Clinical Trial Search Results

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as Machado-Joseph disease and SCA 6). The diseases are rare, slowly progressive, cause increasingly severe neurological difficulties and are variable across and within genotypes. The purpose of this research study is to bring together a group of experts in the field of SCA for the purpose of learning more about the disease.

The research questions are: 1. How does your disease progress over time? 2. What are the best ways to measure the progression? 3. Do some genes, other than the gene that is abnormal in your disease, have any effect on the way the disease behaves? This is a nationwide study and we expect that 800 patients will participate all over the USA. The participants will be in the study for an indeterminate period of time. Study visits will be done every 6 or 12 months depending on the participating site.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This clinical trial studies cancer survivors to identify those who are at increased risk of developing late-occurring complications after undergoing treatment for childhood cancer. A patient's genes may affect the risk of developing complications, such as congestive heart failure, avascular necrosis, stroke, and second cancer, years after undergoing cancer treatment. Genetic studies may help doctors identify survivors of childhood cancer who are more likely to develop late complications.

Background:

- Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. The NIH helped create a network of medical research centers, called the Undiagnosed Diseases Network (UDN), to provide care and answers for these individuals. Objectives: - To improve diagnosis and care for people with undiagnosed diseases. Eligibility: - People with undiagnosed diseases, and their relatives. Design: - Participants will travel to one of the UDN medical centers for a 5-day clinical and research visit. - As part of the visit, UDN healthcare providers may ask participants to have: - Clinically indicated tests and procedures performed including: - A physical exam - Blood and urine tests - A review of health and family history - X-rays and body scans - Surveys - Photographs of the face and body - A special diet to see if the body can handle the food without having a reaction, like vomiting - Video or voice recordings - Other tests and procedures to help reach a diagnosis - Research tests and procedures performed including: - A skin biopsy. For this, a small piece of skin will be taken. - Surveys - Other tests and procedures for research that may not be related to a diagnosis or treatment. - Most participants will be asked to give samples for genetic testing. - Participants may be contacted after their visit to discuss test results. They may also be contacted in the future for interviews and surveys. - Relatives of participants may be asked to give samples for genetic testing. They may be asked to have parts of their visit recorded and to have additional tests. They may also be contacted in the future for interviews and surveys. - Clinical and research information collected will be stored in a database. - Information and samples collected will be shared with others for research purposes.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

The purpose of this phase 1 study is to investigate a new approach in the treatment of cancer, i.e. giving gene-modified immune cells combined with gene-modified stem cells intravenously to reprogram the immune system to attempt to fight the cancer. The goal is to teach the immune system to recognize and kill cancer cells that have the NY-ESO-1 protein with sustained killing activity. The gene-modified cells studied in this experimental protocol are the patient s own white blood cells and stem cells from blood that will be modified in the laboratory using genetic techniques to express the NY-ESO-1 TCR against cancer cells. The gene modification of the white blood cells is an attempt to direct the patient s own immune cells to kill cancer cells. Since these immune cells usually only last for a few months in the body before they die, stem cells will be gene-modified so that the patient s body can attempt to create new immune cells that will kill cancer cells. Gene modification of cells involves the transfer of foreign genetic material (DNA) into a cell, in this case the patient s immune system cells and stem cells. This process will endow the recipient immune cells and descendants of the stem cells with the ability to eliminate cancer cells that express the cancer specific protein, NY-ESO-1. The specific receptor against cancer cells that will be transferred to the immune cells and stem cells is called NY-ESO-1 T cell receptor (or TCR). In this study, the gene-modified immune cells will be given in combination with the gene-modified stem cells to all patients to test the safety, toxicity and tumor responses of administering this combination. The study procedures will start with the collection of stem cells by administering medicines to mobilize the stem cells from the bone marrow and deposit them in the peripheral blood and using a medical device called a blood cell separator. A cell separator will also be used to collect white blood cells. These cells will be grown in the laboratory and then genetically modified with a virus vector (lentivirus vector for stem cells and retrovirus vector for lymphocytes), extensively modified to make sure that it is not infectious. The lentivirus and retrovirus vectors will allow expression of NY-ESO-1 TCR on the surface of cancer cells which directs the immune system to recognize cancer as foreign and kill the cancer cells. The lentivirus will also carry a second gene, Herpes Simplex Virus 1 (HSV1) sr39 thymidine kinase (TK) reporter gene, which will allow the progeny gene modified immune cells to be visualized when a PET scan is performed. The TCR gene modified cells will be tested to make sure that they are not contaminated with bacteria and other organisms, and that they express the appropriate TCR. The stem cells will be frozen prior to administration and the lymphocytes will be administered fr

This study has not yet been registered on ClinicalTrials.gov, which is currently a pre-requisite for display of detailed eligibility criteria.

• If you need assistance with ClinicalTrials.gov registration for an oncology study, please contact the Jonsson Comprehensive Cancer Center's Office of Regulatory Compliance.
• If you need assistance with ClinicalTrials.gov registration for a non-oncology study, please contact the Office of Regulatory Affairs.

This Pediatric MATCH screening and multi-sub-study phase II trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have progressed following at least one line of standard systemic therapy and/or for which no standard treatment exists that has been shown to prolong survival. Genetic tests look at the unique genetic material (genes) of patients' tumor cells. Patients with genetic changes or abnormalities (mutations) may benefit more from treatment which targets their tumor's particular genetic mutation, and may help doctors plan better treatment for patients with solid tumors or non-Hodgkin lymphomas.

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of GVHD, reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to introduce the gene into the patient's own blood stem cells is to engineer and use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings.

The investigators have recently discovered a gene that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and rejection. The goal is to test whether this approach is safe, and whether using gene therapy to change the expression of this particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.

AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).

Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period. Part B is a multiple-ascending dose design with 3 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.