Hereditary disorder of connective tissue

This trial tests whether a single infusion of IM19 CAR-T cells can calm severe lupus that has not responded to standard treatments. Six adults with refractory systemic lupus erythematosus will receive the engineered immune cells, which are designed to target and reset the faulty …

This study tests whether the Butantan-Dengue vaccine is safe and works well in people aged 12–59 with autoimmune rheumatic diseases (like rheumatoid arthritis or lupus) who are stable on low or no immune-suppressing drugs. Participants receive one vaccine dose and are monitored f…

This study is testing the meningococcal B vaccine (Bexsero) in children and young adults aged 2 to 25 who have autoimmune rheumatic diseases like juvenile arthritis or lupus. Researchers want to see if the vaccine triggers a strong immune response and if it is safe, especially fo…

This study tests the safety and immune response of the 20-valent pneumococcal vaccine (PCV20) in children, adolescents, and young adults aged 2-25 with autoimmune rheumatic diseases like juvenile arthritis. All 114 participants will receive one dose of PCV20, and researchers will…

This early-phase trial is testing an injection called GT719 in 10 adults with moderate to severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the therapy might h…

This early study tests baricitinib, a drug already approved for other immune conditions, in 20 people with Job syndrome who also have lupus-like disease or eczema. Participants take the pill daily for 6 months. The main goal is to see if it is safe and tolerable, while also check…

This Phase 1 trial tests surovatamig, a bispecific T-cell engager, in 48 adults with rheumatoid arthritis or systemic lupus erythematosus. The study gives the drug as injections in increasing doses to check safety, side effects, and how the body processes it. It is too early to k…

This study tests a new oral drug, QTX-2101, combined with standard ATRA therapy for adults newly diagnosed with a low-risk form of acute promyelocytic leukemia (APL). The goal is to see if the oral capsule works as well as the current intravenous treatment. About 150 participants…

This study tests whether giving donor white blood cells (lymphocytes) soon after a stem cell transplant can lower the chance of cancer returning in people with high-risk blood cancers like leukemia or lymphoma. Participants receive chemotherapy, a stem cell transplant, and then a…

This phase 2 trial tests whether the drug olverembatinib can safely control chronic myeloid leukemia (CML) in people who are newly diagnosed. About 50 adults will take the drug by mouth. Researchers will track side effects and how well the drug works. The study is currently recru…

This early-stage study tests a new drug, PRT12396, in about 100 people with polycythemia vera or myelofibrosis—rare blood cancers. The main goals are to find a safe dose and check for side effects. Researchers will also look for signs that the drug controls the disease, but this …

This study tests whether anifrolumab, a drug that blocks certain immune signals, can improve blood vessel function and reduce inflammation in people with lupus. About 45 adults with lupus will receive either the drug or a placebo over 8 months. Researchers will measure changes in…

This phase 2 trial tests enasidenib, a daily pill that targets the IDH2 gene mutation, in 40 adults with rare cancers of the nasal cavity or skull base that have spread or come back. Participants take the drug once daily in 28-day cycles, and researchers will track how long the c…

This early-phase trial tests a personalized treatment for people with blood cancers like leukemia. Doctors take a patient's own T cells, modify them in the lab to recognize unique cancer mutations, and infuse them back after a short course of chemotherapy. The goal is to see if t…

This early-stage trial is testing whether two drugs that lower metal levels in the body can help people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) respond better to chemotherapy. The study will enroll 58 adults and aims to find the safest dose and check f…

This study tests whether adding a patient's own concentrated bone marrow cells to a standard hip surgery (core decompression) can prevent the hip ball from collapsing and delay or avoid the need for a hip replacement. It includes 192 adults with early-stage osteonecrosis (before …

This study tests whether giving a chemotherapy drug (decitabine) plus a targeted therapy (ruxolitinib, fedratinib, or pacritinib) before a stem cell transplant helps people with advanced myeloproliferative neoplasms (MPN) get to transplant and have better outcomes. About 25 adult…

This study tests an experimental oral drug called BI 3000202 in 405 adults with moderate to severe systemic lupus erythematosus (SLE). Participants are randomly assigned to one of four doses of the drug or a placebo, taken daily for a year alongside their usual lupus treatments. …

This early-phase trial tests a new type of cell therapy called BCMA-CD19-IL-15/IL15sushi compound CAR T cells in 20 people with severe lupus that hasn't improved with standard treatments. The therapy aims to reset the immune system by targeting and eliminating faulty immune cells…

This early-stage study tests an experimental oral drug, AJ1-11095, in about 76 adults with myelofibrosis (a type of bone marrow cancer) whose disease has stopped responding to standard JAK2 inhibitor therapy. The main goals are to check the drug's safety, find the best dose, and …

This phase 2 trial tests a new drug called SG301, given as a shot under the skin, for people with systemic lupus erythematosus (SLE). About 120 participants whose lupus is not well controlled by standard treatments will receive either SG301 or a placebo over several months. The g…

This study tests a drug called ponatinib in 50 adults with chronic myeloid leukemia (CML) whose cancer did not respond to or they could not tolerate other targeted therapies. The goal is to see if ponatinib can control the disease by blocking a protein that helps cancer cells gro…

This study is for people who have both an autoimmune disease (like rheumatoid arthritis, lupus, or multiple sclerosis) and an advanced cancer that has spread or cannot be removed by surgery. It tests the safety and effectiveness of the immunotherapy drug nivolumab, given alone or…

This Phase 1 trial tests the safety and tolerability of a new drug called AZD5492 in 72 adults with lupus, myositis, or rheumatoid arthritis. Participants receive one or two doses under the skin and are monitored for side effects and how the drug moves through the body. The study…

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome (MDS) who also have anemia. The goal is to see if the drug can reduce the need for blood transfusions and increase red blood cell levels. The study is in earl…

Researchers are comparing olverembatinib, an experimental drug, with bosutinib, an approved drug, in adults with chronic phase chronic myeloid leukemia (CML-CP). The trial enrolls patients whose disease has not responded well to at least two prior targeted treatments. A separate …

This phase 2 trial is testing a new treatment called FT819 for people with lupus nephritis, a serious kidney complication of lupus. FT819 is made from donor immune cells that are engineered to target and destroy faulty B cells. The study will enroll 53 participants and aims to se…

This early-stage trial is testing two experimental drugs, vonsetamig and odronextamab, in people with lupus nephritis that has not responded to standard treatments. The main goal is to check safety and side effects over the long term. Researchers will also see if the drugs can he…

This early-phase trial tests whether giving patients a special infusion of donor immune cells (with certain T and B cells removed) after a stem cell transplant is safe and can help prevent their blood cancer from coming back. The study enrolls high-risk patients with hard-to-trea…

This early-stage study tests a single dose of ABBV-519 in 30 adults with moderate lupus or rheumatoid arthritis. Researchers will check how the drug moves through the body, how it affects immune cells, and whether it is safe. Participants will be followed for about a year to moni…

This early study tests a new medicine, MK-1045, in about 21 people with lupus or rheumatoid arthritis. The main goal is to see if it is safe and tolerable at different dose levels. Participants receive the drug through an IV infusion, and researchers monitor for side effects.

This study tests the drug nirogacestat in 50 premenopausal women with desmoid tumors (a type of non-cancerous but aggressive growth). The main goal is to see if the drug affects ovarian function, such as menstrual periods and hormone levels. Participants take nirogacestat orally,…

This study tests a drug called enpatoran for people with lupus who have active skin rashes or lesions. About 202 participants will take either enpatoran or a placebo twice daily for 24 weeks, alongside their usual treatments. The goal is to see if the drug can significantly impro…

This study is testing whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (a common complication where donor cells attack the patient's body) while still being effective. The trial involves 260 adults with b…

This study tests a standardized, optimized approach to cord blood transplantation for adults with high-risk blood cancers like leukemia and lymphoma. The method includes specific chemotherapy, radiation, and stem cell dosing, followed by careful monitoring. Researchers hope to im…

This study tests an experimental pill called enpatoran for people with lupus who have active skin rashes. About 202 participants will receive either the drug or a placebo for 24 weeks, while continuing their usual treatments. The goal is to see if enpatoran can significantly redu…

This study tests a two-step approach for desmoid tumors, which are non-cancerous but can be painful and grow. First, patients take a daily pill called nirogacestat for three months. Then, one tumor is frozen with a special needle (cryoablation). The goal is to see if this combina…

This early-stage study tests a new treatment called ALLO-329 for people with lupus, myositis, or scleroderma. The therapy uses specially engineered immune cells from a donor to target and calm the overactive immune system. The main goals are to check safety and see if it can impr…

This phase 3 study tests anifrolumab, a biologic drug given by IV, in 100 children aged 5 to 18 with moderate to severe systemic lupus erythematosus (SLE). Participants continue their standard care and are randomly assigned to receive anifrolumab or a placebo. The goal is to see …

This study tests a new treatment called AZD0120 for people with severe lupus that hasn't improved with standard therapies. AZD0120 is a type of CAR T cell therapy that targets two proteins (CD19 and BCMA) on immune cells to calm the overactive immune system. The trial will enroll…

This study tests a medicine called anifrolumab for people with lupus who have not taken strong immune-suppressing drugs before. The goal is to see if the drug can help patients reach remission and reduce or stop their steroid use. About 245 adults will receive weekly injections f…

This study offers continued treatment with deucravacitinib for people with systemic lupus erythematosus (SLE) or related skin lupus who finished earlier trials. It aims to keep the disease under control by providing ongoing access to the medication. Only 35 participants are being…

This early-stage study tests a new drug called SAR448501 (DR-0201) in 62 adults with lupus or rheumatoid arthritis. The drug is a bispecific antibody designed to target the immune system. The main goal is to check safety and find the right dose, not yet to prove it works. Partici…

This early-stage trial will test the safety and tolerability of a new drug, cevostamab, in 46 people with systemic lupus erythematosus, some of whom also have active lupus nephritis. The drug is given intravenously, and researchers will monitor side effects and how the drug moves…

This phase 3 trial tests whether adding the drug sapablursen to standard treatment helps people with polycythemia vera, a blood disorder causing too many red blood cells. About 250 participants who currently need regular blood removal (phlebotomy) will receive either sapablursen …

This phase 3 trial tests whether belimumab (Benlysta) can stabilize or improve lung function in adults with interstitial lung disease caused by connective tissue diseases like rheumatoid arthritis or lupus. About 440 participants will receive either belimumab injections or a plac…

This study tests an experimental drug called dapirolizumab pegol for people with moderate to severe lupus. About 450 participants will receive either the drug or a placebo, added to their usual treatments. The goal is to see if the drug can improve lupus symptoms over 48 weeks.

