52 Clinical Trials for Various Conditions
This phase II trial tests how well pembrolizumab and tazemetostat work to treat patients who have received autologous stem cell transplantation (ASCT) or chimeric antigen receptor (CAR) T cell therapy for aggressive non hodgkins lymphoma. A monoclonal antibody, such as pembrolizumab, is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Tazemetostat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving pembrolizumab and tazemetostat may work better to treat patients who have received ASCT or CAR-T cell therapy for aggressive non hodgkins lymphoma.
This safety and feasibility, open-label study of up to 9 subjects will examine a group of subjects with poorly controlled tophaceous gout (intolerant to or ineffective oral urate lowering agents and loss of prior Pegloticase response) pre-treated with Rituximab to recapture response to Methotrexate-Pegloticase.
The purpose of this project is to study the effects of air pollution toxicants on pregnant mothers' immune health during and after pregnancy. Using already collected samples, this study proposes to evaluate changes in immune function in response to air pollution with the use of innovative technologies, to identify the drivers of immune dysfunction and potential modifiable factors, and to determine how these immune findings are associated with pollution exposure and outcomes of disease.
Pregnancy is a unique period which requires alterations in the immune system to allow for tolerance of a haploidentical fetus. The goal of this study is to measure maternal blood levels of proteins known to promote immune tolerance in early implantation and pregnancy to look for associations between tolerance, miscarriage and failed embryo transfer. Establishing predictive factors of miscarriage and failed in vitro fertilization could have implications for a large portion of couples and serve to guide current and future family planning efforts.
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at tolerization.
This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study, planned in of approximately 25 male participants with congenital hemophilia A who will receive their first (primary) immune tolerance induction (ITI) treatment with alphanate. The study consists of 2 phases: * An ITI Treatment Phase in which all eligible participants will receive ITI treatment with alphanate for a period of up to 33 months. Upon confirmation of complete immune tolerization, participants will then enter a 12-month Prophylactic Phase. If, after 33 months of ITI, a participants has achieved partial immune tolerance, the participants will enter a 12-month Prophylactic Phase. * A 12-month Prophylactic Phase for all participants who meet the criteria for complete or partial success to continue on a prophylactic dosing regimen of alphanate. Due to limited enrollment, this study was early terminated.
The primary purpose of this study was to describe the time to tolerization (i.e., ITI success) with rFVIIIFc in participants within a maximum of 48 weeks (12 months) of ITI treatment.
This study is being done with the purpose of trying to understand if and why transplant recipients may develop tolerance to their transplanted organ. Tolerance means being able to lower or take away immunosuppression (anti-rejection medications) without causing organ rejection.
The purpose of this study is to evaluate whether a concentrate containing both FVIII and von Willebrand Factor (VWF) given at a high dose will induce immune tolerance in subjects who have already experienced and failed ITI with VWF-free FVIII concentrates. The treatment on this study is expected to last up to 33 months.
An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who were then receiving Myozyme® therapy were enrolled into the study, and were followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme® alone). Eligible participants were followed for a minimum of 18 months on treatment or, if a participant was \<6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.
The goal of this study is to identify the immunological factors that influence a patient's response to the presence of the fungus Aspergillus fumigatus (A. fumigatus) in the lungs. In patients with cystic fibrosis (CF), this fungus is not known to cause damage to the lungs, but some patients respond with an allergic reaction that may cause wheeze, cough, or difficulty breathing. Approximately 230 patients will be enrolled with an additional 60 people who do not have CF and who do not have a history of asthma to serve as a comparison group.
The purpose of this study is to see if a low-dose arm or a high dose-arm of immune tolerance is more effective in eliminating inhibitors in patients with hemophilia A.
At the completion of primary follow-up of various Medeor Transplant studies (called parent studies), subjects receiving Medeor's cellular immunotherapy products will be followed annually for up to an additional 84 months (7 years), in order to evaluate for long-term safety.
This is a single arm phase 1 non randomized dose finding study for safety, feasibility and efficacy of deceased donor vertebral body (VB) marrow cell infusion and kidney transplantation.
The study will determine whether patients with functioning Human Leukocyte Antigen (HLA) matched kidney transplants for at least one year and who want to discontinue immunosuppressive drugs can be treated with Total Lymphoid Irradiation (TLI) and rabbit Anti-Thymocyte Globulin (rATG) and an HLA matched donor hematopoietic progenitor cell infusion such that their drugs are successfully withdrawn while maintaining normal renal function.
This research study is to determine if donor blood stem cells given after living, related, HLA antigen (Ag) haplotype match or living, unrelated donor kidney transplantation. Minimal HLA antigen matching will include matching of 2 HLA antigens that can be either HLA A, B, and /or DR. This research will change the immune system such that immunosuppressive drugs can be completely withdrawn or reduced to minimal dose without kidney rejection.
