Germany, Austria, Belgium, Chile, Czechia, France, Italy, Netherlands, Sweden, Switzerland, Hong Kong, Israel
University College, London
Medical Research Council

Recruiting

Gene Therapy for Haemophilia A. (GO-8)

Hemophilia A

United States, United Kingdom
Beth Israel Deaconess Medical Center

Recruiting

Gait Analysis in Neurological Disease

Parkinson's Disease | Parkinsonian Disorders | Multiple System Atrophy | Corticobasal Degeneration | Progressive Supranuclear Palsy | Huntington Disease | Atypical Parkinson Disease | Gait, Frontal

United States
Geneticure, LLC
University of Minnesota; Fairview Health Services

Not yet recruiting

Investigating the Use of Genetics to Guide Pharmacologic Therapy for Hypertension (PGEN for HTN)

Hypertension

United States
Loyola University
National Cancer Institute (NCI)

Recruiting

Adoptive T Cell Immunotherapy for Advanced Melanoma Using Engineered Lymphocytes

Melanoma

United States
Krzysztof Bankiewicz
National Institute of Neurological Disorders and Stroke (NINDS); University...

Recruiting

A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients (AADC)

AADC Deficiency

United States
National Eye Institute (NEI)

Recruiting

Clinical and Molecular Studies in Families With Inherited Eye Disease

Inherited Eye Disease

United States, United Kingdom, China, Pakistan, Philippines, Ukraine, India, Italy
Donald Basel

Recruiting

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NEUROFIBROMATOSIS 1

United States
Mayo Clinic
National Cancer Institute (NCI)

Recruiting

Vaccine Therapy in Treating Patients With Malignant Peripheral Nerve Sheath Tumor That is Recurrent or Cannot Be Removed by Surgery

Neurofibromatosis Type 1 | Metastatic Malignant Peripheral Nerve Sheath Tumor | Recurrent Malignant Peripheral Nerve Sheath Tumor

United States
Shenzhen Second People's Hospital
Guangzhou Women and Children's Medical Center; Shenzhen University

Recruiting

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Adrenoleukodystrophy | Metachromatic Leukodystrophy

China
Yonsei University

Recruiting

Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)

Fabry's Disease

Korea, Republic of
Sun Yat-sen University
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University; Zhujiang Hospital; Guangzhou...

Not yet recruiting

Clinical Trials of Continuous Oxygen Therapy Combined With Blue Light Deprivation in the Treatment of Retinitis Pigmentosa

Retinitis Pigmentosa

China
Emory University
National Center for Complementary and Integrative Health (NCCIH); Children's...

Recruiting

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease (R34 pK/PD)

Sickle Cell Disease

United States
University Medical Centre Ljubljana

Recruiting

Registry of Cell Therapy in Non-Ischemic Dilated Cardiomyopathy (RECORD)

Cardiomyopathy, Dilated

Slovenia
University of California, Los Angeles
National Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital; California Institute for Regenerative Medicine (CIRM) and other collaborators

Recruiting

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Granulomatous Disease, Chronic, X-linked

United States
Sun Yat-sen University

Recruiting

Doxycycline Treatment in Mild Thyroid-Associated Ophthalmopathy

Immune System Diseases | Autoimmune Diseases | Endocrine System Diseases | Thyroid Diseases | Eye Diseases, Hereditary | Graves Ophthalmopathy | Graves Disease | Hyperthyroidism | Thyroid-associated Ophthalmopathy

China
National Cancer Institute (NCI)

Recruiting

Genetic Testing in Screening Patients With Stage IB-IIIA Non-small Cell Lung Cancer That Has Been or Will Be Removed by Surgery (The ALCHEMIST Screening Trial)

Stage IIA Lung Cancer AJCC v8 | Stage IIB Lung Cancer AJCC v8 | Stage IIIA Lung Cancer AJCC v8 | Stage IIIB Lung Cancer AJCC v8 | Stage IB Lung Non-Small Cell Carcinoma AJCC v7 | Stage II Lung Non-Small Cell Cancer AJCC v7 | Stage IIIA Lung Non-Small Cell Cancer AJCC v7

United States, Guam, Puerto Rico
Johns Hopkins University
National Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...