This phase 3 trial tests the drug radotinib in adults with chronic-phase chronic myeloid leukemia (CML) who have not responded to or could not tolerate earlier treatments such as imatinib. Participants take radotinib twice daily, and researchers measure how well it reduces leukem…

This study is watching how well asciminib works and how safe it is in adults with a type of blood cancer called chronic myeloid leukemia. It includes people who are newly diagnosed or have already tried one other treatment. The main goal is to see how many patients reach a very l…

This early-stage study tests an experimental drug called LY4298445 in healthy volunteers and people with lupus or rheumatoid arthritis. The main goal is to check safety and how the body handles the drug. Participants will receive either the drug or a placebo, and the study lasts …

This early-phase trial is testing whether adding abemaciclib to the standard drug ruxolitinib is safe and effective for people with myelofibrosis, a type of bone marrow cancer. The study will enroll 18 adults who have been on ruxolitinib for at least 12 weeks. Researchers will mo…

This trial tests an investigational cell therapy called FT839 in people with active autoimmune diseases, including ANCA-associated vasculitis, inflammatory myositis, rheumatoid arthritis, lupus, and systemic sclerosis. Participants receive FT839 as two intravenous infusions, some…

This study tests a drug called asciminib in children aged 1 to 18 with a specific leukemia (Ph+ CML-CP). The goal is to see if the drug can control the cancer by reducing the number of cancer cells. About 50 children will take the drug, and researchers will check how well it work…

This trial tests rusfertide, an injected drug, in Japanese adults with polycythemia vera, a rare blood cancer that causes too many red blood cells. The goal is to see if rusfertide can keep red blood cell levels under control and reduce the need for phlebotomy (blood removal). Al…

This early-stage study is testing a new drug called cizutamig in about 47 adults with systemic lupus erythematosus (SLE), a chronic autoimmune disease. The main goal is to check if the drug is safe and tolerable, and to see how it moves through the body. Participants must have ac…

This study tests a specially engineered donor graft called Orca-Q in children and young adults with blood cancers who need a stem cell transplant. The graft is designed to help the new cells take hold and reduce complications. The trial is currently on hold, but aims to enroll 40…

This study follows 200 adults with lupus who are starting anifrolumab for the first time, to see how well it works in everyday clinics. Researchers will measure disease activity, skin symptoms, fatigue, and quality of life over time. The goal is to provide real-world evidence to …

This phase 2 trial compares a drug combo (ASTX727 plus iadademstat) against ASTX727 alone for people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The study aims to see if adding iadademstat improves complete response rates. However, the trial …

This study is tracking 125 adults with lupus who are starting a new drug called anifrolumab, given as a shot under the skin. Researchers want to see how many patients achieve remission (no active disease) after 24 months of treatment in real-world clinics, not just in strict rese…

This trial tests two ways to correct flat head syndrome in infants: caregiver-led repositioning and a custom-fit helmet. Researchers will measure head shape changes in treated infants and compare them with a group of typically developing infants. Infants with torticollis will als…

This early-stage trial tests a single infusion of CRISPR-edited immune cells (CTX112) from a healthy donor in 80 adults with severe lupus, scleroderma, or myositis that hasn't responded to standard treatments. The goal is to see if the cells are safe and can reduce disease activi…

This study follows 218 adults with systemic lupus erythematosus (SLE) in Italy who are starting anifrolumab (Saphnelo) for the first time. Researchers want to see if the drug helps patients reach a state of low disease activity while taking 5 mg or less of steroids daily. The stu…

This phase 3 study tests whether nipocalimab can help control moderate to severe systemic lupus erythematosus (SLE), a chronic autoimmune disease. About 600 adults will receive either nipocalimab or a placebo, on top of standard treatments. The main goal is to see if more people …

This study is checking how well the Z1 Femoral Hip System works and how safe it is for people who need hip replacement surgery. About 150 adults with hip problems like arthritis or fractures will be followed for 2 years. The main goal is to see how many implants are still in plac…

This study is testing an oral medication called asciminib in children aged 1 to 18 with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) who have already tried other treatments. The goal is to find the right dose that works as well as it does in adults and t…

This study tests a new antibody called axatilimab, given with or without the chemotherapy drug azacitidine, for people with advanced myeloproliferative neoplasms (MPN), MPN/MDS overlap, or high-risk chronic myelomonocytic leukemia (CMML). The goal is to find the safest dose and s…

This early-stage study tests a single injection of a new drug, LBL-047, in healthy adults and people with mild-to-moderate systemic lupus erythematosus (SLE). The main goal is to check safety and side effects. About 112 participants will receive either the drug or a placebo. This…

This early-stage trial is testing a treatment called CABA-201 for people with active systemic lupus erythematosus (SLE), including those with lupus nephritis. CABA-201 is made from a patient's own immune cells, which are modified to target and temporarily remove B cells—a key dri…

This study tests whether combining dasatinib with ropeginterferon can better control chronic myeloid leukemia in its early phase. About 40 adults newly diagnosed with CML will receive the drug pair to see if it improves disease control and reduces side effects. The goal is to man…

This phase 2 trial is testing whether a drug called asciminib, when added to standard therapy, can help people with chronic myeloid leukemia (CML) achieve a second treatment-free remission. Participants are adults with CML who previously tried stopping their medication but had to…

This study tests a drug called tegavivint in children, teens, and young adults whose solid tumors have come back or stopped responding to treatment. The goal is to find the safest dose and see if it can shrink tumors by blocking a key growth signal inside cancer cells. About 147 …

This phase 2 trial tests whether adding metal detoxification drugs (DMSA and Ca-EDTA) to standard therapy can improve outcomes for adults with intermediate- to high-risk acute myeloid leukemia (AML). Researchers believe lowering metal levels in blood and bone marrow may help cont…

This early-phase study tests a new treatment called Meta10-19 for people with moderate to severe systemic lupus erythematosus (SLE). The therapy uses a patient's own immune cells, modified to better target and destroy faulty B cells that drive the disease. The main goals are to f…

This early-phase study tests the drug TURALIO (pexidartinib) in people aged 3 to 35 whose cancer has not responded to standard treatments. The goal is to find a safe dose and see if it can shrink tumors or control leukemia. Participants take a daily capsule for up to 2 years, wit…

This study tests a one-time treatment called Obe-cel, made from a patient's own immune cells, for severe lupus nephritis that hasn't improved with standard care. About 35 people aged 12 to 65 will receive the infusion and be monitored for up to 24 months. The main goal is to see …

This study tests a new drug called Lu AG13909 in 18 adults with Cushing's disease, a rare condition where the body produces too much cortisol. The goal is to see if the drug can safely lower cortisol levels to normal. Participants will receive the drug through an IV or injection,…

This phase 2 trial tests whether the experimental drug E6742 can reduce lupus disease activity better than a placebo. About 256 adults with lupus will take either E6742 or a placebo pill for 24 weeks. The main goal is to see if more people on E6742 achieve a meaningful reduction …

This study tests a drug called mosunetuzumab in 30 people with lupus, a disease where the immune system attacks the body. The goal is to see if the drug can put lupus into remission so that participants can stop most lupus medications for at least 6 months. However, some people m…

This study looks at whether people aged 60 and older with stable lupus can safely stop taking hydroxychloroquine, a common medication that can cause eye damage over time. About 330 participants will either continue the drug or switch to a placebo and be monitored for one year. Th…

This phase 2 trial is testing whether adding the drug pacritinib to standard treatments (azacitidine or decitabine) can help more people with advanced myeloproliferative neoplasms (MPN) get a stem cell transplant. The study will enroll 27 adults whose MPN has progressed to a more…

This early-stage trial is testing an investigational drug called BEBT-507 in adults with polycythemia vera, a condition where the body makes too many red blood cells. The study aims to find a safe dose and see if the drug can help control blood cell levels, potentially reducing t…

This trial tests whether a single intravenous dose of mesenchymal stem cells, derived from bone marrow, can safely help adults with active lupus nephritis achieve a complete or partial kidney response. Participants receive either the stem cells or a placebo (saline) in a double-b…

This study tests a program called CO-LEAD that trains doctors to use effective communication strategies and a patient survey to identify barriers to taking lupus medications. Researchers will enroll 480 patients and their clinicians at two academic centers. The goal is to see if …

This trial tests whether adding voclosporin to standard lupus nephritis treatment (mycophenolate mofetil and prednisolone) reduces kidney inflammation faster and more completely than standard treatment alone. Adults with active lupus nephritis will be randomly assigned to either …

This early-stage study tests a new therapy, HN2301, in 30 adults with lupus, scleroderma, or rheumatoid arthritis. The main goal is to check safety and find the right dose. Researchers will also look for early signs that the treatment helps control the disease by measuring immune…

This study tests an investigational drug called nirogacestat in about 40 adults with desmoid tumors (a type of non-cancerous growth). The main goal is to check the drug's safety and side effects. Researchers will also see how the tumor responds to treatment.