The Stanford Medical Center Program in Multi-Organ Transplantation and the Division of Bone Marrow Transplantation are enrolling patients into a research study to determine if blood stem cells injected after kidney transplantation, in combination with lymphoid irradiation ,will change the immune system such that immunosuppressive drugs can be completely withdrawn. Patients must have a healthy, completely human leukocyte antigen (HLA)-matched brother or sister as the organ and stem cell donor. One to two months before kidney transplant surgery, blood stem cells will be removed from the donor and the cells will be frozen. After transplant surgery, the recipient will receive radiation and anti-T cell antibody treatments for two weeks to prepare for injection of the stem cells. The stem cells will be injected at the end of the two-week treatment. If the stem cells persist in the recipient, immunosuppressive drugs will be gradually reduced until they are withdrawn completely at least six months after transplantation. Patients will be followed in the Stanford clinics for transplant patients. Patients who live outside of the San Francisco Bay Area must remain near Stanford for six weeks after transplant surgery.
This study will enroll children who have hemophilia A with inhibitors who successfully completed immune tolerance induction per the ISTH criteria (negative inhibitor titer, recovery \>66% of expected, and half-life of \>6 hours with their current FVIII concentrate). Previous to emicizumab, there was only one option for these patients which was to continue FVIII therapy in a prophylaxis mode to prevent bleeding. There was a sense that the ongoing FVIII served to maintain tolerance however no evidence for this notion exists and in fact what limited data is available suggests that continuing FVIII may not be necessary simply to maintain tolerance. To figure out this question, this will be a randomized, controlled 2 arm study which will randomize patients post-successful ITI to emicizumab plus weekly FVIII (for maintenance of tolerance) versus emicizumab alone. Patients will be followed for up to 2 years. We aim to enroll 52 subjects. The FVIII weekly arm can use any factor VIII concentrate and emicizumab is standard of care for inhibitor and non-inhibitor patients.
A randomized controlled study to evaluate the safety, efficacy, and overall benefit of FCR001 cell therapy in de novo living donor renal transplantation.
Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells. The treatment cyclosporine leads to better blood counts. But when this treatment is stopped, the disease may return in 1 in 3 people. The drug sirolimus may help by suppressing the immune system. Objective: To evaluate and compare the usefulness of sirolimus in preventing aplastic anemia from returning after cyclosporine is stopped, compared with stopping cyclosporine alone. Eligibility: People ages 2 and older with SAA who: Have responded to immunosuppressive therapy that includes cyclosporine, and continue to take cyclosporine Are not taking drugs with hematologic effects Design: Participants will be screened with: Medical history Physical exam Blood and urine tests Bone marrow biopsy: The area above the hipbone will be numbed. A thin needle will remove some bone marrow. Participants will be randomly assigned to a group. All will stop cyclosporine. Group 1 will take sirolimus by mouth at the same time each day for 3 months with close monitoring. Group 2 will not receive the study drug but will be monitored closely. Participants will have clinical tests for the first 3 months: Weekly blood test Monthly fasting blood test For group 1, measurements of sirolimus in the blood every 1 2 weeks Participants will have clinic visits at 3 months, 12 months, and annually for 5 years after the study starts. They may have another visit if their SAA returns. These will include: Blood and urine tests Bone marrow biopsy...
The intervention in this trial is intraocular islet transplantation. A single dose of 1000 - 3000 Islet Equivalents (IEQ)/kg recipient body weight (BW) will be infused into the anterior chamber of the eye through a self-sealing incision in the peripheral cornea. The procedure is projected to take approximately 20-30 minutes. Subject will remain flat on their back for 1 - 3 hours after islet infusion to maximize adhesion of the islets to the iris.
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.
An open-label study to assess the safety, tolerability, and efficacy of FCR001 cell therapy in adult recipients 3-12 months after kidney transplantation from a living donor.
This is a study for children and adults who are interested in a new therapy for their allergies to dust mite and timothy grass. The new therapy is called sublingual immunotherapy and the investigators are testing if it is safe and well tolerated.
To determine whether long-term maintenance therapy with a single drug (Myfortic) applied using advanced immunologic monitoring tools in selected patients can lead to superior native kidney function at 2 years without resulting in increased acute rejection episodes or deterioration of liver allograft function.
The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.
The purpose of this study is to determine whether treatment with multiple injections of GAD-Alum will preserve the body's own (endogenous) insulin production in patients who have been recently diagnosed with type 1 diabetes mellitus (T1DM).
