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Clinical Trials About "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" RSS

18:56 EDT 17th September 2019 | BioPortfolio

We list hundreds of Clinical Trials about "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

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Showing "Regenxbio Stellar Gene Therapy Innovator With Multiple Upsides" Clinical Trials 1–25 of 21,000+

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Follow-Up of Patients With Breast Cancer or Multiple Myeloma Previously Treated on a Gene Therapy Clinical Trial

RATIONALE: Studying the long-term effects of gene therapy in patients with breast cancer or multiple myeloma over time may help doctors plan better treatment and follow-up. PURPOSE: This natural history study is collecting health information from patients with breast cancer or multiple myeloma previously treated on a gene therapy clinical trial.


Descriptive Analysis of G-CSF Use in Patients With Breast Cancer, Lung Cancer, or Lymphoma Treated

Purpose: With the existing recombinant human granulocyte colony-stimulating factors (G-CSFs) patents expiring and the FDA approval of new biosimilar and innovator biologics, patients being treated with Grade III and IV myelosuppressive chemotherapy regimens will have additional therapeutic options. This observational study will describe the patient characteristics of new users of G-CSFs. It will describe in the treatment cohorts a primary outcome of hospitalizations for ...

Evaluating Multiple Sclerosis Patients ShOWing A GEnomic Signature of Therapy Response

To develop a test to characterize and monitor Multiple Sclerosis (MS) disease status and therapy response from a participant's home by analyzing the gene expression from participant self-collected blood samples using a novel fingerstick collection kit.


A Trial of CHOP-R Therapy, With or Without Acalabrutinib, in Patients With Newly Diagnosed Richter's Syndrome

The STELLAR trial will assess the effect of acalabrutinib taken in combination with CHOP-R compared to taking CHOP-R alone in patients with newly diagnosed Richter's Syndrome (RS). It will also be a platform to test other new drugs that show potential for treating RS. Chronic lymphocytic Leukaemia (CLL) is the most common blood cancer in adults, usually in their 70s or older. In a few patients, CLL can transform from a slow-growing cancer into an aggressive lymphoma called Rich...

RGX-111 Gene Therapy in Patients With MPS I

RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

Lentiviral Gene Therapy for X-ALD

This is a Phase I/II clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Gene Therapy in Treating Patients With Cancer of The Liver

RATIONALE: Inserting the p53 gene into a person's tumor may improve the body's ability to fight liver cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy with the p53 gene in treating patients who have cancer of the liver that cannot be surgically removed.

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

OBJECTIVES: I. Develop an approach for treating patients with homozygous familial hypercholesterolemia using gene therapy with autologous hepatocytes transduced with a normal low-density lipoprotein receptor gene.

RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a safety and dose ranging study to determine whether RGX-121 is safe and tolerated by patients with MPS II.

Bioequivalence Study of Clopidogrel 75 mg in Two Tablet Formulations Relative to Reference Tablet in Healthy Subjects

Clopidogrel is a potent anti-thrombotic drug that inhibits adenosine diphosphate (ADP)-induced platelet aggregation. This is an open-label, randomized, single dose, three-way cross over, six sequence study to investigate the relative bioavailability of two 75 milligrams (mg) clopidogrel tablet formulations (clopidogrel SB224326 test formulation 1 [Clop F1] and clopidogrel SB224326 test formulation 2 [Clop F2]) compared with the reference product (innovator) in healthy human sub...

Comparative Immunogenicity Study of Multiple Doses of Proposed Pegfilgrastim Biosimilar, INTP5 of Intas Pharmaceuticals Ltd., India Against Neulasta of Amgen Inc., USA in Healthy, Adult Human Subjects.

The study was an assessor-blind, balanced, parallel, randomized, two-treatment, comparative immunogenicity study of multiple doses of subcutaneous (SC) Pegfilgrastim injection (6 mg/0.6 mL; Intas Pharmaceuticals Ltd. proposed biosimilar INTP5 compared to innovator product, US-Neulasta) in healthy, adult, human subjects under fed conditions.

Gene Therapy of Beta Thalathemia Using a Self-inactivating Lentiviral Vector

This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s). The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.

Gene Therapy in Treating Patients With Recurrent Malignant Gliomas

RATIONALE: Inserting the gene for adenovirus p53 into a person's tumor may improve the body's ability to fight cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy in treating patients who have recurrent malignant gliomas.

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

RATIONALE: Inserting the gene for p53 into a person's cancer cells may improve the body's ability to fight cancer. Radiation therapy uses high-energy x-rays to damage tumor cells. PURPOSE: Phase I trial to study the effectiveness of gene therapy plus radiation therapy in treating patients who have non-small cell lung cancer.

Gene Therapy for SCID-X1 Using a Self Lentiviral Vector

This is a Phase I/II clinical trial of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a self lentiviral vector to replace the defective genes with good genes. The primary objectives are to evaluate the safety and efficacy of the ex vivo gene therapy clinical protocol.

Gene Therapy Plus Chemotherapy in Treating Patients With Breast Cancer

RATIONALE: Inserting the p53 gene into a person's cancer cells may improve the body's ability to fight cancer or make the cancer more sensitive to chemotherapy. Combining chemotherapy with gene therapy may kill more tumor cells. PURPOSE: Phase I trial to study the effectiveness of gene therapy plus chemotherapy in treating patients who have breast cancer.

Lentiviral FIX Gene Therapy

This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.

Gene Therapy for WAS

This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.

Multiple CAR-T Cell Therapy Targeting AML

The purpose of this clinical trial is to assess the feasibility, safety and efficacy of multiple CAR T-cell therapy which combines CAR T cells against CLL-1 with CAR T cells targeting CD123 or CD33 in patients with relapsed and refractory AML. The study also aims to learn more about the function of CAR T cells and their persistency in AML patients.

Combination Therapy Using Cellcept and Rebif in RRMS

The purpose of this trial is to examine the benefits of early combination of CellCept® with Rebif® in long-term management of patients with multiple sclerosis. Quantitation of mRNA for MxA gene from ex-vivo lymphocytes obtained from patients receiving both drugs or interferon alone will be used to gauge the usefulness of this combination therapy. In addition we will examine the safety of combination of mycophenolate mofetil and interferon beta 1a in treatment of multiple sc...

Gene Therapy in Treating Patients With Advanced Bladder Cancer

RATIONALE: Inserting the p53 gene into a person's bladder cancer cells may improve the body's ability to fight cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy in treating patients with advanced bladder cancer.

Gene Therapy to Improve Wound Healing in Patients With Diabetes

Patients with diabetes may develop chronic wounds that respond poorly to treatment. Gene therapy with the platelet-derived growth factor-B gene has been shown to help with the healing of chronic wounds. This study will evaluate a new way to deliver the gene to the wound tissue.

Lentiviral Gene Therapy for CGD

This is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.

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

Previous rather poor results in liver transplantation (LT) of patients with advanced hepatocellular carcinoma (HCC) have made the application of LT very limited in treatment of HCC. The advantages of ADV-TK gene therapy highlight its potentiality as adjuvant treatment for HCC patients after LT. We reported here the improved outcome of LT with combined treatment of ADV-TK gene therapy in patients with intermediate or advanced HCC.

UARK 2006-66, Total Therapy 3B: An Extension of UARK 2003-33 Total Therapy

With this study - Total Therapy IIIB - researchers are extending the findings of Total Therapy III based what they have learned from the first two studies (Total Therapy I and II), with new research strategies designed to explore why chromosome abnormalities found in persons with multiple myeloma affect the outcome of drug therapy used in this disease."


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