This early-stage study tests a new drug called IASO-782 in 20 adults with systemic lupus erythematosus (SLE). The main goal is to see if the drug is safe and tolerable. Participants will receive either the drug or a placebo, and researchers will monitor for side effects.

This study tests a new treatment for people with blood cancers like leukemia, lymphoma, and multiple myeloma that have returned or not responded to treatment. Participants receive chemotherapy followed by specially engineered natural killer (NK) cells from donated cord blood. The…

This early-phase study tests a new drug called budoprutug in 30 adults with active lupus who haven't responded well to standard treatments. The drug targets and depletes certain immune cells (B cells) to help control the disease. The main goals are to check safety and how the bod…

This study tests whether a supplement called nicotinamide riboside (NR) can reduce inflammation and improve immune cell function in women with mild-to-moderate lupus. About 78 participants will receive either NR or a placebo for 12 weeks. Researchers will measure changes in immun…

This study tests if a new drug called cenerimod can help people with lupus who have active kidney disease (lupus nephritis). About 300 adults will take either cenerimod or a placebo daily for 76 weeks, on top of their usual treatments. The main goal is to see if cenerimod improve…

This study tests a new type of CAR T-cell therapy that targets two proteins, CD19 and CD22, on cancer cells. It is for children and young adults aged 3 to 39 with B-cell cancers like leukemia or lymphoma that have not been cured by standard treatments. Participants receive their …

This phase II trial is testing whether abemaciclib, a targeted cancer drug, can slow the growth of advanced bone and soft tissue sarcomas that have a specific alteration in the CDK pathway. Participants take the pill twice daily until their disease worsens or side effects become …

This study tests an experimental tablet called VENT-03 for people with active cutaneous lupus erythematosus (CLE), a condition causing painful skin rashes. Twenty-four adults will take either VENT-03 or a placebo for 4 weeks, then everyone gets VENT-03 for 8 more weeks. Researche…

This early study tests a new treatment called CD19/BCMA CAR-T for people with severe autoimmune diseases like lupus and Sjögren's syndrome that haven't improved with standard care. The treatment involves taking a patient's own immune cells, modifying them to target specific immun…

This study tests whether a culturally tailored coaching program delivered through the Health360x app can help women of color with lupus or lupus nephritis better manage their disease. The app provides educational micro-learning modules, push notifications, and tools to track heal…

This study tests a special cord blood transplant for people with high-risk blood cancers like leukemia and lymphoma. Participants get chemotherapy and radiation before the transplant to kill cancer cells and prepare the body. After the transplant, drugs are given to prevent the n…

This trial compares two starting doses of the steroid prednisone for children with lupus-related kidney inflammation (lupus nephritis). The standard high dose can cause serious side effects like growth problems and bone thinning. The study asks whether a lower initial dose can co…

This early-stage study tests an experimental drug, INCA036978, in about 218 adults with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check the drug's safety and find the best dose, either alone or with standard treatment. Participants must have a l…

This study compares two methods to control bleeding during laparoscopic myomectomy (surgery to remove fibroids from the uterus). One method uses a temporary tourniquet to block blood flow, and the other injects a medicine called epinephrine into the uterus to narrow blood vessels…

This study investigates whether a 6-week anti-inflammatory diet can improve pain and fatigue in people with systemic lupus erythematosus (SLE). Participants follow a specially designed diet while researchers track changes in patient-reported outcomes like pain interference and fa…

This study tests whether temporarily stopping methotrexate for 2 weeks after receiving a pneumonia vaccine (PCV20) helps the vaccine work better in adults with autoimmune rheumatic diseases like rheumatoid arthritis or lupus. About 192 participants with stable, low disease activi…

This early-stage trial tested the safety of a new drug called NTR-1011 in 36 healthy adults and people with lupus or rheumatoid arthritis. The study aimed to find a safe dose and see how the drug behaves in the body. It is a first step toward possibly developing a new treatment f…

This phase 2 trial tests fimepinostat, a drug that blocks two cancer-related pathways, in adults with Cushing disease caused by a pituitary tumor. The goal is to see if the drug can lower cortisol levels to normal within four weeks. The study compares two doses and includes patie…

This early-stage study tests a new drug, CPTX2309, to see if it is safe and tolerable. It involves 64 healthy adults and people with moderate to severe rheumatoid arthritis or lupus. Researchers will monitor side effects, lab results, and immune responses to determine the drug's …

This phase 1 trial tests the safety and tolerability of pacritinib, a pill that blocks JAK2 and IRAK1, in people with VEXAS syndrome—a rare, severe autoinflammatory disorder caused by a UBA1 gene mutation. Up to 15 participants will take pacritinib daily for up to 12 cycles, star…

This early-stage trial tests an experimental cell therapy called NEUK203-215 in adults aged 18-65 with severe autoimmune diseases like lupus or scleroderma that have not responded to other treatments. The therapy uses immune cells from a healthy donor, engineered to target and at…

This phase 1 trial tests a new drug called GNC-038, a tetra-specific antibody designed to target multiple pathways involved in lupus. The study includes adults with moderate to severe lupus who are on stable standard treatment. Researchers are evaluating the drug's safety, tolera…

This study tests a drug called blinatumomab in infants under 1 year old who have a specific, hard-to-treat type of leukemia (KMT2A-rearranged). The goal is to improve how long these babies stay cancer-free and to help them live longer. About 160 infants from around the world will…

This study looks at whether people with rheumatic diseases (like rheumatoid arthritis or lupus) can safely stop their immunosuppressant medications for a shorter time before elective shoulder replacement surgery. The goal is to see if this approach lowers the chance of disease fl…

This early-stage trial will test whether a personalized immune cell therapy called CAR T-cells is safe for 8 adults with severe lupus that hasn't improved with standard treatments. Participants will receive a single infusion of their own genetically modified cells after a short c…

This study compares two surgical techniques for men with complex Peyronie's disease, a condition causing painful penile curvature. Researchers will test whether a newer, microsurgery-based method (subtunical microdissection) works better than the standard plaque incision and graf…

This phase 2 trial tests whether TQH3906, a pill that blocks a protein called TYK2, can safely control lupus symptoms. About 198 adults with lupus will receive either the drug or a placebo for up to 48 weeks. The main goal is to see if more people on the drug achieve a meaningful…

This study is testing the long-term safety of a drug called ianalumab in people with lupus who have already completed a previous study with the same drug. About 550 participants will receive either ianalumab or a placebo monthly or quarterly for up to several years. The main goal…

This study tests whether adding pioglitazone (a diabetes drug) to standard tyrosine kinase inhibitors (TKIs) can help people with chronic myeloid leukemia (CML) who lost remission after stopping TKI therapy. Participants take the combination for at least six months, then stop all…

This study tests a stem cell transplant using a reduced-intensity conditioning regimen (lower doses of chemotherapy and radiation) followed by a donor stem cell infusion. The goal is to treat various blood cancers and blood disorders while lowering the risk of graft-versus-host d…

This study follows people with chronic myeloid leukemia (CML) who have already benefited from the drug asciminib in earlier Novartis trials. Researchers want to see how safe the drug is over the long term. About 347 participants will continue taking asciminib or other similar dru…

This early-phase trial is testing a new drug called MTS109, made with mRNA technology, for people with moderate to severe autoimmune diseases like lupus and scleroderma. Fifteen participants will receive several injections over a month to see if it is safe and tolerable. The goal…

This study tests an experimental drug called IMC-002 (also known as IMM0306) in people with active systemic lupus erythematosus (SLE), a chronic autoimmune disease. The trial compares the drug against a placebo, both given alongside standard care, to see if it can reduce disease …

This phase II trial tests whether adding targeted total marrow irradiation to standard chemotherapy before a stem cell transplant can improve outcomes for people with high-risk acute myeloid leukemia, chronic myeloid leukemia, or myelodysplastic syndromes. The study will enroll 3…

This Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of…

This study tests whether a new pill (gecacitinib) plus a weekly injection (pegylated interferon) can help people with polycythemia vera, a condition where the body makes too many red blood cells. The goal is to bring blood counts back to normal and ease symptoms. Thirty adults wi…

This early-stage study tests a new drug (GSK5926371) in people with lupus and similar autoimmune diseases. The drug is designed to target and calm specific immune cells that attack the body. The main goal is to check if the drug is safe and how the body handles it, with 54 adults…

This study tests whether a 'treat-to-target' approach—where doctors adjust treatment every 6 weeks to aim for remission—works better than usual care for people with lupus. About 606 participants who are not in remission will be followed for 120 weeks. The goal is to see if this s…

This study tests if the drug sirolimus can help adults with lupus who also have very low platelet counts (a condition called immune thrombocytopenia). Participants will receive either sirolimus or a placebo for 24 weeks, then all will receive sirolimus for another 24 weeks. The g…

This study tests a new treatment called CART19 for teens and young adults (ages 12-29) with severe lupus that hasn't improved with standard treatments. CART19 uses a patient's own immune cells, modified in a lab to target and calm the overactive immune system causing lupus. The g…

This early-stage trial tests a cell therapy called vididencel in 24 adults with chronic myeloid leukemia (CML) who still have traces of cancer despite taking standard medication. The goal is to see if adding vididencel to their current treatment is safe and might eventually allow…

This study tests the drug belimumab (Benlysta) in 30 people who were diagnosed with lupus within the last two years. Participants will receive either the drug or a placebo for one year, and then some may switch to placebo in the second year. The goal is to see if early treatment …

This early-phase trial tests whether adding NK cells (a type of immune cell) to the standard drug rituximab can safely help people with severe lupus or lupus-related kidney disease. The study will enroll 18 adults in China and focus on safety and signs of disease improvement. It …

