Inherited aplastic anemia

This study tests a stem cell transplant using cells from mismatched unrelated or partially matched family donors for people with severe sickle cell disease and other transfusion-dependent anemias. The approach removes certain immune cells (T-cells) from the donated stem cells to …

This early-stage trial tests a one-time gene therapy called APR-2020 in 4 children with a severe form of Diamond-Blackfan anemia. The treatment uses the child's own blood stem cells, modified to correct the genetic defect, and given back as an infusion. The goal is to see if it i…

This early-phase study tests a new drug called DSB2455 in adults with advanced cancers (breast, ovarian, prostate, pancreatic, or brain metastases) that have a specific DNA repair problem. The main goals are to check safety and see if the drug shrinks tumors. About 180 participan…

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study e…

This phase 2 trial tests a stem cell transplant from a donor for people with high-risk sickle cell disease, thalassemia, and other red blood cell disorders. Participants receive drugs and low-dose radiation before the transplant to help the new cells take hold. The study will tra…

This study tests a drug called afatinib in 25 adults with Fanconi anemia who have advanced head and neck cancer that cannot be removed by surgery. Afatinib is a pill that blocks a protein helping cancer grow. The goal is to see if it can shrink tumors and control the disease. Res…

This study tests a device that removes specific immune cells from donated stem cells before transplant. The goal is to lower the risk of graft-versus-host disease, where donor cells attack the patient's body. Up to 90 children and young adults (ages 0-30) will receive these treat…

This expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…

This phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…

This study tests a new way to treat Fanconi Anemia, a rare genetic disorder that causes bone marrow failure. Doctors will give patients donor stem cells that have been specially filtered to remove certain immune cells, along with an experimental antibody called JSP191. The goal i…

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 phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…

This study tests a stem cell transplant approach for people with Fanconi Anemia who don't have a perfectly matched donor. The goal is to see if using lower doses of chemotherapy before the transplant can reduce serious side effects. About 70 participants will receive a transplant…

This study explores whether MRI scans can safely and accurately detect cancerous or precancerous mouth lesions in people with Fanconi Anemia, a rare genetic condition that raises cancer risk. Researchers will compare MRI results to standard biopsies in 80 adult participants. If s…

This study tests whether an artificial intelligence (AI) algorithm can screen for coronary artery disease (CAD) by analyzing facial images. Researchers will enroll 1,392 high-risk adults (those with diabetes, high blood pressure, or over 65) and use the AI to classify them as hig…

This study tests whether a personalized facial rehabilitation program can help people with systemic sclerosis who have facial tightness and mouth problems. About 60 adults with this rare autoimmune disease will either receive the rehab program or standard care. The goal is to see…

This study is testing whether hypnosis can lower anxiety in people having facial surgery under local anesthesia. Sixty-six adults who are at least mildly anxious will either get standard care or extra hypnosis support. Researchers will measure anxiety levels before and after surg…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

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 looks at anxiety related to cancer screenings in adults with Fanconi anemia, a rare condition that raises cancer risk. Researchers will observe 20 participants during their regular screening visits, noting body language, worries, and the clinic environment. No treatmen…

This study follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…

This pilot study investigates whether regularly chewing a hard gum for four weeks can produce measurable changes in facial shape. Twenty healthy adults aged 20 to 40 will chew a standardized hard gum every other day, and researchers will use 3D facial imaging to track any shifts …

This study investigates how acetaldehyde, a byproduct of alcohol, affects cells in the mouth and whether it may increase oral cancer risk. Healthy volunteers and individuals with Fanconi anemia (a condition linked to higher cancer risk) will consume a single dose of alcohol and p…

This study aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

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 checks if people with Diamond Blackfan Anemia (ages 3-30) can produce enough blood stem cells for future gene therapy. Participants receive two drugs to move stem cells from bone marrow into the blood, then researchers measure the cell count. No actual stem cell collec…

This study is building a large database of people with Diamond Blackfan anemia, a rare inherited bone marrow condition that causes anemia. Researchers will collect medical information from up to 900 participants to better understand how the disease develops and progresses. The go…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

This study is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…