Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD)
Chronic Granulomatous DiseaseThe purpose of this study is to find a dose for a twice daily regimen for posaconazole (PSZ) as prophylactic treatment in children with CGD, based on the PSZ trough level.
Efficacy Study of Botulinum Toxin (BOTOX) Injections to Treat Vocal Fold Granulomas
Contact Ulcer of Vocal FoldsGranuloma of Vocal Cords3 moreVocal process granulomas are inflammatory masses caused in part by vocal trauma that arise in the posterior aspect of the vocal folds and result in throat pain, difficulty swallowing, hoarseness, and globus sensation. Antireflux therapy treats most granulomas, but many are recalcitrant to this therapy or take months to years to resolve. Botulinum toxin injection into the thyroarytenoid muscle has been effectively employed for recalcitrant granuloma, but causes significant voice loss, occasional difficulty swallowing and, in our clinical experience, is associated with significant recurrence. The investigators propose injecting another muscle in the larynx called the interarytenoid muscle with botulinum toxin type A to maintain the benefit of injection with less loss of voice. The investigators have shown the effectiveness of this treatment in a small, retrospective analysis.
Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease
Chronic Granulomatous Diseases (CGD) and Liver LesionsBackground: - Abscesses are a pocket of infection in an organ or tissue. Patients with a disease called chronic granulomatous disease (CGD) often develop these abscesses. CGD is an inherited disorder that affects how white blood cells function. Liver abscesses in people with CGD often require surgery to remove them and treat the infection. However, some people with CGD cannot have full surgery because it would be too risky. Researchers want to try a procedure called radiofrequency ablation (RFA) to treat these liver abscesses. RFA can usually be done without a major operation. This study will see if RFA is a safe and effective treatment for liver abscesses in patients with CGD. Objectives: - To see if RFA is a safe and effective treatment for CGD-related liver abscesses. Eligibility: - Individuals between 18 and 75 years of age with CGD who have liver abscesses that cannot be treated with surgery. Design: Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Imaging studies will be performed on the liver. Participants will have RFA for the abscesses. RFA is an image-guided technique that heats and destroys specific tissue, such as tumor tissue. It will target any abscesses on the liver. After the procedure, participants will stay in the hospital for monitoring before being released. Participants will have regular follow-up visits for up to 1 year after treatment. Blood and urine samples will be collected. Additional imaging studies will be performed.
Ultraviolet B (UVB) Light Therapy in the Treatment of Skin Conditions With Altered Dermal Matrix...
KeloidScleroderma4 moreThis research study will evaluate the effectiveness of high dose UVB light therapy in the treatment of keloid (or hypertrophic scar), scleroderma, acne keloidalis nuchae, old burn scars, granuloma annulare or related conditions.
Comparing Xperguide vs. Conventional Methods During Percutaneous Image Guided Procedures
InfectionCancer3 moreBackground: - Procedures that use medical tools in or near a possible abnormality in the body often use computed tomography (CT) scans to locate the abnormality and guide the path that a needle will take to collect a sample of tissue. Xperguide and electromagnetic (EM) tracking are two new procedures being studied to help guide the needle. Xperguide is software that uses CT images to help the doctor choose the needle path. EM tracking uses special medical tools with miniature coils that act like a Global Positioning Satellite (GPS) device to show the location of the needle in the body. Xperguide and EM tracking have been used in humans and have good results, but they have not been compared with each other and regular CT to determine whether they are better than the standard approach. Objectives: - To compare the results of Xperguide, electromagnetic tracking, and regular computed tomography during a guided percutaneous procedure. Eligibility: - Individuals at least 18 years of age who are required to have a CT-guided percutaneous procedure. Design: Participants will be screened with a physical examination and medical history, and the results of any previous imaging studies will be examined before study enrollment. After a pilot phase, the study will involve two phases to compare the results of the different procedures. The first phase will involve comparing Xperguide to CT, and the second will involve comparing Xperguide to EM tracking. Phase 1 participants will be assigned to one of two procedure groups: Group 1 will have Xperguide, and Group 2 will have regular CT. Participants who are scheduled to have repeated procedures (like a biopsy before and after chemotherapy) will be randomized for the first procedure and the second procedure will be done using the other method. Phase 2 participants will be assigned to one of two procedure groups: Group 1 will have Xperguide, and Group 2 will have EM tracking. Participants who are scheduled to have repeated procedures (like a biopsy before and after chemotherapy) will be randomized for the first procedure and the second procedure will be done using the other method. Standard post-procedure followup care will be given after the study procedure is completed.
Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)
Granulomatous DiseaseChronic2 moreChronic Granulomatous Disease (CGD) is a rare inherited disorder in which patients suffer from severe infection and inflammation. The first indication of disease usually appears in early childhood. The basic defect found to be lie in specialised white blood cells called phagocytic cells, which are responsible for engulfing and destroying germs. In CGD, there is a defect in an enzyme (known as NADPH-oxidase) that is responsible for generating bleach like substances that are important for killing some important germs. In the form of the disease known as X-CGD (which accounts for two thirds of patients), there are defined mistakes in a gene called gp91-phox, which is a key part of the NADPH-oxidase. In many cases, patients can be protected from infection by constant intake of antibiotics. However, in others potential life-threatening infections break through. In some cases patients also develop serious inflammation requiring high doses of drugs such as steroids. CGD can be cured by bone marrow transplant, but the best results are available when there is matched donor available. Transplant from unmatched donor have a much worse outcome. Gene therapy of CGD can be performed by introducing a normal copy of human gp91-phox gene into the blood forming stem cells of patients' bone marrow by using a gene carrier (in this study called lentiviral vector). After treatment of the bone marrow cells in a specialised laboratory are given back to the patient and will grow into functional phagocytic cells.
Bevacizumab in Reducing CNS Side Effects in Patients Who Have Undergone Radiation Therapy to the...
Adult Anaplastic AstrocytomaAdult Anaplastic Ependymoma85 moreBevacizumab may reduce CNS side effects caused by radiation therapy. This randomized phase II trial is studying how well bevacizumab works in reducing CNS side effects in patients who have undergone radiation therapy to the brain for primary brain tumor, meningioma, or head and neck cancer.
Comparison Of Doxycycline And Common Salt For Treatment Of Umbilical Granuloma: A Randomised Control...
Umbilical Granulomait is the study for the disease called umbilical granuloma to look for the better treatment option which can be performed by healthcare physicians and local nurses and even parents
Denosumab in Subjects With Giant Cell Rich Tumors of Bone
Aneurysmal Bone CystsGiant Cell Granuloma3 moreAn open-label, multi-center, phase 2 study of the efficacy of denosumab in subjects with giant cell rich tumors of bone. The population will consist of subjects with the following tumor types: aneurysmal bone cysts (ABC), giant cell granuloma (GCG) and other giant cell rich lesions (primary bone, non-malignant).
Lentiviral Gene Therapy for CGD
Chronic Granulomatous DiseaseThis is a Phase I/II clinical trial of gene therapy for treating Chronic Granulomatous Disease using a high-safety, high-efficiency, self-inactivating lentiviral vector TYF to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the TYF-CGD gene transfer clinical protocol.