View clinical trials related to Gene Transfer.
Filter by:SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.
The purpose of the study is to evaluate the safety and efficacy of catheter mediated endocardial adenovirus-mediated vascular endothelial growth factor-D (AdVEGF-D) regenerative gene transfer in patients with refractory angina to whom revascularisation cannot be performed.
Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: - The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin. Nerves stop working normally in people with GAN. This causes problems with walking and sometimes with eating, breathing, and many other activities. GAN has no cure. Over time, GAN can shorten a person s life. Researchers want to see if a gene transfer treatment may help people with GAN. Objectives: - To see if a gene transfer is safe and shows potential to help people with GAN. Eligibility: - People age 3 and older with GAN. Design: - For 1 month following gene transfer participants must live full-time within 100 miles of the NIH. - Participants will be screened by phone and in person. They will take many tests. Some are listed below. Their medical records will be reviewed. Their caregivers may be contacted. - Participants will have a total of about 27 visits, weekly, monthly, and then yearly over 15 years. They will include many of the tests below. - Physical and nervous system exams. - Blood, urine, and stool samples. - Nerve, lung, heart, and eye tests. - Questionnaires. - MRI scans, nerve biopsies, and spinal taps. Participants will be sedated for some tests. - Speech, memory, muscle, and mobility tests. - Skin biopsy (small sample removed). - Participants will take many medicines. Some require intravenous lines. - Participants will get the gene transfer through an injection by spinal tap into their cerebrospinal fluid, which flows around the brain and spinal cord. The genes are packed in a modified virus that carries the genes to cells in their body. Participants safety is not guaranteed.
This study will provide follow-up evaluations of breast cancer or multiple myeloma patients who received gene therapy (gene transfer) as part of their participation in an NIH protocol. Gene therapy is a new technology, which may involve a permanent change in the patient s genetic code. Therefore, although the risk of long-term harmful effects of this therapy is very small, the Food and Drug Administration requires prolonged monitoring of patients health status. Patients previously enrolled in NIH protocols 96-C-0007, 93-C-0208, 92-C-0161, or 92-H-0057 will be followed under the current protocol. No further gene therapy will be provided in this study. Patients health status will be evaluated for an indefinite period of time, or as long as they are willing to be monitored. They will provide a blood sample once a year and will be interviewed about their health status twice a year for the first 5 years after gene therapy and once a year thereafter. These procedures are done to look for the development of any diseases such as cancer, neurological disorders, autoimmune or blood disorders that may be related to side effects of the gene transfer.