This early-phase trial is testing a new treatment called zamto-cel, a CAR-T cell therapy, for people with severe lupus or scleroderma that hasn't improved with standard treatments. The study will enroll 48 participants and primarily look at safety and side effects. Participants f…

This study looks at how patients with Familial Mediterranean Fever (FMF) and Still's disease (including systemic juvenile idiopathic arthritis and adult-onset Still's disease) use the drug canakinumab (Ilaris) over the long term. Researchers will track 160 patients across Europe …

This early-phase trial tests Arnovie101, an mRNA-based CAR-T therapy given directly into the bloodstream, in 5 people with lupus or autoimmune hemolytic anemia. The goal is to see if it is safe and how the body processes it. Researchers will also check if it reduces disease activ…

This study tests a stem cell transplant method that removes certain immune cells (T-cells and B-cells) from the donor cells. The goal is to reduce graft-versus-host disease, a serious complication where donor cells attack the patient's body. The trial includes 20 children and you…

This early-phase trial is testing a new treatment called GC012F (CD19-BCMA CAR-T cells) for people with severe lupus that hasn't improved with standard medications. The therapy uses a patient's own immune cells, modified to target and destroy faulty B cells driving the disease. T…

This early study tests a new treatment called IASO207 for people with active lupus that hasn't responded to standard therapies. The treatment works by creating special immune cells inside the body to target and calm the overactive immune system. The study will check if it's safe …

This study tests a new daily pill called JBI-802 in people with certain blood cancers (myeloproliferative neoplasms and MDS/MPN) who have too many platelets. The trial has two phases: first, finding a safe dose in about 30 adults, then checking if it works. Participants take the …

This early-stage study tests a new drug, GT801, in 22 adults with moderate to severe autoimmune diseases that haven't responded to standard treatments. The main goals are to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

This study is testing Bearing nsPVA particles, used during a procedure called uterine artery embolization, to treat uterine fibroids in 100 women. The goal is to see if these particles safely block blood flow to fibroids, reducing symptoms like pain and heavy bleeding. Participan…

This early-phase trial tests a new drug called GT801 in 22 people with moderate-to-severe autoimmune diseases that haven't responded to other treatments. The main goal is to check safety and find the right dose. Researchers will also look for signs that the drug helps control the…

This phase 2 trial is testing elacestrant, a pill that breaks down estrogen receptors, in 30 people with advanced uterine sarcomas that are fueled by estrogen. The goal is to see if the drug can shrink tumors or slow their growth. Participants must have tumors that strongly expre…

This study watches children and adults who are already taking or about to start Ilaris for hereditary periodic fever syndromes or systemic juvenile idiopathic arthritis. Researchers will track side effects and how well the drug controls symptoms over 16 weeks. No new medicine is …

This study tests a new treatment called QT-219CX, which uses specially engineered immune cells (CAR-T cells) to target and calm the overactive immune system in people with autoimmune diseases like lupus, kidney disease, and scleroderma. The treatment is given as a one-time infusi…

This pilot study tests a stem cell transplant method that removes specific immune cells (alpha/beta T cells and B cells) from the donor cells before transplant. The goal is to reduce the risk of graft-versus-host disease, a serious complication, while still allowing the transplan…

This study is for people with symptomatic desmoid tumors that are growing and cannot be treated with surgery or freezing. Researchers are testing a procedure that delivers chemotherapy (doxorubicin) directly into the tumor through a small tube in an artery, aiming to shrink the t…

This study tests if temporarily blocking the blood supply to the uterus and ovaries during minimally invasive fibroid surgery can reduce blood loss. About 120 women with large or numerous fibroids will be randomly assigned to receive the vessel-blocking technique or standard surg…

This study tests whether a cord blood transplant can help children and young adults (up to age 26) with blood cancers like leukemia and lymphoma. Participants receive the transplant as their first or second treatment. The main goal is to see how many are cancer-free one year afte…

This program provides nirogacestat to people with serious desmoid tumors or NOTCH gene mutation tumors who have no other treatment options and cannot join a clinical trial. Each case is reviewed individually by the company, ethics board, and health authorities. The goal is to con…

This phase II trial tests different drug combinations to prevent graft-versus-host disease (GVHD) in 400 blood cancer patients receiving stem cell transplants from unrelated donors. Participants will receive either thymoglobulin or a combination of cyclophosphamide with other imm…

This early-stage study tests a new treatment called HBI0101 CAR T for people with severe autoimmune diseases like lupus, multiple sclerosis, and rheumatoid arthritis that haven't improved with standard treatments. The main goal is to find a safe dose and check for side effects in…

This study tests a new medicine called MIL62 for people with systemic lupus erythematosus (SLE), a chronic autoimmune disease. About 316 adults with active lupus will receive either MIL62 or a placebo to see if it safely reduces disease activity and allows lower steroid use. The …

This study tests a new way to treat early-stage avascular necrosis (bone death) of the hip. Doctors take a patient's own bone marrow stem cells, grow them on a special bone scaffold in the lab, and then implant this 'tissue-engineered bone' into the damaged area. The goal is to s…

This Phase 2 trial is testing a drug called ES014 in 50 adults with desmoid tumors, a type of non-cancerous but often painful growth. The drug is given as an IV infusion every two weeks. The main goal is to see if the tumors shrink, while also checking safety and quality of life.

This study tests a new treatment called anti-CD19/BCMA universal CAR-T cells for people with severe autoimmune diseases like rheumatoid arthritis, lupus, and scleroderma. The therapy aims to reset the immune system by targeting and removing faulty B cells. About 72 adults will re…

This study tests whether ruxolitinib works better than current standard therapies (hydroxycarbamide or interferon alpha) for people with high-risk polycythemia vera, a blood cancer that increases red blood cells. About 586 adults aged 18 and older with high-risk features will be …

This study tests a program called Pediatric Medication Therapy Management (pMTM) for children with complex medical conditions who take five or more medications. The program involves a thorough review of all medicines, optimizing the regimen, and creating a clear medication plan. …

This early study is testing a new treatment called CD19 CAR-T therapy for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard medications. The therapy involves taking a patient's own immune cells, reprogramming them to target and …

This phase 2 trial is testing the drug asciminib in 20 adults with chronic myeloid leukemia who have a specific genetic change (T315I mutation) and cannot take the standard drug ponatinib. The goal is to see if asciminib can control the leukemia and improve outcomes. Participants…

This study follows 600 Korean adults with chronic myeloid leukemia who are taking Scemblix (asciminib) to see how safe and effective it is in everyday medical practice. Researchers will track side effects and how well the drug controls the disease at 12 and 24 weeks. The goal is …

This early-stage study tests an injection called GT719 in 30 people with moderate to severe autoimmune diseases like lupus that haven't responded to standard treatments. The main goal is to check safety and find the right dose, while also looking at whether it helps control disea…

This study tests a new treatment for adults with severe lupus that hasn't improved with standard therapies. Doctors will use a person's own immune cells, modified to target specific lupus-driving cells (CD19 or BCMA), and give them back as a one-time infusion. The goal is to cont…

This early-phase study tests a new automated method (CliniMACS Prodigy) to select CD34 stem cells for patients with blood cancers who need a stem cell boost or second transplant. The goal is to reduce the risk of severe graft-versus-host disease (GVHD) while improving blood cell …

This study compares two surgical techniques to reduce blood loss during open fibroid removal. Forty women with symptomatic uterine fibroids will be randomly assigned to either bilateral uterine artery ligation or a pericervical mechanical tourniquet. The goal is to see which meth…

This study follows 120 Israeli children and teens with type 1 diabetes who use the Medtronic 780G system, a closed-loop insulin pump that automatically adjusts insulin. Researchers will track blood sugar control, sleep, diet, and emotional well-being over two years. The goal is t…

This study looks at whether offering personalized follow-up, including video visits, helps more people with pseudoxanthoma elasticum (PXE) get proper care. PXE causes calcium buildup in skin, eyes, and blood vessels, leading to vision loss and other problems. The researchers will…

This study tests an experimental oral drug called cirtuvivint for people with advanced soft-tissue sarcoma that has not responded to prior treatment. About 25 participants in Spain will take the drug to see if it can stop or slow cancer growth for at least 3 months. The drug targ…

This early-phase trial is testing a new type of cell therapy called QT-219C, which uses donor-derived CAR-T cells to target and destroy faulty immune cells in people with severe autoimmune diseases like lupus, nephrotic syndrome, and vasculitis. The study will enroll 15 participa…

This study follows about 400 people with certain blood cancers (like essential thrombocythemia and myelofibrosis) who are already taking bomedemstat and benefiting from it. The goal is to collect more information on the drug's long-term safety and how well it controls the disease…

This study tests whether adding asciminib to standard tyrosine kinase inhibitors (TKIs) can help people with chronic myeloid leukemia (CML) safely stop all medication and remain in remission. It includes 69 adults who have been on TKIs for at least 5 years but previously failed t…

This study tests whether giving a Chinese herbal medicine called Realgar-Indigo naturalis formula (RIF) together with standard vitamin A therapy right away can lower the risk of early death in people with a fast-growing blood cancer called acute promyelocytic leukemia (APL). Abou…

This study looks at how well and how safely the drug asciminib works for people newly diagnosed with a type of leukemia called chronic myeloid leukemia (CML). About 200 adults in China will take the drug and be followed for at least 12 months. The main goal is to see how many ach…

This early-stage study tests a new drug called ELVN-001 in people with chronic myeloid leukemia (CML) whose cancer has not responded to or who cannot tolerate other treatments. The main goals are to check the drug's safety and find the best dose. About 250 adults with CML will ta…

This study tests the drug trabectedin in 16 people aged 16 and older with advanced mesenchymal chondrosarcoma, a rare bone cancer that has spread or cannot be removed by surgery. Participants must have already tried at least one chemotherapy. The goal is to see if trabectedin can…

