A Study to Evaluate Efficacy, Safety, and Pharmacokinetics of XEMBIFY® Plus Standard Medical Treatment...
HypogammaglobulinemiaBacterial Infections1 moreThe primary purpose of the study is to evaluate whether weekly administered XEMBIFY® plus Standard Medical Treatment (SMT) over a one-year period will reduce the rate of major bacterial infections per participant per year in participants with hypogammaglobulinemia (HGG) associated with B-cell chronic lymphocytic leukemia (CLL) in comparison to the Placebo plus SMT group.
Immune Disorder HSCT Protocol
Immune Deficiency DisordersSevere Combined Immunodeficiency12 moreThis 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.
Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and...
Chronic Lymphocytic LeukemiaHypogammaglobulinemiaStudy Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia
Role of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy: Immunoglobulin Stopping...
Haematological MalignancyHypogammaglobulinemiaThe aim of the study is to find out if patients with blood cancers receiving immunoglobulin (Ig) for the purpose of preventing infections can safety stop immunoglobulin after six months of therapy, and take oral antibiotics instead to prevent serious infections. Patients may be eligible to join this study if they are aged 18 years or above, have an acquired hypogammaglobulinaemia secondary to a haematological malignancy, and have been receiving intravenous or subcutaneous Ig for longer than 6 consecutive months. Participants will be randomised (allocated by chance) to one of three treatment groups, as follows: Stop immunoglobulin (IVIg or SCIg) and be given oral antibiotics to take every day (ARM A) Stop immunoglobulin (IVIg or SCIg) and be given oral antibiotics to keep at home to use as soon as symptoms of an infection develop (ARM B) Continue receiving immunoglobulin (IVIg or SCIg) - this is the usual care group (ARM C) The duration of each treatment is for 12 months from study entry. Participants will be asked to attend a screening/baseline visit so that their treating clinician can assess their eligibility for the trial and collect baseline data. If eligible for the trial, participants will then be randomly allocated to one of the three treatment groups. Once randomised, active participation in the study will last for 13 months. During this period, participants will be asked to return to the hospital for a study visit every 3 months, with monthly telephone visits to check-in on your progress between each in-person visit. Participants will also be asked to complete a study diary, recording treatment compliance and signs/symptoms of infection experienced throughout the study period. Types of assessments and data collected will include: Medical history, demographics, physical examination, blood tests, stool sample, quality of life questionnaires, information about your general health, hospitalisations, medications and procedures. In order to assess and compare the cost-effectiveness of the treatment groups, the study team will also request authorisation from participants to access their Medicare Benefits Schedule (MBS), Pharmaceutical Benefits Scheme (PBS), and Australian Immunisation Register (AIR) data.
Immunoglobulin Deficiency a Treatable Cause of Fatigue in Patients With Multiple Sclerosis (MS)?...
HypogammaglobulinemiaMultiple Sclerosis1 moreThe investigators hypothesize that hypogammaglobulinemia (defined as IgG serum concentration <7.0g/L) is a treatable cause of fatigue in people with MS: The primary objective is to prove the link between hypogammaglobulinemia and fatigue in patients with multiple sclerosis. The secondary objective is to show that fatigue is mediated via frequent infections in people with MS and hypogammaglobulinemia.
COVID-19 Vaccine Responses in PIDD Subjects
X-linked AgammaglobulinemiaXLA5 moreThe goal of our study is to assess the cellular immune responses of participants with antibody deficiency disease before and after immunization with SARS-CoV-2 mRNA vaccines.
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
Immunologic Deficiency SyndromesChediak-Higashi Syndrome12 moreOBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II. Determine relevant outcomes of this treatment in these patients including quality of survival, extent of morbidity and mortality from complications of the treatment (e.g., graft versus host disease, regimen related toxicities, B- cell lymphoproliferative disease), and completeness of functional immunoreconstitution.
Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
MyelokathexisInfections3 moreBackground: - WHIMS (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis Syndrome) is a rare disease. It can cause cancers, infections, and warts. Researchers want to see if a drug called plerixafor can treat WHIMS. Objective: - To compare plerixafor versus granulocyte colony stimulating factor (G-CSF) for preventing infections in people with WHIMS. Eligibility: - People ages 10-75 with WHIMS who have a CXCR4 gene mutation. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. They may have heart and spleen tests and body scans. They may have samples of skin or warts taken. Researchers may take photographs of warts. Participants will start twice daily self-injections of G-CSF. Their doctors will decide the dosage. Initial Period (4-12 weeks) Participants will: continue the injections and their usual antibiotics and/or immunoglobulin have blood drawn keep a daily health diary Participants will visit the clinic for 2 days without injections. Adjustment Period 1 (8 weeks): Participants will: continue twice daily injections from home continue the daily health diary have blood tests every 2 weeks. Treatment Year 1: Participants will receive either plerixafor or G-CSF injections twice daily continue the health diary have blood tests every 2 months visit the clinic about every 4 months At the end of year 1, participants will visit the clinic for an evaluation. They will switch to the other study drug. They will have an 8-week adjustment and 1-year treatment period. At the end of year 2, participants will visit the clinic to complete their injections and go back to their previous G-CSF regimen. Participants will continue their daily health diary and have blood tests for 5-6 months.
Post-bypass Prophylactic IVIG in Infants and Neonates
HypogammaglobulinemiaCongenital Heart DiseaseThe purpose of this study protocol is to determine if administering Intravenous Immunoglobulin (IVIG) for treatment of cardiopulmonary bypass (CPB) induced hypogammaglobulinemia in the early post-operative period can impact post-surgical outcomes (i.e., infection, fluid overload, and associated morbidities).
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)...
Primary ImmunodeficiencyAgammaglobulinemia2 moreThe purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).