Recruiting

The Cancer of the Pancreas Screening-5 CAPS5)Study (CAPS5)

Lynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation Carrier

United States
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Molecular Testing for the MD Anderson Cancer Center Personalized Cancer Therapy Program

Glioma | Hematopoietic and Lymphoid Cell Neoplasm | Malignant Solid Neoplasm | Melanoma | Sarcoma

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Ponatinib Hydrochloride as Second Line Therapy in Treating Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib Mesylate, Dasatinib, or Nilotinib

Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Philadelphia Chromosome Positive, BCR-ABL1 Positive Chronic Myelogenous Leukemia

United States
AHS Cancer Control Alberta

Recruiting

A Study of Genetic Based Chemotherapy Dosing for Breast Cancer Patients

Breast Neoplasms

Canada
Duke University
Genzyme, a Sanofi Company

Recruiting

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Pompe Disease

United States
University of Illinois at Chicago

Recruiting

Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

Sickle Cell Disease

United States
Center for International Blood and Marrow Transplant...

Recruiting

CIBMTR Research Database

Solid Tumors | Gene Therapy | Allogeneic Stem Cell Transplantation | Autologous Stem Cell Transplantation | Blood Cancers | Non-malignant Disease | CAR-T

United States
St. Jude Children's Research Hospital

Recruiting

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

Mucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, Hematologic

United States
National Cancer Institute (NCI)

Recruiting

Follow-Up Study of Subjects Previously Enrolled in Poxviral Vector Gene Transfer Studies

Breast Cancer | Lung Cancer | Prostate Cancer | Colon Cancer | Liver Cancer

United States
National Cancer Institute (NCI)

Recruiting

Evaluation for NCI Surgery Branch Clinical Research Protocols

Melanoma | Colorectal Cancer | Lung Cancer | Bladder Cancer | Synovial Cell Cancer

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

Enrolling by invitation

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Chronic Granulomatous Disease | p47 | Autosomal Recessive

United States
PTC Therapeutics

Active, not recruiting

A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants

AADC Deficiency

United States, Israel, Taiwan
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

Belgium, Canada, United States, Israel, United Kingdom, Denmark, France, Italy, Netherlands, Switzerland, Spain
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

United States, Canada, Israel, United Kingdom, Denmark, France, Belgium, Italy, Netherlands, Switzerland, Spain
David Williams

Active, not recruiting

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome

United States
Kenneth K. Tanabe, MD
National Cancer Institute (NCI)

Terminated

rRp450-Phase I Trial in Liver Metastases and Primary Liver Tumors

Liver Metastases | Primary Liver Cancers

United States
Pfizer

Active, not recruiting

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

United States, Spain, Belgium, Korea, Republic of, Taiwan, Canada, Australia, Germany, Japan, France, Italy, United Kingdom, Russian Federation, Israel, Switzerland
AbbVie
AbbVie

Enrolling by invitation

Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD

Gene Therapy | AMD | nAMD | Wet AMD | Neovascular Age-Related Macular Degeneration (nAMD) | wAMD

United States
Pfizer

Active, not recruiting

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Hemophilia A

United States
Alexion Pharmaceuticals, Inc.

Terminated

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

Wilson Disease

United States, Spain, Taiwan, France, Czechia, Hungary, Turkey, United Kingdom, Austria, Germany, Israel, Serbia, Australia, Japan, Denmark, Korea, Republic of, New Zealand, Canada, Russian Federation, Poland, Singapore, Hong Kong
AbbVie
AbbVie

Enrolling by invitation

Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy (RGX-314 SRLTFU)

Wet Macular Degeneration | Neovascular Age-related Macular Degeneration

United States
Amgen

Active, not recruiting

A Study Evaluating Rocatinlimab in Moderate-to-severe Atopic Dermatitis (ROCKET-IGNITE) (ROCKET-Ignite)

Atopic Dermatitis

United States, China, Spain, Korea, Republic of, Taiwan, Greece, Slovakia, Germany, Japan, Italy, Hungary, Croatia, Brazil, Poland, Canada, Portugal, Czechia, Argentina, Latvia, Netherlands, Puerto Rico

Search clinical trials for: Genetic Therapy

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