This early-phase trial is testing a new treatment called KN5601, which uses a patient's own immune cells (NK cells) that have been engineered to target and destroy harmful B cells. The goal is to see if it is safe and can help children aged 5 and older with rheumatic diseases lik…

This early-stage study tests a new drug called HB2198 in 30 adults with moderate to severe lupus, including kidney inflammation. The drug is designed to temporarily remove certain immune cells (B cells) that drive the disease. Participants receive two doses and are monitored for …

This early-phase trial tests whether giving patients extra gamma delta T-cells—a type of immune cell that can kill cancer—after a half-matched bone marrow transplant can improve outcomes. The study includes up to 38 adults with leukemia or myelodysplastic syndromes. The goal is t…

This phase 3 trial compares the experimental drug givinostat to the standard drug hydroxyurea in 220 adults with high-risk polycythemia vera, a blood cancer that increases red blood cells and raises clot risk. The study measures how well each drug controls blood counts and spleen…

This phase 3 trial is testing whether direct oral anticoagulants (DOACs) like apixaban or rivaroxaban are better than low-dose aspirin at preventing blood clots in people with JAK2V617F-positive myeloproliferative neoplasms (blood cancers). The study plans to enroll 1308 high-ris…

This study tests whether new drug combinations can better prevent graft-versus-host disease (GVHD) in people with blood cancers who receive a stem cell transplant from a mismatched unrelated donor. About 358 participants will get either the standard GVHD prevention or one of two …

This early-phase trial is testing a new drug called CBX-250 in people with certain blood cancers (like acute myeloid leukemia) that have come back or not responded to standard treatments. The main goals are to check the drug's safety and find the best dose. About 72 participants …

This early study is testing an experimental therapy called EVM18001 in 12 people with active, hard-to-treat lupus, myasthenia gravis, or scleroderma. The main goal is to see if it is safe and to find the right dose. Researchers will also look at how the body processes the therapy…

This phase 2 trial is testing whether biologic drugs (belimumab or telitacicept) can improve kidney outcomes in people with active lupus nephritis better than standard therapy. About 50 participants will be randomly assigned to one of three treatment groups for six months. The go…

This study tests whether a higher dose of the drug cabergoline, taken for about 6 months, can achieve lasting remission in women with small prolactinomas (non-cancerous pituitary tumors that cause excess prolactin). 70 premenopausal women will be randomly assigned to either the h…

This early-stage trial tests a new treatment called AFN50 for people with autoimmune diseases that haven't responded to standard therapies. AFN50 is a type of CAR T-cell therapy designed to target and destroy faulty immune cells. The study will enroll 18 adults to check if the tr…

This study tests a new drug called CMS-D002 for women with uterine fibroids who have heavy menstrual bleeding. About 120 women aged 18-50 will take either the drug or a placebo daily for 12 weeks. The goal is to see if the drug can reduce bleeding to normal levels and shrink fibr…

This study tests a new treatment using a patient's own immune cells, modified to target and attack faulty immune cells that cause autoimmune diseases. It is for people with severe, hard-to-treat conditions like lupus. The goal is to see if this therapy is safe and can control the…

This study tests a new biologic drug called GR1803 in 44 people with systemic lupus erythematosus, an autoimmune disease. The main goal is to check safety and see if the drug can control lupus activity. Participants must have moderate to severe lupus and have tried standard treat…

This early-stage study tests a new injection called AFN50 in 18 adults with autoimmune diseases like lupus. The main goal is to check safety and see how the body reacts, including changes in immune cells. Participants must have active disease despite standard treatments.

This early-phase trial is testing a personalized cell therapy called CAR-T, designed to target and destroy faulty immune cells in people with active autoimmune diseases like lupus, scleroderma, and Sjogren's syndrome. Up to 20 participants will receive the treatment after a short…

This expanded access program provides DNG64-CAR-V, a gene therapy, to patients with advanced pancreatic cancer, sarcoma, or breast cancer who have exhausted standard treatments. The therapy is given intravenously or directly into tumors, alone or with other cancer drugs. Early st…

This early-stage study tests a new treatment called QT-019B for people with refractory systemic lupus erythematosus (SLE), a severe form of lupus that hasn't responded to standard therapies. The treatment uses specially engineered immune cells (CAR-T cells) from a healthy donor t…

This early-stage trial is testing a new treatment called YTS109 for people with severe autoimmune diseases like lupus and Sjogren's syndrome that have come back or not responded to standard care. About 18 adults will receive a single infusion of these specially engineered immune …

This early-stage study tests a new cell treatment called YTS109 in 18 adults with hard-to-treat autoimmune diseases like lupus and scleroderma. Participants receive a single infusion of these specially designed cells. The main goal is to check if the treatment is safe, and also t…

This study tests whether the drug regorafenib can help prevent bone sarcomas from coming back after initial treatment. About 168 people aged 12 and older with various bone sarcomas will be randomly assigned to take regorafenib for up to 12 months or to receive standard monitoring…

This phase 2 trial tests whether the drug regorafenib can help keep high-grade bone sarcomas from growing in patients who still have some disease after standard treatment. About 36 people aged 12 and older will take regorafenib daily for up to 12 months. The main goal is to see h…

This early-phase study tests a new treatment called TI-0032-III for people with severe autoimmune diseases like lupus and scleroderma that haven't improved with standard care. The treatment uses a special injection to create CAR-T cells inside the body, which then target and dest…

This study tests whether a daily supplement containing vitamin D, EGCG (from green tea), D-chiro-inositol, and vitamin B6 can shrink uterine fibroids in women who are eligible for surgery. Sixty women will take either the supplement or a placebo for three months. The main goal is…

This early-stage study is testing a new treatment called PTOC1 cells, which are a type of CAR-T cell therapy, for people with severe systemic lupus erythematosus (SLE) that hasn't improved with standard treatments. The therapy uses a patient's own immune cells, modified in a lab …

This study tests two drugs, ergometrine and carbetocin, injected into the uterus during fibroid removal surgery to see which reduces blood loss more. Forty women with symptomatic fibroids will be randomly assigned to receive one of the drugs. The goal is to find a better way to c…

This Phase 3 trial tests ivosidenib, a daily pill that targets a specific genetic mutation (IDH1), in 136 adults with advanced or metastatic conventional chondrosarcoma that cannot be surgically removed. Participants are randomly assigned to receive either ivosidenib or a placebo…

This study tests a new drug, YK012, in people with moderate to severe systemic lupus erythematosus (SLE). The drug is designed to target certain immune cells. The trial will check for safety, side effects, and early signs that the drug might help control lupus. About 189 adults a…

This early-stage trial tests a new type of CAR-T cell therapy for people with refractory systemic lupus erythematosus (SLE), a severe form of the autoimmune disease. The treatment uses a patient's own immune cells, modified to target two proteins (BCMA and CD70) on harmful cells.…

This phase 2 trial tests a personalized approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Instead of a one-size-fits-all chemotherapy and radiation regimen, doctors adjust the doses based on each patient's risk. The goal is to improve the…

This pilot study is testing whether high-intensity focused ultrasound (HIFU) can safely destroy soft tissue sarcoma and small desmoid tumors without surgery. About 10-16 adults with these tumors will receive a single outpatient treatment using a CE-approved device. The goal is to…

This early-phase study tests a new treatment called RD06-05, which uses a patient's own immune cells modified to target and reset the immune system in autoimmune diseases like lupus, multiple sclerosis, and vasculitis. The goal is to check safety and how well the body tolerates i…

This study compares telitacicept, a newer biologic drug, against the standard treatment cyclophosphamide for lupus patients who also have interstitial lung disease (ILD). About 100 adults with early lupus and ILD will receive either telitacicept or cyclophosphamide, plus usual ca…

This phase II trial is testing a shorter course of proton therapy for people with chordomas or chondrosarcomas at the base of the skull. Instead of many sessions, patients receive radiation in just 5 consecutive days. The goal is to see if this approach is safe, controls tumor gr…

This early-stage study tests an experimental drug called UB-VV410 in 21 adults with active lupus (including lupus nephritis) that hasn't improved with standard treatments. The main goals are to check safety, find the best dose, and see early signs of effectiveness. Participants w…

This early-stage trial tests a new medicine called IMSB301 in people with rare diseases where the immune system attacks the body. The study includes up to 6 participants aged 12 and older. The main goal is to check if the drug is safe and how it behaves in the body.

