PAX5-related B lymphopenia and autism spectrum disorder

This study tests whether a stem cell transplant can cure people with serious immune system disorders. Participants, aged 4 to 69, receive healthy donor stem cells to rebuild their immune system. The goal is to see if the transplant can permanently fix the immune defect and elimin…

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if…

This study tests whether a stem cell transplant from a healthy donor can help people with severe primary immunodeficiencies. Participants aged 4 to 75 receive chemotherapy before the transplant to prepare their body. The goal is to see if the procedure is safe and can improve imm…

This study will enroll 1,000 adults with both opioid use disorder and a mental health condition. Participants will receive standard medication for opioid use disorder, with some also getting a team-based support program called MISSION. The goal is to see if MISSION helps people s…

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This study tests a program called Tunawiri (meaning 'thrive') that adds mental health screening, counseling, and support to regular HIV care for pregnant and postpartum women in Kenya. The goal is to improve depression, anxiety, and HIV treatment success. Nine hundred women will …

This study tests a gamified mobile health app designed to reduce suicide risk in adults with mental disorders after they leave the hospital. Researchers will enroll 640 participants in Shenzhen, China, and use game-like features to encourage treatment adherence and follow-up visi…

This study tests whether testicular tissue frozen before puberty can be transplanted back into adult men to restore sperm production. Five men who had fertility-threatening treatments as children will receive their own preserved tissue. The goal is to see if sperm can be found in…

This study is checking whether unlicensed cord blood units are safe to use for stem cell transplants in children and adults with blood disorders. Researchers will closely watch for any infusion-related problems. The goal is to see if these units can be a safe option for patients …

This study tests whether the drug lusutrombopag can help restore platelet levels faster in patients who have had a stem cell transplant for blood disorders. About 45 adults will receive the drug after transplant, and doctors will check platelet counts at 21 days. The goal is to i…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This study looks at whether giving a longer course (10 days instead of 5) or adding another antiviral (remdesivir) to the standard Paxlovid treatment helps immunocompromised people clear COVID-19 faster. About 256 adults with mild or no symptoms will be randomly assigned to diffe…

This phase III trial tests V-IMMUNE, an intravenous immunoglobulin (IVIG) product, in about 50 people with primary immunodeficiency (PID). Participants, aged 2 and older, will switch from their current IVIG to V-IMMUNE every three weeks for a year. The study aims to see if V-IMMU…

This study tests a new antibody replacement therapy (BP-SCIG 20%) given as a weekly injection under the skin. It aims to see if it is safe and effective at preventing serious infections in people with primary immunodeficiency, a condition where the immune system cannot produce en…

This study tests whether a non-invasive brain stimulation technique called TMS can help people with mental illness quit smoking. Forty daily smokers with psychiatric conditions will receive either TMS or standard care. The goal is to see if TMS is practical and effective in real-…

This study is for adults with blood disorders who are getting a stem cell transplant from a matched donor. Researchers want to see if giving a special potato starch along with a drug that lowers iron (deferasirox) can help prevent serious complications like graft-versus-host dise…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study tests a program called SKILLS that focuses on common factors across different mental health conditions, rather than treating each disorder separately. About 60 adults with stable psychiatric diagnoses will take part. The goal is to see if this approach reduces symptoms…

This study tests a program called SUPERCEDE, which provides intensive case management to veterans who are at risk for suicide and receive care both from the VA and outside community providers. The goal is to improve care coordination, reduce suicidal thoughts, and improve daily f…

This pilot study tests whether adding ear acupressure (small seeds taped to ear points) to standard nicotine patches or gum can reduce cravings, withdrawal symptoms, and anxiety in psychiatric inpatients who must stop smoking during their hospital stay. Sixty participants will be…

This study is testing whether a virtual reality version of a therapy exercise is practical and helpful for adults (ages 18-65) with psychiatric disorders. Participants will try one VR session and answer questions about their experience. The goal is to see if this approach is acce…

This study tests an AI chatbot called Aurora that guides people through creating a personal life story to improve emotional well-being and identity. It includes healthy adults, adults with mental health conditions in residential care, and older adults aged 65+. The chatbot is not…

This study is testing a type of talk therapy called Emotion Regulation Therapy (ERT) delivered over video for people in New York who struggle with worry, overthinking, or being hard on themselves. The goal is to see if the therapy helps people better manage their emotions and fee…

This study tests whether a team of a psychologist, speech therapist, and motor therapist can help children aged 2-7 with neurodevelopmental disorders eat more foods and have fewer mealtime struggles. 24 children will be split into two groups: one gets the team therapy, the other …

This study looks at how well and how safely a new sleep medication, lemborexant, works for people who have both a mental health condition and trouble sleeping. About 121 adults will take the drug for 8 weeks, and researchers will track changes in their sleep quality and any side …

This study looks at whether watching Netflix or Disney shows in a small group for 90 minutes each week, over 6 weeks, can improve emotional awareness and reduce anxiety and depression. About 70 adults who have been in mental health treatment for at least 6 months will take part. …

