View clinical trials related to Deficiency Diseases.
Filter by:This study aims to assess whether administering intravenous iron early in pregnancy, compared to standard oral iron treatment, can enhance hemoglobin levels before delivery and reduce the need for blood transfusions in patients with iron deficiency anemia. Patients diagnosed with iron deficiency anemia were randomly assigned to receive either oral or intravenous iron. Before treatment initiation, patients completed a symptom questionnaire baseline hemoglobin, and ferritin levels were measured. Follow-up visits occurred four weeks later and at 24 to 28 weeks gestation, involving reassessment of symptoms, laboratory testing, and monitoring of treatment adherence. Final hemoglobin levels were determined before delivery, and data on the need for blood transfusion at delivery were recorded.
Iron deficiency anemia (IDA) is a widespread condition affecting many people globally. Despite the many cases of IDA, there are few studies that compare the effectiveness of different types of iron supplements with a greater emphasis on the cost of supplements and their tolerability. This study aims to conduct a comparison between different iron supplements to determine the superiority of the iron supplements Ferrous Ascorbate (EBMfer) and Ferrous Fumarate (Eurofer) over polysaccharide iron (FeraMax). This comparison will be conducted by determining the change in hemoglobin levels for participants in this trial over a 3-month period.
POAM is a multicenter, randomized, controlled, internal pilot trial, using a conventional, parallel group, two-armed design at 3 cardiac surgery centres in Canada. The study is designed to assess the feasibility of a future, definitive RCT investigating whether, in patients with chronic iron-deficiency anemia undergoing cardiac surgery, IV iron therapy in the postoperative period (initiated shortly after surgery, and repeated at 42 days after surgery, if needed) improves clinical outcomes (days alive and out of hospital at 90 days after surgery; DAOH-90) relative to placebo.
The aim of this prospective, observational cohort study is to assess the impact of iron deficiency anaemia on the incidence of perioperative complications and the quality of recovery after surgery in patients undergoing colorectal cancer surgery. The main questions the study aims to answer are: - whether the presence of preoperative iron deficiency anaemia leads to a poorer quality of postoperative recovery in patients undergoing colorectal cancer surgery - whether different combinations of complete blood count parameters (red blood cell indices) could be suitable diagnostic tools for the detection of iron deficiency in the latent stage (without laboratory-confirmed anaemia) in colorectal cancer patients. Blood samples for laboratory analyses will be collected from each study patient admitted to the surgical ward one day prior to elective surgery and on the first postoperative day during the stay in the intensive care unit. The pre-operative laboratory analyses include a complete blood count and serum iron status parameters (iron concentration, ferritin concentration, TIBC, UIBC and TSAT). Laboratory parameters analysed on the first postoperative day include complete blood count, serum concentration of electrolytes (Na, K, Ca, Cl, Mg), serum concentration of urea and creatinine, parameters of haemostasis (aPTT, PT, INR), serum concentration of C-reactive protein and procalcitonin. Data about overall morbidity, intraoperative complications, quality of postoperative recovery, red blood cell transfusion rate, all-cause infection rate, antibiotic usage, as well as length of hospital stay will be collected. The researchers will compare the group of patients with iron deficiency anaemia, the group of patients with iron deficiency in the latent stage and the control group to determine whether patients with iron deficiency have a higher incidence of perioperative complications and impaired recovery after surgery. The researchers will investigate whether iron deficiency can be detected at an early stage, when anaemia is not yet present, by calculating various red blood cell indices.
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. This is a Phase 1/2, open-label, multicenter, safety and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The primary objective of this study is to evaluate the safety and tolerability of up to two dose levels of ECUR-506 following intravenous (IV) administration of a single dose.
Study Procedures: All patients will undergo the following: Informed consent will be obtained from all the participants in this study before enrolling in this study and all participants will be subjected to a detailed clinical assessment including: a detailed history, general, abdominal examinations, Investigations. 1. History taking: - Personal history: name, age, occupation and address. - Menstrual and obstetric history: Date of LMP, expected date of delivery which will be calculated according to Naegle's rule and gestational age. In addition to history of presence of any menstrual irregularities, duration. - Past History: of Anemia in previous pregnancy, other diseases like Thalassemia, sickle cell anemia, liver or renal diseases or any other condition that may affect hemoglobin. 2. Medical examination: - General: Assessment of complexion and vital data (blood pressure, pulse, capillary refill) - Abdominal examination to assess fundal height. 3. Investigations to perform will include: Laboratory: Complete blood count (microcytic hypochromic anemia) Imaging: Ultrasound to assess biometry to exclude fetal growth restriction. Women will be divided in two groups with 20 in each group, the first group will receive one tab of ferrous gluconate 300mg administered orally twice per day for 4 weeks and the second group will receive lactoferrin sachets 100mg with ferrous gluconate 300mg twice per day for 4 weeks Patients were assigned to take the medication orally; once daily before breakfast, and Pravotin (100 sachets were be dissolved each in ¼ glass of water and taken before breakfast). Patients were advised to avoid the intake of tea, coffee, milk, milk products, antacids and calcium preparation within 2 hours before or after iron capsules. Women will be told to record side effects as nausea, vomiting, abdominal discomfort and constipation. Women will have a blood sample (CBC) withdrawn after 2 to 4 weeks to assess rise in pregnant anemia.
The goal of this clinical trial is to evaluate, if a change in dosing strategy in oral iron supplementation from leads to an improvement of iron absorption and consequently a faster therapy in patients with iron deficiency anemia. The main question the study aims to answer is: Does a treatment effect (measured by change in hemoglobin after 12 weeks of treatment) exist between daily oral iron supplementation and interval administration (every second day)? Participants will receive oral iron supplementation every second day with double the standard dose (intervention group). Researchers will compare this group with the active control group, that receives oral iron supplementation every day with the standard dose. Participants will present for three patient visits (at baseline, after 6 and after 12 weeks of treatment).
This project aim to investigate whether wireless capsule endoscopy(WCE) has a similar diagnostic yield as conventional endoscopy in detecting the cause of iron deficiency anemia (IDA)
The goal of this observational study is to characterize the epidemiology and natural history of MPS diseases by building a retrospective and prospective collection of extensive phenotypic data from French MPS patients.
The study of splenomegaly, and the follow-up of splenectomized patients, is one of the causes of referral of these patients to pediatric gastroenterology and oncohematology clinics, and adult internal medicine and hematology. The study and management of splenomegaly is well described among the different medical specialties to which these patients arrive. After the application of the different algorithms and the different studies that are carried out, these splenomegaly are identified as being of hepatic, infectious, inflammatory, congestive, hematological origin and primary causes. Despite these studies of splenomegaly, approximately 10-15% of these patients still remain undiagnosed. Several studies have suggested that there is an increased frequency of MGUS (monoclonal gammopathy of undetermined significance) and/or multiple myeloma (MM) among Gaucher patients. Regarding ASMD (Acid Sphingomyelinase Deficiency), few studies have been published but it seems the 21% of patient with ASMD has MGUS and 15% ASMD patients have MGUS. Moreover, patients with MGUS and Gaucher disease (GD) are at increased risk of developing MM. The objective of the present study is to increase the diagnostic sensitivity of these unknown splenomegalys, or unknown splenomegaly patients with MGUS or multiple myeoloma who remain in consultations, using the usual diagnostic clinical procedures of unknown splenomegaly and unknown splenectomy patients, where we include the extraction of a blood sample for dry drop test (DBS), where the determination of the enzymatic/genetic activity will be carried out for Gaucher disease (GD) and acid sphingomyelinase deficiency (ASMD) , analysis of LisoGl1 and LisoSM.