This early-stage study tests a new treatment called RXIM002 for people with severe autoimmune diseases that haven't responded to standard therapies. The treatment uses a special RNA molecule to create immune cells that target and destroy faulty B cells, which are causing the dise…

This early-stage trial tests a new treatment for people with autoimmune diseases like lupus and multiple sclerosis that haven't responded to standard therapies. The treatment uses a patient's own immune cells, modified in a lab to target and destroy the faulty cells causing the d…

This early-phase study is testing a new type of immunotherapy called universal CAR-T cells in 12 people with moderate to severe lupus that hasn't responded to standard treatments. The cells are designed to target and destroy faulty immune cells driving the disease. The main goals…

This Phase 1 trial tests a new, fast-made CAR-T cell therapy for people with refractory systemic lupus erythematosus (SLE) who haven't improved with standard treatments. The therapy uses a patient's own immune cells, modified to target and attack lupus-causing cells. The study wi…

This study tests a new daily pill called TERN-701 for adults with chronic myeloid leukemia (CML) who have not responded well to other treatments. The trial has two parts: first, finding the safest dose, then testing that dose in more people. About 180 participants will take the p…

This study tests whether the shingles vaccine (Shingrix) is safe and triggers a good immune response in people with lupus, a condition that affects the immune system. About 224 adults with lupus will receive either the vaccine or a placebo, and researchers will monitor for lupus …

This study follows about 40 adults in Saudi Arabia with chronic-phase chronic myeloid leukemia (CML) who have already tried two other targeted therapies. Researchers will track how well asciminib controls the cancer, its side effects, and how consistently patients take the medica…

This study tests low-dose methotrexate, a common arthritis drug, in 54 people with three rare blood cancers: polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers want to see if it can shrink spleens, improve symptoms, and lower blood counts safely. If succ…

This early trial tests if blinatumomab, a drug used for certain blood cancers, can help children with severe lupus that hasn't improved with other treatments. Six children will receive two short courses of the drug and be monitored for a year. The main goals are to check safety a…

This Phase 3 trial tests whether a drug called obinutuzumab, combined with mycophenolate mofetil (MMF), can control lupus nephritis without the need for oral steroids. About 196 people with active lupus kidney disease will be randomly assigned to either the obinutuzumab/MMF regim…

This phase 2 trial tests whether adding two drugs, ruxolitinib and abatacept, to standard transplant care can prevent graft-versus-host disease (GVHD) in 60 patients with high-risk myeloid cancers like AML and MDS. Participants receive a stem cell transplant from a partially matc…

This study tests whether freezing desmoid tumors with a needle (cryoablation) works better than standard chemotherapy for people whose tumors have grown after a period of watchful waiting. About 150 participants will be randomly assigned to one of the two treatments. If the tumor…

This study is testing whether custom-made or physician-modified stent grafts can safely repair complex aortic aneurysms in people who cannot use standard devices or undergo open surgery. The grafts are tailored to each patient's anatomy, with small holes to keep blood flowing to …

This study follows 300 people with active systemic lupus erythematosus (SLE) who are taking anifrolumab, a drug that blocks a key immune signal. Researchers will track how many achieve remission or low disease activity over 5 years, as well as side effects and quality of life. Th…

This trial tests a one-time treatment using a patient's own immune cells, engineered to target and destroy B cells, which drive autoimmune diseases like lupus, scleroderma, and myositis. 24 adults with active disease will receive the therapy after a short chemotherapy. The main g…

This study tests whether adding navtemadlin to the standard drug ruxolitinib helps people with myelofibrosis who aren't getting enough benefit from ruxolitinib alone. About 600 adults with myelofibrosis will first take ruxolitinib, then those with a poor response will be randomly…

This study tests whether taking misoprostol before myomectomy (surgery to remove uterine fibroids) can reduce blood loss during the operation. 80 women aged 18-43 with symptomatic fibroids will receive either misoprostol or a placebo before surgery. The goal is to see if this sim…

This study looks at how different doses of the drug cabergoline affect blood sugar control in people with prolactinoma, a type of pituitary tumor. Researchers will randomly assign 60 adults to different treatment strategies and measure their glucose levels after a sugar drink. Th…

This study looks at whether blocking the blood supply to desmoid tumors (using tiny particles injected into the arteries) can shrink the tumor and reduce pain without using chemotherapy. It will follow 20 people aged 12 and older who have painful, growing desmoid tumors that cann…

Researchers at Semmelweis University are building and testing artificial intelligence algorithms that read standardized transvaginal ultrasound images. The study includes women aged 18 to 45 who have suspected endometriosis or adenomyosis, a confirmed uterine mass scheduled for s…

People with lupus can develop kidney inflammation called lupus nephritis, which is easier to treat when caught early. This study enrolls adults with lupus who are at higher risk for kidney problems and assigns them by chance to either standard care alone or standard care plus wee…

This study aims to improve how doctors diagnose Cushing's syndrome, a condition caused by too much cortisol. Researchers will test the accuracy of the desmopressin stimulation test in 140 people aged 18-70 who have or may have Cushing's, plus healthy volunteers. Participants will…

This study tests whether a rapid triage test (suPAR) can help doctors make better decisions about admitting or discharging children with fever. Over 5,000 children aged 2 months to 5 years in sub-Saharan Africa will be randomly assigned to standard care or standard care plus the …

This study tests whether a special PET scan (FET PET/CT) can better locate small pituitary tumors that cause Cushing disease. About 50 adults with confirmed Cushing disease who are scheduled for pituitary surgery will receive the scan before their operation. The goal is to see if…

This study tests whether a new type of PET scan using a radioactive tracer called 18F-FCH can more accurately locate overactive parathyroid glands or tumors before surgery. About 193 adults scheduled for parathyroid removal will receive both the new scans and a standard 4D-CT sca…

This study looks at whether two simple blood markers—albumin-to-globulin ratio and prognostic nutritional index—can help predict disease activity in people with lupus. Researchers will measure these markers in 200 participants and compare them with standard disease activity score…

This study is looking for substances in the blood that can help doctors diagnose osteonecrosis of the femoral head (a condition where the hip bone dies) much earlier. Currently, many cases are missed or misdiagnosed. Researchers will collect blood from 300 people with suspected h…

This study will screen 13,000 children in China using urine tests and ultrasound to find kidney disease early. The goal is to see how common kidney problems are in kids and to build a system to predict who is at risk. Children with abnormal results will be referred for further ca…

This study is testing whether a special PET scan using [18F]FET can better locate small pituitary tumors that cause Cushing's disease. Standard MRI misses these tumors in up to 30% of cases. Researchers will compare PET results with MRI and surgical findings in 20 adults to see i…

This early study tests a special PET/MRI scan using a radioactive tracer called FET to find small pituitary tumors that don't show up on regular MRI. The goal is to improve detection of these hormone-producing tumors in 5 adults with confirmed biochemical disease. If the scan wor…

This study tests whether a specific ultrasound measurement can reliably detect superficial endometriosis on the uterosacral ligaments. Researchers will compare ultrasound findings with laparoscopic surgery results in 64 women undergoing gynecological surgery. If a clear cutoff th…

This study aims to develop a non-invasive method to detect and track fibrosis (scarring) in myelofibrosis, a serious blood disorder. Currently, doctors must perform a bone marrow biopsy to check fibrosis. The researchers will test whether a blood test and a special PET scan can i…

This study tests a new ultrasound method to help surgeons find pituitary tumors during surgery. Researchers will compare ultrasound images with MRI scans to see if the new technique can better identify tumors in people with Cushing's disease and other pituitary tumors. About 110 …

This study is testing a new blood test (the HMEC test) to see if it can better identify which patients with thrombotic microangiopathy (TMA) have a specific immune system problem driving their disease. TMA is a severe condition that damages kidneys and the brain. The researchers …

This study is testing a new blood-based test that aims to predict which medication will work best for each person with rheumatic diseases like rheumatoid arthritis or lupus. Researchers will take a blood sample and analyze immune cells to create a personalized treatment profile. …

This trial tests whether placing a special gel inside the uterus after fibroid removal surgery can prevent the formation of scar tissue (adhesions) that may harm fertility. Women aged 18 to 45 who are scheduled for laparoscopic or abdominal myomectomy and have no pre-existing ute…

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

This study tests a laser treatment called OSC/SS to prevent retinal detachment in people with Stickler Syndrome, a genetic condition that raises the risk of vision loss. About 500 children and adults will receive the procedure in one or both eyes and be followed for 5 years. Rese…

This study tests whether taking green tea extract (EGCG) and vitamin D3 daily can prevent uterine fibroids from growing back after surgical removal. About 240 women who have had fibroid surgery will receive either the supplements or a placebo. The goal is to see if this simple co…

This study tests whether using a carotid ultrasound (a neck scan) to find hidden plaque in arteries can help prevent heart attacks and strokes in people with inflammatory rheumatic diseases like rheumatoid arthritis or lupus. About 1,944 participants aged 50 and older will be spl…

This phase 2 trial tests a vaccine called CMV-MVA Triplex, given to stem cell donors before they donate. The goal is to boost the donor's immunity against cytomegalovirus (CMV), which can then be passed to the transplant patient. Researchers hope this will prevent CMV infection i…

Researchers are testing whether a handheld device that stimulates the vagus nerve through the skin can reduce musculoskeletal pain and fatigue in people with systemic lupus erythematosus. Eighteen participants will use the device for five minutes daily for 28 days, with some rece…

This study tests a program called EASE that combines psychological support with regular symptom checks and early access to palliative care. Researchers want to see if it reduces traumatic stress and physical symptoms in people newly diagnosed with acute leukemia. Half of the 266 …

Researchers are testing whether a quadratus lumborum nerve block reduces pain after laparoscopic myomectomy, a surgery to remove uterine fibroids while keeping the uterus. The block is a temporary anesthetic injection into a lower back muscle. In this double-blind, randomized tri…

This trial tests whether two simple distractions — watching a betta fish or playing with a hand puppet — can lower pain, fear, and anxiety in children aged 5 to 7 during a blood draw. One group will use the fish, another the puppet, and a third will receive standard care. Researc…

This study tests whether Pilates, a gentle mind-body exercise, can help manage pain and other symptoms in people with hypermobile Ehlers-Danlos syndrome or hypermobility spectrum disorder. One hundred adults with persistent pain will be randomly assigned to either Pilates classes…

This trial tests whether a three-day low-residue diet and deeper muscle relaxation, alone or together, can improve the surgical view during vNOTES hysterectomy (a scarless procedure through the vagina). The study involves 160 women having hysterectomy for non-cancer conditions. R…

This study tests whether the drug letrozole can improve symptoms and shrink fibroids in women with uterine fibroids. 140 participants will receive either letrozole or a placebo daily for 12 weeks. The main goal is to see if letrozole reduces symptom severity and improves quality …

This study tests whether giving misoprostol (a drug often used for stomach ulcers and labor) before hysteroscopic fibroid removal can reduce the amount of fluid absorbed by the body during surgery. Too much fluid can be dangerous. Forty women with small fibroids (1-3 cm) will rec…

