Active clinical trials for "Deficiency Diseases"

This study will evaluate bioequivalence of two formulations of Ferric Carboxymaltose as measured by serum total iron, in adult patients with iron deficiency anemia.

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.

Over recent years there has been a lot of research looking at how the bacteria in our gut affects our health. Some medications are known to cause changes in gut bacteria. Many patients that are prescribed iron report gastrointestinal side effects. This research project aims to see if the cause of the gastrointestinal side effects is due to iron causing changes in the gut bacteria. This can be detected via measuring the levels of hydrogen and methane and other compounds in the breath and stool.

The World Health Organization recommends daily iron supplementation for infants and children (6 months-12 years). Based on the low cost and high bioavailability and efficacy, ferrous sulfate is typically the first choice for supplementation and fortification. The recommended dose of iron is set high to deliver adequate absorbed iron due to low rates of dietary iron absorption, which is typically <10%. Thus, the majority of dietary iron is not absorbed and travels to the colon. Unabsorbed iron in the colon may select for enteric pathogens at the expense of beneficial commensal bacteria and increase infection risk, including the clinical incidence of diarrhea. The objective of this study is to compare the effects of iron as ferrous sulfate (FeSO4) or FeSO4-enriched Aspergillus oryzae (Ao iron) on the growth and virulence of common enteric pathogens using an in vitro fecal fermentation model. Stool samples will be collected from children following ingestion of an iron supplement as either FeSO4 or Ao iron. Stool samples will be spiked with common enteric pathogens and outcome measures will be determined following in vitro fecal fermentation.

Iron supplementation is a common approach to address iron deficiency with recommendations for women of childbearing age, particularly those at risk of iron deficiency. Because of its considerably higher absorption, ferrous sulphate is the common iron compound used in iron supplements. However, concerns about iron supplements arise from the knowledge that a large portion of the supplement consumed is not absorbed. This unabsorbed iron travels to the colon and, in preclinical studies, has been shown to promote the growth of enteric pathogens at the expense of beneficial commensal bacteria and increase infection risk, including the clinical incidence of diarrhea. The objective of this study is to compare the effects of iron as ferrous sulfate (FeSO4) or FeSO4-enriched Aspergillus oryzae (Ao iron) on the growth and virulence of common enteric pathogens using an in vitro fecal fermentation model. Stool samples will be collected from women of reproductive age following ingestion of an iron supplement as either FeSO4 or Ao iron. Stool samples will be spiked with common enteric pathogens, and outcome measures will be determined following in vitro fecal fermentation.

A multi-center,randomized,double-blind,controlled clinical trial was used to investigate the effectiveness of the treatment of infant iron deficiency anemia by taking ferralia and different contents lactoferrin.

This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of a novel medical food utilizing a nutritional strain of yeast for management of Iron Deficiency Anemia.

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 study will conducted to evaluate and comparison between the effectiveness, safety and acceptability of lactoferrin alone, ferrous fumarate alone and combination of both for the treatment of mild to moderate IDA during pregnancy.

The main objective of this study is to evaluate the efficacy of intravenous iron sucrose in increasing preoperative haemoglobin values in patients with colo-rectal neoplasm and iron deficiency anemia, compared to the standard treatment with oral iron. It will also determine whether intravenous iron sucrose administration improves outcomes such as postoperative haemoglobin values, serum ferritin values, transfusional needs, postoperative complications, or length of hospital stay.