317 Clinical Trials for Various Conditions
This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency Diseases (PIDD). The study will be conducted at 5-7 centers in the United States, with subjects receiving six (28 day cycle) or seven (21 day cycle) doses of Asceniv™ during the study.
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES
The primary objective is to demonstrate the bioequivalence of Gammaplex® 10 intravenous immunoglobulin (IGIV) and Gammaplex® 5% IGIV with respect to area under the curve within a 28-day dosing interval (AUC0-28) in a cohort of adult subjects. The secondary objectives are to demonstrate the bioequivalence of Gammaplex® 10 IGIV and Gammaplex® 5% IGIV with respect to area under the curve within a 21-day dosing interval (AUC0-21) in adult subjects; to assess the pharmacokinetics of Gammaplex 10 IGIV and Gammaplex 5% IGIV including Immunoglobulin G (IgG) trough levels and to investigate the safety and tolerability of Gammaplex 10 IGIV and Gammaplex 5% IGIV in adults subjects; to assess the pharmacokinetics of Gammaplex 10 IGIV including IgG trough levels and to investigate the safety and tolerability of Gammaplex 10 IGIV in pediatric subjects.
The main objective of the study is to determine the pharmacokinetics profile of Subgam-VF. The secondary objectives are to assess the safety of Subgam-VF and refine the dose adjustment coefficient for Subgam-VF needed for subjects switching from prior intravenous immunoglobulin (IGIV) therapy.
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.
This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary Immunodeficiency Diseases (PIDD).
This protocol is designed to ascertain whether the bacteriophage 0X174 neoantigen is safe and effective as an antigen used in the evaluation of primary and secondary immune responses. Bacteriophage 0X174 is given intravenously 2 billion PFU/Kg of body weight; small blood specimens of 3-5 ml (about 1 teaspoon) are collected after 15 minutes, 7 days, 14 days, and 28 days. Blood is collected at intervals following the administration of the bacteriophage and the number of phage/ml is determined by the agar overlay method using suspension of E. coli C and serially diluted patient's serum. Phage-specific IgG and IgM are measured by neutralization assay. Capacity of switch from IgM to IgG is determined.
The purpose of this study is to determine if NABI-IGIV (10%) \[Immune Globulin Intravenous (Human), 10%\] is safe and effective in preventing serious bacterial infections (SBIs) in the treatment of patients with primary immune deficiency disorders (PIDD) when compared to historical control data.
The main objective of this study is to see if GAMMAPLEX is efficacious with respect to Food and Drug Administration (FDA) minimal requirements (no more than 1 serious, acute, bacterial infection per subject per year) in subjects with Primary Immunodeficiency Diseases (PID). The secondary objectives are to assess the safety and tolerability of GAMMAPLEX and to determine if GAMMAPLEX has a pharmacokinetic (PK) profile comparable with that of intact Immunoglobulin G (IgG) in subjects with PID.
The main aim of this study is to learn if TAK-881 is safe and well tolerated during long-term use in adults and children with PIDD who are eligible and completed study TAK-881-3001 (NCT05755035). The participants will receive the first dose of TAK-881 immunoglobulin (IgG) infusion at the same dosage as the last dose administered in study TAK-881-3001 (NCT05755035). After the first TAK-881 infusion in study TAK-881-3002, the dosing interval (can be adjusted by participant/caregiver) and/or the dose of TAK-881 can be adjusted by the study doctor to every 2, 3 or 4 weeks at scheduled site visits. Participants will visit the clinic every 12 weeks until the End of Study (EOS) visit.
The main aim of this study is to compare the pharmacokinetics (PK) of subcutaneous (SC) administration of TAK-881 with HYQVIA in participants with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The participants who are already receiving intravenous immunoglobulin G (IGIV)/conventional subcutaneous intravenous immunoglobulin G (cIGSC) will be treated with HYQVIA or TAK-881 with the same dose and dosing interval of immunoglobulin for up to 24 weeks. Participants will need to visit the clinic every 3 or 4 weeks during the duration of the study.