This study tests whether injecting the local anesthetic bupivacaine into the small surgical cuts and the vaginal cuff at the end of a laparoscopic hysterectomy can lower postoperative pain. 120 women having the surgery for non-cancer reasons will be randomly assigned to receive t…

This study compares two online treatments for chronic pain: an 8-week cognitive behavioral therapy program (16 hours total) and a single 2-hour pain relief skills class. Researchers want to see which works better for reducing pain intensity and interference. The study will enroll…

This study tests whether playing Kho-Kho, a traditional Indian tag game, can improve agility and reaction time in school children aged 8 to 12. Over 6 weeks, 48 kids will either play Kho-Kho or do free play three times a week. Researchers will measure changes in agility and react…

This study tests a short behavioral treatment delivered over Zoom to help adults with fibromyalgia, lupus, chronic pelvic pain, or chronic low back pain. The goal is to reduce pain, disability, and related issues like anxiety or depression. Participants will learn strategies to m…

This study tests whether online group programs to manage fatigue can help people with lupus. Participants will either receive a fatigue booklet alone or also attend online group sessions (4 or 7 weeks long). The goal is to see if the programs reduce fatigue impact and improve qua…

This study is testing a new telehealth program designed to support brain health in teenagers with childhood-onset lupus. The program teaches problem-solving, emotion regulation, and communication skills over 10 online sessions. Researchers want to see if the program is practical …

This Phase 3 trial tests whether the experimental drug KLH-2109 can reduce heavy menstrual bleeding in people with uterine fibroids. About 254 participants will take either KLH-2109 or a standard treatment for 24 weeks, tracking their bleeding and visiting the clinic for safety c…

This study tests whether spraying a low-dose lidocaine solution under the diaphragm during single-incision laparoscopic surgery can reduce shoulder pain afterward. About 100 women having surgery for benign gynecological conditions like ovarian cysts or uterine fibroids will take …

This study looks at how three different levels of physical activity—45, 90, or 150 minutes per week—affect arthritis symptoms like pain and mobility. Researchers will enroll 285 adults with arthritis, rheumatoid arthritis, gout, fibromyalgia, osteoarthritis, or lupus. Participant…

This phase 2 trial tests whether a curcumin supplement (C3 complex with piperine) can reduce inflammation and improve symptoms in people with certain blood disorders, including CCUS, low-risk MDS, and MPNs. Thirty participants will receive either curcumin or a placebo for 12 mont…

This study compares High-Intensity Focused Ultrasound (HIFU), a non-invasive procedure using sound waves to destroy fibroids, to myomectomy, a surgical removal. About 112 women aged 18-50 with symptomatic fibroids will be randomly assigned to one treatment. Researchers will track…

This study tests whether adding a gentle electrical acupoint stimulation (TEAS) to a standard three-drug anti-nausea regimen can further reduce vomiting after laparoscopic surgery. Researchers will compare active TEAS to a sham (fake) version in 780 women at high risk for postope…

This study tests N-Acetylcysteine (N-AC), an antioxidant, in 27 adults with myeloproliferative neoplasms (MPN) like essential thrombocythemia, polycythemia vera, or myelofibrosis. The goal is to find the best dose that safely reduces symptom burden, measured by a symptom score. P…

This study looks at whether an advanced practice nurse (APN) can help people with chronic myeloid leukemia (CML) by catching mild side effects early. About 60 adults starting or changing oral therapy will be followed. The goal is to see if nurse-led care improves quality of life,…

This study tests whether a 6-month adapted physical activity program can improve endurance in children and teenagers with Marfan syndrome. About 30 participants aged 7 to 17 will take part. The program focuses on safe, tailored exercises to help them be more active without puttin…

This study looks at whether the Alexander technique, a method that teaches better posture and movement habits, can help children aged 5 to 9 with Duchenne muscular dystrophy control their upper limbs and feel better day-to-day. About 33 kids will take part, and researchers will m…

This study tests whether sensory exercises can improve repetitive behaviors, communication, social interaction, and motor skills in children with autism. About 26 children aged 5 to 11 will take part. Researchers will measure changes using standard questionnaires before and after…

This study tests whether a daily pill called relugolix can safely reduce heavy menstrual bleeding in women with uterine fibroids. About 120 premenopausal women will receive either relugolix or a placebo for several weeks. The main goal is to see if the drug lowers blood loss to n…

This study tests whether playing with a therapeutic toy doll before getting a shot can reduce fear, anxiety, and pain in children aged 4-6. Half the kids will practice the injection steps on a doll first, while the other half get the shot as usual. Researchers will measure fear a…

This study tests whether a mindfulness-based emotion regulation training program can help children aged 7-11 who are hospitalized for at least four days feel less afraid and better control their emotions. Half of the 54 participants will receive the training alongside usual care,…

This study is testing whether extracorporeal shockwave therapy can help men with stable Peyronie's disease. The treatment uses sound waves applied to the penis to target scar tissue. Researchers will measure changes in erectile function and quality of life in 50 participants over…

This phase 2 trial is testing whether the drug ruxolitinib (Jakafi) can reduce symptoms like fatigue and itching in people with low-risk essential thrombocythemia (ET) or polycythemia vera (PV). About 60 participants will take the pill daily, and researchers will measure symptom …

This study follows about 200 adults with polycythemia vera (a blood disorder) who do not have an enlarged spleen and are starting treatment with ropeginterferon alfa-2b. Researchers will track symptoms, blood cell counts, and side effects over 36 months in real-world clinics. The…

This study tests if a type of talk therapy called Emotional Awareness and Expression Therapy (EAET) can help reduce widespread pain in people with lupus. Thirty adults with lupus and fibromyalgia or chronic pain will attend 8 weekly online sessions. Researchers will check if the …

This study looks at whether doing adapted physical activity every day can improve sleep quality in children aged 5 to 16 who are being treated for blood cancer. About 30 children will take part, with some exercising daily and others just once over four days. Parents will track sl…

This study is testing a program called TEACH that teaches coping skills to young people (ages 12-22) with childhood-onset lupus. The goal is to see if it helps reduce common symptoms like fatigue, pain, and depression. About 175 participants will take part, and the program is des…

This study aims to uncover genetic changes that cause autoimmune diseases in people native to Peru. Researchers will collect blood, urine, and stool samples from 300 participants, including patients with conditions like lupus and rheumatoid arthritis, their family members, and he…

This study is tracking 500 pregnant women with lupus to see if the drug anifrolumab is linked to birth defects or pregnancy loss. Researchers will compare women who took anifrolumab during early pregnancy to those who did not. The goal is to gather real-world safety data, not to …

This study is for people with certain autoinflammatory diseases (like NOMID, DIRA, CANDLE, SAVI, and others) and their healthy relatives. Researchers want to learn more about what causes these diseases and how they affect the body over time. Participants will have medical tests a…

This study aims to learn more about how lupus (SLE) develops and changes over time by observing patients and their relatives. Up to 2000 people aged 3 and older with known or suspected lupus, along with their family members, will provide medical history, blood and urine samples, …

This study looks at whether certain genes make some people more likely to develop eye damage from the drug Plaquenil, which is used for autoimmune diseases like lupus and rheumatoid arthritis. Researchers will compare people who have had eye damage from Plaquenil with those who h…

This study looks at the causes and effects of food allergies and related conditions like eczema and eosinophilic esophagitis. Researchers will collect blood, skin, and genetic samples from up to 1,800 people over many years. The goal is to find biomarkers and pathways that explai…

This study watches over 3,000 adults with moderate-to-severe lupus to see if the drug anifrolumab increases the risk of serious infections or cancer compared to standard treatments. No new medicine is given—researchers simply analyze health records from people already prescribed …

This study looks at the long-term health of people who were cured of Cushing disease before age 21. Researchers want to understand how past high cortisol levels affect the body and mind years later. Participants will complete surveys and have check-ups every 5 years. The goal is …

This study tracks 16 breastfeeding mothers with lupus who are taking anifrolumab. Researchers will measure how much of the drug appears in breast milk and in the mother's and baby's blood. The goal is to understand potential exposure and effects on the breastfed infant.

This study tracks 240 pregnant women with lupus who have taken anifrolumab (Saphnelo) to see if the drug affects pregnancy or baby health. Researchers will monitor for birth defects and other complications. The goal is to gather safety information, not to test a new treatment.