This study will determine whether treatment with a combination of the drugs daclizumab and denileukin diftitox can eliminate the need for long-term daclizumab treatment in adult patients with uveitis. Denileukin diftitox kills white blood cells called lymphocytes that cause inflammation and may be the cause of uveitis. Patients 18 years of age and older with uveitis in one or both eyes who are on daclizumab therapy and have not had a disease flareup in 6 months may be eligible for this study. Candidates are screened with a medical history, physical examination, eye examination (including vision test, examination of the front of the eye, and pupil dilation for examination of the retina at the back of the eye), blood tests and a questionnaire about their vision and daily activities. After screening, participants undergo the following procedures: * Daclizumab/ denileukin diftitox treatment. Patients receive their regular dose of daclizumab intravenously (through a vein). The interval between doses is increased by 1 week after each dose. When the doses are 10 weeks apart, the daclizumab is stopped. Patients who experience a flare or uveitis are treated with intravenous denileukin diftitox and possibly local injections of steroids around the eye or increasing or adding other medicines as needed to control the uveitis. * Fluorescein angiography to look for blood vessel abnormalities in the eyes. A yellow dye is injected into an arm vein and travels to the blood vessels in the eyes. Pictures of the retina (the back portion of the eye) are taken with a special camera that flashes a blue light into the eye. The pictures show if any dye has leaked from the vessels into the retina, indicating possible abnormalities. * Ultrasound and urine tests at enrollment and after 1 year to check kidneys, lymph nodes and pelvic area. * Blood tests at enrollment and every 3-6 months for laboratory and immunology tests and other research tests on blood cells to examine the immune response. * Follow-up visits approximately every 6 weeks for 2 years for repeat examinations to determine the response to treatment and drug side effects.
This study will examine whether the combination of the drugs daclizumab and sirolimus can effectively treat adults with uveitis, an eye inflammation. Daclizumab is a monoclonal antibody that is designed to prevent a specific chemical interaction needed for immune cells called lymphocytes to produce inflammation. Sirolimus is an immune-suppressing drug that also controls lymphocyte activity and is marketed to prevent organ rejection in kidney transplants. Patients 18 years of age and older with non-infectious uveitis in one or both eyes who are being treated with daclizumab and have not had a relapse or disease flare for 6 months before entering this trial may be eligible for this study. Candidates are screened with a medical history and physical examination, blood tests, complete eye examination, and pregnancy test for women who can have children. Women of child-bearing potential who enroll in this study will have a pregnancy test every 12 weeks. After enrollment, participants have the following additional exams: * Fluorescein angiography: This test is done to check for abnormalities of eye blood vessels. A yellow dye is injected into an arm vein and travels to the blood vessels in the eyes. Pictures of the retina are taken with a special camera that flashes a blue light into the eye. The pictures show if any dye has leaked from the vessels into the retina, indicating possible abnormalities. This test is done at enrollment and after 1 year, unless additional tests are needed for medical management. * Pelvic ultrasound and urine test: These tests are done at enrollment and after 1 year to check the kidneys, lymph nodes, and pelvic area. * Blood tests: Blood tests are done at enrollment and every 3 to 6 months for laboratory and immunology study Patients receive daclizumab subcutaneously (under the skin) or in infusions at regularly scheduled visits for 52 weeks. At each treatment, blood pressure, pulse, breathing rate, and temperature are monitored. After the first 52 weeks, patients whose disease remains quiet increase the time between injections to 6 weeks and then to 8 weeks. Patients who are doing well at this time may stop daclizumab. One or 2 days after the first daclizumab treatment, patients receive 6 mg of sirolimus by mouth. Their blood pressure, pulse, breathing rate, and temperature are monitored for at least 60 minutes. Two days after the first dose, patients begin 2-mg maintenance doses every other day for 2 weeks. If there are no intolerable side effects, the dose is increased to 2 mg daily for the next 2 weeks. Patients who have no intolerable side effects at that dose continue the medication for another 4 weeks. Patients who experience intolerable side effects may decrease the medication to every other day or withdraw from the study. After week 78 of the study, if the daclizumab treatments are stopped, the sirolimus dose is reduced within 8 weeks and may eventually be discontinued if the patient continues to do well. Patients who experience any of the following will leave the study: * Inflammation flare that requires concomitant treatment with additional systemic immunosuppressive medications, such as prednisone or cyclosporine * Disease flares more than twice in the first year * Any disease flares in the second year
The purpose of this study is to determine whether a combined bone marrow and kidney transplant will be effective in treating stage II or greater multiple myeloma and associated kidney failure. This study will determine whether transplant rejection and the need for immunosuppressive drugs are decreased with this combined transplant approach.