Summary for SCGAM-03: Clinical phase III study to monitor the safety, tolerability and efficacy of subcutaneous human immunoglobulin (Octanorm) in patients with primary immunodeficiency diseases who have completed the SCGAM-01 trial. Summary for SCGAM-03 in Canada: Clinical phase III study to monitor the safety, tolerability and efficacy of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases, including (but not limited to) those who have completed the SCGAM-01 trial
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.
This study will try to identify mutations in the genes responsible for primary immunodeficiency disorders (inherited diseases of the immune system) and evaluate the course of these diseases in patients over time to learn more about the medical problems they cause. The immune system is composed of various cells (e.g., T and B cells and phagocytes) and other substances (complement system) that protect the body from infections and cancer. Abnormalities in the gene(s) responsible for the function of these components can lead to serious infections and other immune problems. Patients with Wiskott-Aldrich syndrome, adenosine deaminase (ADA) deficiency. Participants will undergo a medical and family history, physical examination, and additional procedures and tests that may include the following: 1. Blood tests for: routine laboratory studies (i.e. cell counts, enzyme levels, electrolytes, etc.); HIV testing; immune response to various substances; genetic testing; and establishment of cell lines to maintain a supply of cells for continued study 2. Urine and saliva tests for biochemical studies 3. Skin tests to assess response to antigens such as the viruses and bacteria responsible for tetanus, candida, tuberculosis, diphtheria, chicken pox, and other diseases. 4. Skin and lymph node biopsies for tissue and DNA studies 5. Chest X-ray, CT scans, or both to look for cancer or various infections. 6. Pulmonary function test to assess lung capacity and a breath test to test for H. pylori infection. 7. Dental, skin and eye examinations. 8. Treatment with intravenous immunoglobulins or antibodies to prevent infections. 9. Apheresis for collecting white blood cells to study cell function. In this procedure, whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are then removed, and the red cells, platelets and plasma are returned to the body, either through the same needle or through a second needle placed in the other arm. 10. Bone marrow sampling to study the disease. A small amount of marrow from the hipbone is drawn (aspirated) through a needle. The procedure can be done under local anesthesia or light sedation. 11. Placental and umbilical cord blood studies, if cord blood is available, to study stem cells (cells that form blood cells). Information gained from this study may provide a better understanding of primary immunodeficiencies, leading to better diagnosis and treatment. In addition, study participants may receive medical and genetic counseling and may be found eligible for other NIH studies on these diseases.
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
Patients with chronic lymphocytic leukemia (CLL) are at increased risk of infections as compared to age matched controls, with infections being a major cause of morbidity and mortality. Previous studies have shown that patients with CLL have both hypogammaglobinemia and impaired humoral immunity as defined by vaccine responses to both polysaccharide and peptide antigens. Attempts at decreasing infections in CLL have included therapy with prophylactic antibiotics and intravenous immunoglobulin. In general clinical practice and in previous studies, patients have started IV immunoglobulin replacement therapy if they have a history of serious infection or hypogammaglobinemia (defined as Immunoglobulin G below 500-600 g/dL), but vaccine responses have not been evaluated. This study will identify CLL patients with humoral immunodeficiency by checking both Ig levels and vaccines responses. In patients with impaired humoral immunity, the investigators will use subcutaneous immunoglobulin replacement to show this intervention will increase Ig levels, protective antibody titers, and be well tolerated.
The purpose of this study is to investigate the efficacy, safety, tolerability, and pharmacokinetic profile of the investigational medicinal product (IMP) and to determine, on the basis of historical control data, how it compares with other 10% intravenous immunoglobulin (IGIV) products currently licensed in North America for the treatment of subjects with primary immune deficiency diseases (PIDD).
This aim of this research project is to determine if low to moderate level exercise can have an impact on stress, fatigue, and quality of life for individuals diagnosed with a primary immunodeficiency disease. This 8-week study will compare participants engaging in a semi-customized, home exercise program (exercise intervention group) to participants performing normal activities (non-exercise control group). This study will track stress, fatigue, and quality of life in individuals with a diagnosis of primary immunodeficiency disease, using standardized questionnaires, journals, and interviews.
This is a safety study to evaluate the risk of low blood pressure in subjects with Primary Immune Deficiency disorder (PID) treated with Bivigam™ or another commercial product under real world conditions. No study medication will be provided to subjects in this study. Study physicians will make all treatment decisions according to their usual practice and will provide prescriptions for his/her subjects, as appropriate. The only addition is the collection and structured documentation of data generated through usual practice.
