Chronic Granulomatous Disease clinical trials at University of California Health
3 in progress, 2 open to eligible people
open to eligible males ages 23 months and up
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other microorganisms. Chronic granulomatous inflammation may compromise vital organs and account for additional morbidity. CGD is thought to affect approximately 1 in 200,000 persons, although the real incidence might be higher due to under-diagnosis of milder phenotypes. The first gene therapy approaches in X-CGD have shown that effective gene therapy requires bone-marrow (BM) conditioning with chemotherapy to make space for the gene-modified cells to engraft. These studies demonstrated that transplantation of gene modified stem cells led to production of white blood cells that could clear existing infections. However, some trials using mouse-derived retroviral vectors were complicated by the development of myelodysplasia and leukemia-like growth of blood cells. This trial will evaluate a new lentiviral vector that may be able to correct the defect, but have much lower risk for the complication. This study is a two-part, prospective non-controlled, non-randomized Phase I/II clinical trial to assess the safety, feasibility and efficacy of cellular gene therapy in patients with chronic granulomatous disease using transplantation of autologous bone marrow CD34+ cells transduced ex vivo by the G1XCGD lentiviral vector containing the human CGD gene. Primary objectives include evaluation of safety and evaluation of efficacy by biochemical and functional reconstitution in progeny of engrafted cells and stability at 12 months. Secondary objectives include evaluation of clinical efficacy, longitudinal evaluation of clinical effect in terms of augmented immunity against bacterial and fungal infection, transduction of CD34+ hematopoietic cells from X-CGD patients by ex vivo lentivirus-mediated gene transfer, and evaluation of engraftment kinetics and stability. Approximately 3-6 patients will be treated per site with a goal of 16 total patients to be treated with G1XCGD lentiviral vector.
at UCLA UCSF
open to eligible people ages 18 years and up
Background: - There are very few documents to help young adults living with advanced cancer discuss their concerns and end-of-life preferences. A new document, Voicing My CHOiCES, allows young adults to explain what kind of care they would want if they became unable to communicate or make medical decisions on their own. Researchers want to study if this document is helpful. Objective: - To study if Voicing My CHOiCES can reduce anxiety, improve sense of support, and improve communication about advanced care planning. Eligibility: - Adults 18 to 39 years old being treated for cancer. Design: - Participants will answer questions about their age, gender, employment, religion, health, and marital status. They will also complete several brief questionnaires: 1. General Anxiety Short Form 2. Peace, Equanimity and Acceptance in the Cancer Experience 3. Functional Assessment of Social Support 4. Quality of Communication 5. Prior Communication about Advanced Care Planning - Then a health care professional will introduce Voicing My CHOiCES . Participants will review the document and comment on parts they find relevant. They will also say if any important items are missing. Participants will complete 3 pages of the document with the assistance of a health care provider. They will be asked for positive and negative observations. - The second stage of the study will take place about 1 month later. Participants will repeat the brief questionnaires listed above. They will be asked if they shared any of the preferences they described when completing the 3 pages of Voicing My CHOiCES during visit 1 with a family member, friend, or health care provider. Research staff will ask the participant for permission to contact the people they spoke with in order to learn whether their conversations about the document were helpful. They will ask for feedback on how to make Voicing My CHOiCES more helpful.
at UC Irvine
Sorry, accepting new patients by invitation only
Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation (BMT) has been shown to be curative. However the risks of transplantation are high and not all patients with CGD may need to undergo this high risk procedure. This study will determine the long term medical condition and daily functioning of participants with CGD after a transplant and if possible, compare these results to participants who do not undergo a transplant.
at UCLA UCSF