Agranulocytosis

This Phase 3 study tests whether the drug mavorixafor can reduce serious infections and increase neutrophil levels in people with chronic neutropenia—a condition where the body doesn't make enough infection-fighting white blood cells. About 176 participants will receive either ma…

This trial tests whether a new drug called albipagrastim alfa can prevent severe neutropenia (dangerously low white blood cell counts) in children with sarcoma who are receiving chemotherapy. The drug is designed to stimulate the body's production of infection-fighting neutrophil…

This trial tests whether a low dose of the antifungal drug liposomal amphotericin B can prevent invasive fungal infections in children aged 3 to 17 who have prolonged neutropenia (very low white blood cell counts) after cancer treatment or stem cell transplant. The drug is given …

This study tests a daily injection called elamipretide in 48 people with genetically confirmed Barth syndrome, a rare condition that causes muscle weakness and heart problems. Participants will receive either the drug or a placebo for 72 weeks. The main goal is to see if the drug…

This study compares two treatments—Shengbai Oral Liquid (a herbal remedy) and Leucogen tablets (a standard drug)—for moderate neutropenia (low white blood cells) caused by anti-tumor drugs in breast cancer patients. Sixty participants will be randomly assigned to receive one trea…

This study tests whether a single injection of Telpegfilgrastim works better than daily Filgrastim shots to prevent fever and low white blood cells in children and young adults (ages 6-24) with solid tumors receiving strong chemotherapy. About 132 participants will receive either…

This phase II trial tests whether taking leucogen alongside standard ribociclib therapy can prevent severe neutropenia (dangerously low white blood cell counts) in women with early-stage hormone-positive breast cancer. About 94 participants will receive the combination and be mon…

This study is for people with blood cancers like leukemia who are getting chemotherapy or a stem cell transplant, which can cause very low white blood cell counts (neutropenia) for over a week. The goal is to see if the antibiotic eravacycline can prevent serious bacterial infect…

This Phase 2 trial tests whether adding trilaciclib to the chemotherapy drug lurbinectedin can reduce severe neutropenia (a dangerous drop in white blood cells) in people with small cell lung cancer that has spread. About 30 participants will receive both drugs intravenously. The…

This study tests the drug luspatercept in 50 adults with clonal cytopenias of uncertain significance (CCUS), a condition where low blood cell counts increase the risk of developing blood cancers like leukemia. Participants receive injections every three weeks for up to 48 weeks. …

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study tests whether a drug called mecapegfilgrastim can safely prevent moderate-to-severe myelosuppression (low blood cell counts) caused by chemotherapy and radiation for lung or esophageal cancer. About 30 adults aged 18-75 will receive the drug alongside their standard ca…

This study aims to see if a home lactate meter and a standard glucose meter give accurate readings compared to lab tests in people with glycogen storage disease types Ia, Ib, and XI. Ten participants will have an 8-hour hospital stay with hourly blood draws and finger-stick tests…

This study looks at whether a low dose of the antifungal drug liposomal amphotericin B can prevent serious fungal infections in adults with blood cancers or who have had stem cell transplants and have a prolonged low white blood cell count (neutropenia). The trial enrolls 30 pati…

This real-world study looks at whether a drug called mecapegfilgrastim can prevent neutropenia (a low white blood cell count) in cancer patients after chemotherapy, radiation, or immunotherapy. The study will follow 200 adults with solid tumors who are at high or moderate risk of…

This study tests two oral medications—diosmin-hesperidin (a flavonoid supplement) and loratadine (an allergy drug)—to prevent bone pain caused by G-CSF, a drug that boosts white blood cells in blood cancer patients. Researchers will compare each drug alone and in combination agai…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study aims to learn more about Chediak-Higashi syndrome, a rare genetic disorder that causes light skin and hair, easy bruising, and frequent infections. Researchers will observe up to 60 patients over time, collecting clinical and genetic data to better understand the disea…

This study aims to collect DNA samples and detailed eye exam data from 1,000 people with rare inherited eye diseases like aniridia, Best disease, and albinism. Participants provide a saliva or blood sample and share their eye health records. The goal is to expand a research repos…

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

This study is creating a registry for people with Shwachman-Diamond Syndrome (SDS) or similar conditions. Researchers will collect medical records and biological samples from up to 5,000 participants to understand how the disease progresses and what treatments work best. The goal…

This study explores whether cancer patients can safely monitor their own blood counts at home during chemotherapy. About 265 participants from Denmark, Poland, and Germany will test a home monitoring device for several cycles. The goal is to reduce unnecessary hospital visits, lo…

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…