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Clinical Trials About "Gene Therapy Clinical Study in Adult PKU" RSS

20:13 EDT 20th June 2019 | BioPortfolio

We list hundreds of Clinical Trials about "Gene Therapy Clinical Study in Adult PKU" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

More Information about "Gene Therapy Clinical Study in Adult PKU" on BioPortfolio

We have published hundreds of Gene Therapy Clinical Study in Adult PKU news stories on BioPortfolio along with dozens of Gene Therapy Clinical Study in Adult PKU Clinical Trials and PubMed Articles about Gene Therapy Clinical Study in Adult PKU for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Gene Therapy Clinical Study in Adult PKU Companies in our database. You can also find out about relevant Gene Therapy Clinical Study in Adult PKU Drugs and Medications on this site too.

Showing "Gene Therapy Clinical Study Adult" Clinical Trials 1–25 of 30,000+

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Gene Therapy Clinical Study in Adult PKU

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.


A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead ...

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.


Feasibility of Individualized Therapy for Recurrent GBM

The current study will test the ability and likelihood of successfully implementing individualized combination treatment recommendations for adult patients with surgically-resectable recurrent glioblastoma in a timely fashion. Collected tumor tissue and blood will be examined using a new diagnostic testing called UCSF 500 Cancer Gene Panel which is done at the UCSF Clinical Cancer Genomics Laboratory. The UCSF 500 Cancer Gene Panel will help identify genetic changes in the DNA ...

Gene Mutations in Patients With Advanced Prostate Cancer That Is Not Responsive to Hormone Therapy

RATIONALE: Gene mutations may make prostate cancer cells unable to attach to androgens. This may permit the growth of prostate cancer. Gene testing may improve the identification of patients with advanced prostate cancer. PURPOSE: Clinical trial to study the androgen receptor gene in patients with prostate cancer that is not responsive to hormone therapy.

Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1

Phase 3, open-label, single-arm, single-dose, trial of AVXS-101 (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). Up to 30 patients < 6 months (< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.

Clinical Study of Gene-Eden-VIR/Novirin

This study measured the changes in health-related complaints by analyzing charts of individuals, who are infected with a latent virus, who have used Gene-Eden-VIR/Novirin.

Choroideremia Gene Therapy Clinical Trial

Phase II gene therapy study, involving a total of 6 male patients with choroideremia. The study will be conducted at the Bascom Palmer Eye Institute, University of Miami. Patients will be required to attend a total of 11 study visits over a 24 month period with an additional 3 year follow-up.

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.

Clinical Characterization on PDE6A-related Retinitis Pigmentosa in Preparation to a Gene Therapy Trial

Mutations in the PDE6A gene - encoding the -subunit of the rod cGMP-phosphodiesterase - account for 1% of autosomal recessive retinitis pigmentosa (arRP) through impaired regulation of cGMP levels in the rod outer segment. This study aims for a detailed clinical characterization of patients with PDE6A mutations in preparation of a clinical gene replacement study (phase I/II safety trial).

The Influence of Gene Polymorphism on Clinical Outcomes in Patients Undergoing PCI

Dual antiplatelet therapy with aspirin and thienopyridines is an essential treatment in patients undergoing percutaneous coronary intervention (PCI). However, despite intensified antiplatelet treatment, some of the patients undergoing PCI develop thrombotic stent occlusion, suggesting incomplete platelet inhibition due to thienopyridine resistance. Some patients develop bleeding event because of the improper dosage and covariation. This observational study is designed for clari...

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.

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.

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.

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.

THOR - Tübingen Choroideremia Gene Therapy Trial

An open label monocentric phase II trial in adult males with a clinical phenotype of choroideremia and a confirmed molecular diagnosis of a null mutation in the gene encoding REP1 to assess the anatomical and functional outcomes, as well as the safety of a single subretinal injection of rAAV2.REP1 in 6 subjects with genetically confirmed choroideremia for up to 24 months.

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.

Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children

Study the clinical manifestations and gene mutation of Taiwanese ALD patients

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.

Gene Therapy for Alzheimer's Disease Clinical Trial

This Phase I clinical trial is the first step in testing gene therapy. This study is called a "Safety/Toxicity" study by the Food and Drug Administration, and primarily aims to determine whether the experimental protocol is safe for humans. It will determine whether the study procedure causes side effects in humans, and may also give us a preliminary sense of whether this will be effective in combating Alzheimer's disease in humans.

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.


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