Active clinical trials for "Deficiency Diseases"

The indication of this study is To evaluate the safety and immunogenicity of a SARSCoV- 2-derived multi-peptide vaccine in combination with the TLR1/2 ligand XS15 in adults with congenital or acquired B-cell/antibody deficiency

The aim of the study is to recruit 30 patients who have been referred to Auerach Hematology Oncology Assoc. for the treatment of iron defeciency anemia with iv iron (Ferumoxytol 1020 mg) administered via infusion pump over 15 minutes. To ascertain acute safety and tolerability of ferumoxytol during immediate post dosing observation period. Assess additional safety profile and laboratory assessment at follow up visit and interim telephone follow up.

The purpose of this study is to determine whether ST10-021, an oral ferric iron preparation, is safe and effective in the treatment of iron deficiency anaemia (IDA) in subjects with non-active ulcerative colitis (UC).

Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment.

Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.

This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease. The study objective is to evaluate and quantify skeletal responses as compared to baseline in Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional objectives were to assess the usefulness of various skeletal parameters, such as bone pain, bone crises, bone mineral density, and serum and urine bone markers, as indicative of treatment response and may be useful in dose management.

Evaluating the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients with Iron Deficiency Anemia and an Unsatisfactory Response to Oral Iron under Study Protocol 1VIT17044

Iron deficiency anemia (IDA) affects nearly half a million young children in the United States. Most children take liquid iron medicine by mouth for at least 3 months. However, some children take longer to get better with this medicine. This study is trying to compare different ways of giving iron medicine to young children. For young children in the US, the main cause of IDA is nutritional, or not having enough iron in the foods they eat. This often happens when kids drink too much cow milk and/or not eating enough foods that have a lot of iron. Iron deficiency is most common in children ages 1 to 4 years of age, during a time that is important for brain development. More severe and long-lasting IDA is associated with worse brain development outcomes. That is why researchers want to understand the fastest way for kids with IDA to get better. Standard treatment is oral iron medicine for 3 to 6 months. Many children do not take their iron medicine the full amount of time needed because of side effects like abdominal discomfort, nausea, constipation, and bad taste. Different factors can contribute to patients not completing their IDA therapy. Many families do not understand how important it is to treat IDA or do not have the motivation to continue the medication. This study will offer different methods for treating IDA, including a different method to taking the oral iron therapy. This new method gives oral iron by increasing a family's understanding and motivation. Another research study that interviewed families of young children with IDA found ways that helped the patients to continue their therapy. Using that information, a website called IRONCHILD was created to help motivate parents to get their children to continue the oral iron medicine. Research studies that compare these different IDA treatment methods in young children are needed and could have benefits to short-term clinical and long-term brain development. However, we do not know whether families of young children with IDA will be willing to participate in this type of study that has different treatment methods (oral iron therapy and oral iron therapy with a web-based adherence intervention). The goal of this clinical research study is to learn which of the two methods of care will be the best way for children with iron deficiency anemia to receive therapy.

This study aims to compare the efficacy of lactoferrin versus iron supplementation versus two combined supplementation on iron deficiency anemia in female medical Ain Shams students by using iron profile.

The primary objective is to demonstrate the efficacy of ferric carboxymaltose (FCM) given in a simple dosing regimen in correcting iron deficiency anaemia (IDA), by demonstrating non-inferiority to treatment with the currently approved intravenous (IV) iron therapy of iron sucrose (IS, Venofer™) in the Chinese population. The secondary objectives are to assess the safety of FCM compared to IS in the Chinese population and to evaluate the effect of FCM compared to IS on relevant laboratory parameters (haematology, chemistry, iron parameters) in the Chinese population.