Study to Evaluate the Efficacy and Safety of Ibrexafungerp in Patients With Fungal Diseases That...
Invasive CandidiasisMucocutaneous Candidiasis8 moreThis is a multicenter, open label, non-comparator, single arm study to evaluate the efficacy and safety of ibrexafungerp (SCY-078) in patients ≥ 18 years of age with a documented fungal disease that has been intolerant or refractory (rIFI) to Standard of Care (SoC) antifungal treatment.
Natural History of Individuals With Immune System Problems That Lead to Fungal Infections
Chronic Mucocutaneous CandidiasisInvasive Aspergillosis2 moreBackground: - The immune system is made up of special cells, tissues, and organs that fight infections. Problems with this system may lead to frequent, severe, or unusual fungal infections. These infections are often difficult to treat. Researchers want to collect blood and tissue samples from people who have unusual, persistent or severe fungal infections or immune problems that increase the risk of these infections. Objectives: - To collect medical information and samples for a long-term study of people with immune system problems that lead to fungal infections. Eligibility: People with a history of fungal infections caused by immune system problems. Parents, children, and siblings of this group. Healthy volunteers not related to the first two groups. Design: This long-term study may last for up to 10 years. Those in the study may need to provide new information about every 6 months. The procedures for each person may vary with the particular diagnosis and the extent of fungal infection. Healthy volunteers may have only one or two visits. At the first visit, those in the study will have a full medical history and physical exam. They will also provide blood. Research procedures may include the following: Saliva, urine or stool testing Mouthwash collection for DNA testing Collection of cheek cells, nail clippings, or vaginal fluid Tests of leftover tissue or body fluid from previous medical procedures Skin or oral mucous membrane biopsy Collection of white blood cells Followup visits will involve a physical exam and updated medical history. Blood, saliva, urine, or nail clipping samples may be taken for ongoing studies. Any additional tests or exams required by the study doctors may also be done. Participants may withdraw from the study pool at any time.
Rituximab for Anti-cytokine Autoantibody-Associated Diseases
Pulmonary Alveolar Proteinosis (PAP)Severe Mucocutaneous CandidiasisBackground: Healthy people have white blood cells that protect them against bacteria, viruses, and fungi. However, some people have diseases which cause the body to make white blood cells that do not work properly. These white blood cells can attack the body s own proteins. These types of diseases are called anti-cytokine autoantibody-associated diseases. They can cause severe illnesses and even death. They are also difficult to treat with standard drugs. Rituximab is a drug used to treat rheumatoid arthritis. It attacks white blood cells that do not work properly. Currently, it is not approved for treating anti-cytokine autoantibody-associated diseases. However, researchers think that it may be able to help treat people with these immune diseases. Objectives: - To see if rituximab is a safe and effective treatment for anti-cytokine autoantibody-associated diseases. Eligibility: Individuals at least 18 years of age who have anti-cytokine autoantibody-associated diseases. Participants must also be enrolled in a related immune disorder study at the National Institutes of Health. Design: The study will last 24 months. Participants will take rituximab for 6 months and have follow-up visits for the remaining 18 months. Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Other samples will be collected as needed if participants currently have an infection. Participants will enter the hospital for 1 week at the start of treatment. They will have four doses of rituximab given 2 days apart. This first treatment will be monitored with frequent blood tests. Over the next 6 months, participants will have four more doses of rituximab given about 1 month apart. Treatment will be monitored with frequent blood tests and sample collections as needed. There will be four follow-up study visits at 3, 6, 12, and 18 months after the last dose of rituximab.
CAMB/MAT2203 in Patients With Mucocutaneous Candidiasis
CandidiasisChronic MucocutaneousThis is an open-label, dose-titration trial to study the efficacy, safety, and pharmacokinetics of oral cochleate amphotericin B (CAMB) in the treatment of mucocutaneous candidiasis infections in patients who are refractory or intolerant to standard non intravenous therapies.