This study tests a multi-sensory therapy called Heal, which uses calming sounds, gentle vibrations, and soft lighting to help detained youth (ages 12-18) manage their emotions before counseling sessions. The goal is to see if this brief, non-verbal intervention improves emotional…

This study tests whether sailing can help people with psychiatric disorders feel better. Forty adults in France will either take sailing lessons or continue their usual care. Researchers will measure changes in quality of life, mental well-being, and self-esteem to see if sailing…

This study tests a new approach called ENGAGE, where children with disabilities (like autism or cerebral palsy) help set their own therapy goals. About 96 kids aged 5-12 and their therapists will be split into two groups: one using ENGAGE and the other using usual therapy. Resear…

This study tests whether a speech therapy that involves parents can improve early social and communication skills in children aged 6 months to 5 years with neurodevelopmental disabilities. One group gets the parent-focused therapy, while the other gets standard speech therapy. Re…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

This study is looking for the genes that cause certain immune system problems. Researchers want to understand why some people get sick and others don't, even in the same family. Up to 5,000 people with immune disorders and their relatives will give blood or cheek cell samples for…

This study will analyze DNA from up to 20,000 people, including those with immune disorders and their relatives, to find genetic variants linked to conditions like atopy, autoimmunity, and primary immunodeficiency. Researchers will also study how best to return genetic results to…

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study aims to find the genetic causes of inherited immune problems that affect white blood cells called lymphocytes. Researchers will review medical records and collect blood samples from up to 5,000 people with suspected immune disorders, as well as their relatives. The goa…

This study follows 450 babies born with serious heart defects to find early signs of developmental delays by 6 months of age. Researchers want to understand why over half of these children face learning, thinking, or behavior challenges later in life. The goal is to identify risk…

This study looks at how genetic immune system problems might lead to gum disease and other mouth issues. Researchers will collect samples like saliva, plaque, and blood from 700 people, including those with immune defects, healthy adults with gum disease, and healthy adults witho…

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

This study aims to learn more about childhood-onset behavioral, psychiatric, and developmental disorders by observing people of all ages who have these conditions, along with their family members. Researchers will collect medical histories, perform psychiatric assessments, and ma…

This study aims to collect blood, saliva, and other samples from up to 3,000 people with primary immunodeficiency (PID) or their relatives. Researchers will analyze the samples to find genetic causes of PID. The goal is to better understand these inherited immune disorders, which…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study compares two ways to help adults with mental health issues get follow-up care. One group gets usual care after an emergency room visit, while the other uses a new system that books a psychiatrist appointment within 48 hours through their general doctor. Researchers wil…

This study aims to learn more about primary immunodeficiency disorders (PIDs), which weaken the immune system and make people more prone to infections. Researchers will compare genetic and health data from people with PIDs, their healthy relatives, and healthy volunteers to find …

This study looks at how the brain responds to sensory inputs like sight and sound in 200 children and adults, some with neurodevelopmental disorders (NDD) and some without. Participants will have EEG recordings during sensory tasks, complete behavioral tests, and practice percept…

This study looks at whether a tool called the Decision Helper helps people with mental health conditions take a more active role in choosing their treatment. About 62 adults in Danish outpatient mental health clinics will have their consultations recorded and analyzed. The goal i…

This study looks at the mental health of 200 teenagers aged 12 to 16 who attend special educational institutes (ITEPs) in France. Researchers will use psychiatric interviews to find out how many have conditions like depression or anxiety. The goal is to better understand their ne…

This study surveys 200 adult women with primary immune deficiency diseases about their use of intrauterine devices (IUDs), tampons, and menstrual cups. Researchers want to see if these products lead to infections like pelvic inflammatory disease or toxic shock syndrome. By collec…

This study aims to learn how sleep changes as children's brains grow, by collecting sleep and development data from healthy children and those with conditions like autism or ADHD. About 244 children ages 6 months to 8 years will have overnight sleep studies, developmental tests, …

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study aims to improve suicide prevention for people who move between healthcare and the criminal-legal system. Researchers will link health records, Medicaid data, and jail booking information to identify individuals at risk and coordinate care. Over 1,000 adults with past p…

This study aims to understand the causes and progression of Common Variable Immunodeficiency (CVID) and related inborn errors of immunity. Researchers will observe up to 500 patients over time using blood tests, imaging, and other exams. The goal is to find better ways to diagnos…

This study aims to find new genetic causes of primary immunodeficiency, a condition where the immune system doesn't work properly, leading to frequent or severe infections. Researchers will compare the genes of 500 people with suspected immunodeficiency, their relatives, and heal…

This study looks at how well vaccines against respiratory viruses (like flu or COVID-19) work in people with weakened immune systems, including those with blood cancers. Researchers will measure both antibody and T cell responses after vaccination. The goal is to understand why v…

This study aims to better understand primary immune deficiencies (PID), a group of diseases where the immune system doesn't work properly. Researchers will collect blood, stool, urine, and tissue samples from 300 people with PID and healthy volunteers to analyze immune responses.…

