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Displaying 1 - 20 of 55

Open Actively Recruiting

Genetic Studies in Inherited Ocular Disorders

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

Age Group: All
Contact: Lindsey Pyers
Open Actively Recruiting

Molecular Genetic Studies in Neurological Diseases

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

Age Group: All
Contact: Darice Wong
Open Actively Recruiting

Genetic Modifiers of Duchenne and Becker Muscular Dystrophy

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

Age Group: All
Contact: Emilie Douine
Open Actively Recruiting

Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

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.

Gender: All
Age Group: All
Contact: Aaron Fisher
Open Actively Recruiting

Genetic and Gene Expression Profiling of Small Cell Carcinoma of the Bladder

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

Phase: N/A
Age Group: Adults
Contact: Arnold Chin
Open Actively Recruiting

Integrating Clinical Aneurysm Quantitative Analysis with Aneurysm Genetic Risk Factors

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

Age Group: Adults
Contact: Arineh Aghakhani
Investigator:
Ai-Chi Chien
Open Actively Recruiting

Genetic Analysis in Identifying Late-Occurring Complications in Childhood Cancer Survivors

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.

Phase: N/A
Gender: All
Age Group: All
Contact: Roshani Tasker
Open Actively Recruiting

Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network

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.

Gender: All
Age Group: Children
Contact: Naghmeh Dorrani
Open Actively Recruiting

Genetic Characterization of Vascular Malformations and Vascular Tumors [English and Spanish Consent Forms]

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

Phase: N/A
Age Group: All
Contact: Phillip Monteleone
Open Actively Recruiting

Marijuana Smoking, Genetic Polymorphisms, and Their Interactions on Risk and Survival of Lung and Upper Aerodigestive Tract Cancers

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

Phase: N/A
Age Group: Adults
Contact: MINGYAN ZHANG
Investigator:
Zuo-Feng Zhang
Open Actively Recruiting

In vitro studies of gene therapy for genetic diseases by gene insertion into human bone marrow cells

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

Phase: N/A
Age Group: All
Contact: JACKSON CONNIE
Open Actively Recruiting

Immune and Genetic Characteristics in Patients with Systemic Lupus Erythematosus versus other Auto-Immune Diseases and Healthy Controls

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

Age Group: Adults
Contact: TIFFANY TRAN
Open Actively Recruiting

Collection of Bone Marrow, Blood, and Peripheral Blood Stem Cells from Patients with Genetic Diseases of the Immune and Blood Systems

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

Age Group: All
Contact: JACKSON CONNIE
Open Actively Recruiting

Los Angeles Case-Control Study of Genetic and Environmental Factors for Risk and Survival of Lung and Upper Aerodigestive Tract Cancers

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

Phase: N/A
Age Group: Adults
Contact: MINGYAN ZHANG
Investigator:
Zuo-Feng Zhang
Open Actively Recruiting

Genetically Engineered PBMC and PBSC Expressing NY-ESO-1 TCR After a Myeloablative Conditioning Regimen to Treat Patients With Advanced Cancer

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

Phase: Phase I
Primary Purpose: Treatment
Gender: All
Age Group: All
Contact: Elizabeth Seja
Open Actively Recruiting

Genomic Evaluation of Individuals and Family Members with Rare Diseases

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

Age Group: All
Contact: Emilie Douine
Open Actively 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)

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.

Phase: Phase II
Primary Purpose: Screening
Gender: All
Age Group: Children
Contact: Mikayla Marvis Henderson
Open Actively Recruiting

Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.

Gender: All
Age Group: Adults
Contact: Jennifer Huynh
Open Actively Recruiting

Tamibarotene Plus Venetoclax/Azacitidine in Participants With Newly Diagnosed AML

Tamibarotene is being studied as a treatment for participants with a type of leukemia called acute myeloid leukemia, or AML for short. Tamibarotene is being studied as a treatment for participants with AML whose cancer has a specific genetic abnormality characterized by the overexpression of the retinoic acid receptor alpha (RARA) gene. This genetic profile is found in about 3 of every 10 people with AML.

During the trial, tamibarotene will be given with 2 other drugs that are already used together to treat people who have AML and who cannot start treatment with standard chemotherapy.

Phase: Phase II
Primary Purpose: Treatment
Gender: All
Age Group: Adults
Contact: Bruck Habtemariam
Open Actively Recruiting

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

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.

Phase: Phase 1/Phase 2
Primary Purpose: Treatment
Gender: All
Contact: Emilie Douine