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
This is an open-label phase III study with a 12-week wash-in/wash-out period followed by a 12-month efficacy period. The main goals of the study are to assess the efficacy of octanorm in preventing serious bacterial infections (SBI) compared with historical control data and to evaluate the pharmacokinetic (PK) characteristics of octanorm.
Primary: * Demonstrate the utility of an electronic data capture (EDC) system (CareExchange™) using infusion nurse and patient measured physical, quality of life (QOL), respiratory, laboratory, and disability assessments in patients with Primary Immunodeficiency Disease (PIDD). Secondary: * Change in Intravenous/Subcutaneous Immunoglobulin (IVIg/SCIg) dose effects measured outcomes. * Change in IVIg/SCIg dose timing effects measured outcomes. * Change in patient status is reflected in measured outcomes. * Assess the value to physicians from collected outcomes data. * Identify types of patients by response to IVIg/SCIg therapies (well maintained, problematic, etc.). * Change in response rate as measured by outcomes to IVIg/SCIg therapies by disease state, co-morbidities, and demographics.
Study Design: This is a 2 arm non-interventional trial that will compare the occurrence of adverse drug reactions between Octagam 5% and other marketed IVIG infusion treatments.
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.
The main objective is to determine the efficacy of Gammaplex by measuring the number of serious acute bacterial infections during treatment with Gammaplex over a 12 month period. The secondary objectives are to assess the safety and tolerability of Gammaplex and to compare the data collected from adult subjects with PID from the GMX01 study
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
This is a multi-institution, single arm, non-randomized pilot study coordinated by the Pediatric Blood and Marrow Transplant Consortium. Eligible patients will have severe combined immunodeficiency syndrome (SCID) or severe T-cell immunodeficiency disorder. Patients with these disorders do not have properly functioning immune systems. Without treatment, these disorders result in early childhood death. The standard treatment used for these diseases is to give the patient a stem cell transplant from a matched donor. The donor cells can be from a family member, an unrelated marrow donor or umbilical cord blood. The donor source will impact on transplant risks and approaches to the preparative regimen. There have been many different preparative regimens used for patients with SCIDS or severe T-cell immunodeficiency syndromes. Some patients have gotten no preparative regimen, while others have gotten only antithymocyte globulin (ATG; immune proteins made in horses that, when given, will kill lymphocytes). Still other patients have gotten conventional chemotherapy. In children treated with nothing or ATG alone, there is an increased risk of graft failure or only partial engraftment. When this happens, patients need life-long therapy with immunoglobulins to support the immune system. Children treated with chemotherapy generally have full immune recovery, but also may have major side effects from the chemotherapy. The side effects include infection, organ failure and infertility. This protocol, in combination with a parallel study with a separate preparative regimen, will attempt to answer the question of which patients with primary immunodeficiencies need a preparative regimen and what intensity is needed. Patients will be enrolled according to disease type and donor source. The purpose of this study is to see how much chemotherapy is actually needed for the transplant to work. To be able to do this and still make the transplant work, the drugs used to temporarily weaken the immune system will be strengthened. In groups, patients will be treated with lowering doses of the busulfan to find the lowest dose of this drug that is needed to get full immune recovery. The investigators hope this regimen will result in complete immune system recovery while limiting the side effects of chemotherapy. A second purpose of this study is to track the recovery of different parts of the immune system. The investigators also want to identify whether the recovery is coming from donor stem cells or from the patient. The patient will be admitted to the hospital to have the transplant and is expected to stay for up to 4 to 6 weeks. The preparative regimen will be made up of busulfan, fludarabine and antithymocyte globulin (ATG). After the preparative regimen, the cells from the donor will be given. To try and keep the patient's body from rejecting the donor cells and the donor cells from attacking the patient's body (graft-versus-host disease, or GVHD), cyclosporine will be given. The investigators will draw an extra 2 - 4 teaspoons of blood at specified time points to test for immune recovery and donor cell chimerism (the portion of the blood that belongs to the donor). Standard bone marrow transplant (BMT) clinical care will be provided with respect to pretransplant evaluation, peritransplant support, and posttransplant follow-up.