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study looks at medical records of 1000 children with conditions like autism, epilepsy, and Down syndrome to find common biological patterns. Researchers want to understand what causes these disorders and how children respond to treatments. The goal is to improve diagnosis an…

This study gathers basic health information from 2000 people receiving stem cell transplants for blood cancers or immune disorders. The goal is to track survival and complications to help doctors and researchers improve future treatments. Participants must be getting a standard t…

This study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

This study looks at ways to help families show up for their scheduled appointments at clinics for autism and other neurodevelopmental disorders. Researchers will test different types of reminder messages and see if having a patient navigator call high-risk families makes a differ…

This study looks at whether a program with a nurse case manager can help people leaving prison visit a doctor or clinic instead of the emergency room. About 488 adults with a history of substance use and a chronic health condition will take part. Some will get extra support from …

This study looks at how gut bacteria and the immune system interact in children and adults receiving stem cell transplants or CAR-T therapy. Researchers will collect stool and blood samples over time to find patterns linked to complications like infections or graft-versus-host di…

This study looks back at medical records of about 100 people in Japan with primary immunodeficiency who received Cuvitru. The goal is to track serious side effects like allergic reactions, blood clots, and meningitis. No new treatment is given—researchers are simply analyzing exi…

This study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

This study aims to find the genetic causes behind certain allergic and immune system disorders. Researchers will collect blood, saliva, and tissue samples from up to 10,000 people of all ages, along with some family members. No treatments are given; the goal is simply to learn mo…

This study follows 102 women who had fertility preservation before cancer treatment to see what happens long-term. Researchers want to know if they get pregnant naturally, use stored eggs or tissue, or decide not to have children. The goal is to improve support and care for femal…

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study observes 100 people with rare genetic neurodevelopmental disorders like Baker Gordon Syndrome and Syt-1 disorder. Researchers will track symptoms, collect genetic and imaging data, and create cell models to better understand these conditions. The goal is to improve dia…

This study is creating a collection of blood samples from 2,000 people with various blood disorders. The samples will be stored and used for future research approved by an ethics board. No treatments or drugs are being tested, and participants continue their normal care.

This study looks at whether a recovery-focused program can help people with severe mental illness living in supported housing. About 72 participants will either receive the new program or standard care. The goal is to see if focusing on personal goals improves daily functioning a…

This study looks at families who carry a change in the DDX41 gene, which may raise the risk of blood cancers like leukemia. Researchers will collect health questionnaires and saliva samples from up to 910 people to track who develops blood problems and when. The goal is to better…

This study looks at how JAK-inhibitor drugs affect the immune system and tests a gene therapy approach in cells from patients with primary immunodeficiency diseases. Researchers will collect blood samples from 20 adults to measure immune cell changes and gene editing efficiency. …

This study looks at using a needle placed into the bone marrow (intraosseous infusion) to give fluids and medicines quickly to critically ill patients with blood diseases. Researchers will enroll 52 adults aged 18-65 to see how often the first attempt works and how safe it is. Th…

This study follows 1,000 people who have certain genetic changes (hnRNP and others) that may cause developmental delays, autism, seizures, or muscle problems. Researchers collect medical records, school reports, and questionnaires to see how these gene variants affect behavior, t…

This study looks at how blood cells interact and what goes wrong in inherited bleeding disorders like Glanzmann thrombasthenia. Researchers will analyze blood samples from up to 60 healthy volunteers and patients to find genetic defects. The goal is to better understand these con…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study is creating a program called START-VET to help veterans with mental illness or substance use disorders who have recently left prison. Researchers will work with 20 veterans to develop transition materials and tools. The goal is to improve quality of life and reduce cri…

This observational study follows 300 adults receiving strategic psychotherapy—a short-term, goal-focused talk therapy—in private practice. Participants fill out questionnaires about their symptoms and well-being before and after treatment. The goal is to see if this therapy leads…

This study tests whether a child-focused goal-setting method called ENGAGE helps children with neurodevelopmental disabilities do better in therapy. About 96 kids aged 5-12 and their therapists will be split into two groups: one using ENGAGE and one using usual care. Researchers …

This study looks at whether a quick ultrasound of the thigh muscle can detect muscle loss (sarcopenia) in people who have finished cancer treatment. Researchers will compare the ultrasound results with a standard whole-body scan (DEXA) in 55 adults aged 18-74 with certain cancers…

This pilot study is testing whether a simple saliva test can help doctors prescribe mental health medications more safely and effectively. 200 adults in Manitoba who are starting or changing psychiatric drugs will provide a DNA sample. The results will be shared with their doctor…

This study is collecting blood, urine, and stool samples from 500 children with autoimmune diseases and healthy controls. Researchers will analyze these samples to find molecular patterns that could help diagnose these conditions and guide treatment decisions. The goal is to crea…

This study looks at whether training psychiatric care teams to talk about sexual health with young patients (ages 15 and up) increases those conversations. Researchers will survey 770 teens hospitalized for psychiatric disorders about their sexual health and any side effects from…