This study aims to learn more about the genetics and natural history of autoinflammatory diseases, which cause repeated fevers and inflammation. Researchers will study up to 5,000 people, including patients, their relatives, and healthy volunteers. Participants provide blood and …

This study aims to uncover the genetic causes of pituitary and hypothalamic tumors in children. Researchers will collect blood and tumor samples from up to 2,000 participants to analyze DNA and look for inherited patterns. The study also trains doctors and tests new MRI technique…

This study is observing 223 people with active lupus (including kidney inflammation) who haven't gotten better with steroids and at least two immunosuppressants. Researchers want to see how well today's standard treatments work for these tough cases. The goal is to measure how ma…

This study collects blood and tissue samples from 2000 people—some with cancer and some without—to create a blinded reference set. Researchers will use these samples to evaluate how well new blood tests can detect many types of cancer early. The goal is to improve the accuracy of…

This study follows 442 pregnant women with moderate-to-severe lupus to see if taking the drug anifrolumab (Saphnelo) during pregnancy affects the health of mothers or their babies. Researchers will compare women who took the drug during pregnancy to those who did not, tracking bi…

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 looks at how well a non-invasive navigation system helps surgeons place the hip cup during total hip replacement. About 118 people with hip conditions like arthritis or hip dysplasia will take part. The goal is to see if the actual cup position matches the planned posi…

This observational study follows up to 20,000 children and young adults with pediatric-onset rheumatic diseases, such as juvenile idiopathic arthritis. Researchers collect data on treatments, safety, and long-term outcomes to better understand these conditions. The registry does …

This study aims to create a network of biobanks across Italy that collect and store biological samples and health data from people with rare diseases and their families. The goal is to standardize how samples are collected and shared, so researchers can access high-quality materi…

This study is a large registry that collects health information and leftover tumor or blood samples from up to 75,000 children and young adults with cancer. The goal is to track outcomes and gather data to help doctors find better ways to treat and care for these patients over ti…

This study aims to better understand why some people's immune cells attack their own body, causing autoimmune diseases like lupus and scleroderma. Researchers will analyze blood samples from 200 adults with these conditions to identify and measure specific harmful immune cells. T…

This study is a pregnancy registry that gathers information from women with chronic neurological conditions like multiple sclerosis, NMOSD, and myasthenia gravis who are planning pregnancy, currently pregnant, or within one year postpartum. Participants can contribute blood, brea…

This study looks at a type of immune cell called monocytes in people with certain blood cancers (polycythemia vera, essential thrombocythemia, and myelofibrosis). Researchers will analyze blood samples from 70 newly diagnosed patients to find unique patterns in these cells. The g…

This study looks at whether tyrosine kinase inhibitors (TKIs), the standard treatment for chronic myeloid leukemia (CML), might be linked to erectile dysfunction (ED) in men. Researchers will track 350 male patients who started TKI therapy, using questionnaires and medical record…

This study surveys women under 40 with lupus who are starting cyclophosphamide, a drug that can damage ovaries and cause infertility. Some received an additional drug called GnRHa to protect their ovaries. Researchers will compare menstrual health and fertility between those who …

This study investigates why African Americans, particularly the Gullah community from the Sea Islands of South Carolina and Georgia, are at higher risk for systemic lupus erythematosus (SLE). By analyzing genetic and environmental factors in 750 participants—including people with…

This observational study is working to create a more objective way to measure pain in children with juvenile idiopathic arthritis, lupus, or fibromyalgia. Researchers will follow 90 participants for one year, collecting data during regular clinic visits. The goal is to develop a …

This study investigates whether gut bacteria and the chemicals they produce influence inflammation in people with autoinflammatory diseases such as FMF, CAPS, and MKD. Researchers will analyze stool and blood samples from 300 patients to measure gut-derived metabolites and assess…

This registry will follow 1,000 adults with chronic-phase CML who are taking approved TKIs (like Gleevec or Tasigna) as their first or second treatment. Over 5 years, researchers will track side effects, treatment changes, and patient-reported experiences to understand how these …

This study is a national registry in China that will track 500 women who experience an aortic dissection (a tear in the main artery) during pregnancy or within 12 weeks after giving birth. Researchers will collect information on treatments and outcomes for both mothers and babies…

This study examines why some patients with essential thrombocythemia (ET) later develop polycythemia vera (PV), a related blood condition. Researchers will review medical records of 678 adults to identify risk factors for this change and compare outcomes between those who progres…

This study aims to better understand VEXAS syndrome and other autoinflammatory conditions by analyzing participants' DNA from blood, saliva, or skin samples. Researchers hope to discover new genetic variants that cause or contribute to these diseases. The study enrolls people of …

This study is creating a large collection of medical data and blood samples from 450 adults with autoimmune diseases (rheumatoid arthritis, lupus, or scleroderma) and 50 healthy volunteers. Researchers will use advanced lab techniques to find unique biological markers that could …

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This study follows 500 adults with lupus to see if blood and urine tests can reliably detect kidney inflammation (lupus nephritis). Participants give samples during routine visits, and doctors track their kidney health over time. The goal is to find simple, non-invasive markers t…

This study looks at gum disease in people with rare kidney disorders like Alport syndrome, Fabry disease, and tuberous sclerosis, as well as lupus. Researchers will compare 100 participants to those with chronic kidney disease and healthy controls. They aim to understand how comm…

This study follows 300 adults with systemic lupus erythematosus (SLE) who are seen by a team of specialists at a hospital in Brest, France. Researchers will collect data on disease patterns, symptoms, and test results over time. The goal is to better understand lupus and improve …

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 aims to find the genetic causes of Ollier disease and Maffucci syndrome, rare disorders that cause cartilage tumors and increase cancer risk. Researchers will study 100 people aged 2 and older using blood tests, scans, and genetic sequencing. Participants will stay at …

This study is collecting skin and blood samples from 830 people with chronic inflammatory skin diseases like psoriasis, eczema, and lupus. Researchers want to find biological markers that can predict how well a person will respond to treatment after one year. The goal is to move …

This study looks at whether targeted cancer drugs called TKIs affect brain health and well-being in people with chronic leukemia. Researchers will compare 400 patients who receive TKI therapy to those who do not. The goal is to understand if these treatments cause cognitive chang…

This study looks at how well the shingles vaccine (Shingrix) works in people with lupus compared to healthy people of the same age and sex. Researchers will measure immune cell responses after two doses and follow participants for up to 5 years. The goal is to understand if the v…

This study aims to learn how well heart scans (MRI, ultrasound, PET) can detect and predict outcomes in people with suspected or known inflammatory heart diseases. Researchers will follow 5,000 adults for one year to see how often heart involvement is confirmed and how it affects…

This study looks at how the body processes venetoclax when the tablet is crushed and mixed into a liquid. It includes 30 children and young adults with blood cancers who already take venetoclax this way. The goal is to measure drug levels in the blood and spinal fluid to ensure t…

This study is testing whether a specialized blood test (MAIPA) can accurately identify autoimmune thrombocytopenia (ITP) in patients with low platelet counts. Currently, ITP is diagnosed by ruling out other causes, which can be slow and uncertain. Researchers will collect blood s…

This study is testing a new technique called optical genome mapping (OGM) to find genetic changes in blood cancers like polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers will compare OGM with standard genetic tests in 300 patients who need a bone marrow…

This study looks at people with multidirectional shoulder instability (loose shoulder joints) often linked to hypermobility disorders. Researchers want to see if electrical stimulation and specific exercises can improve shoulder control and reduce joint slipping. 60 participants …

This study looks at 600 teens and young adults (ages 14-26) with conditions like juvenile arthritis, fibromyalgia, or past surgeries to understand why some develop long-lasting pain while others don't. Researchers will examine pain sensitivity, immune markers, and social factors …

This study aims to find new genes that cause pituitary tumors, including those that lead to acromegaly (gigantism) and prolactinomas. Researchers will collect blood and tissue samples from up to 10,000 people with these tumors and their family members. By analyzing DNA, they hope…

This study looks at how a type of cell death called ferroptosis, which is driven by iron, might play a role in lupus. Researchers will take blood samples from 120 people with lupus and compare them to people with other autoimmune diseases. The goal is to find new clues for future…

This study reviews the medical records of 300 people with multiple osteochondromas (a rare bone condition causing multiple bony growths) who had surgery at a specialized hospital in Italy. Researchers want to understand how many surgeries patients typically need, what types of su…

This study follows 100 people with desmoid-type fibromatosis, a rare tumor, for 12 months without giving any treatment. The goal is to see how often the tumors stay stable or shrink on their own. Participants get regular MRI or CT scans to track changes. The results could help do…

This study looks at how the immune system reacts to myeloproliferative neoplasms (MPN), a group of blood cancers. Researchers will collect blood and bone marrow samples from 150 adults with or without MPN to find special immune cells (T cells) that recognize and attack MPN cells.…

This study looks at whether a detailed MRI analysis called radiomics can predict if a desmoid tumor will grow back after cryoablation (freezing treatment) better than standard methods. Researchers will follow 21 patients treated at one hospital to compare radiomics with usual siz…

This study is building a registry of 1,211 people in Brazil who have inherited heart conditions like cardiomyopathy, arrhythmias, and Marfan syndrome. Researchers will collect DNA samples and medical information to discover which genes are most commonly affected and how often the…

This study is collecting health information, lifestyle data, and biological samples from up to 100,000 adults to help researchers better understand diseases like lupus, celiac disease, and kidney disease. Participants provide data and samples through a direct-to-patient platform.…

This study is building a large database and biobank from 2200 people with chronic inflammatory diseases like lupus, multiple sclerosis, severe asthma, and psoriasis. Researchers will collect blood samples and track disease activity over time using standard medical scores. The goa…

This study creates a nationwide registry and biobank for children diagnosed with myeloproliferative neoplasms (MPNs)—rare blood cancers that cause overproduction of blood cells. By collecting medical data and blood samples from up to 500 children across Germany, researchers hope …

This study looks at specific immune cells (iNKT cells and monocytes) in people with lupus to see how certain receptors on these cells change when the disease is active versus stable. Researchers will collect extra blood samples from 50 lupus patients during routine visits. The go…

This study looks at 60 children with lupus to find early signs of heart problems using non-invasive ultrasound. The goal is to identify which children are at higher risk for heart issues. No new treatments are being tested.

This observational study is following 700 pregnant women with lupus or antiphospholipid syndrome across nine medical centers. Researchers are looking at specific proteins in the blood to see if they can predict serious pregnancy complications like fetal death, preterm delivery, o…

This study looks at how certain natural chemicals in the body affect blood vessel health in people with pseudoxanthoma elasticum (PXE), a rare genetic disease that causes calcium buildup in tissues. Researchers will measure levels of these chemicals in 45 adults with PXE to bette…

This study follows 300 people who had surgery to remove bone tumors (like osteosarcoma or chondrosarcoma). Researchers want to see how well the bone heals and how much function returns over 3 years. Participants fill out questionnaires about their daily activities. No new drug or…

This study aims to find the genes and DNA changes that cause cherubism, a rare condition that affects the jaw bones. Researchers will study blood and tissue samples from up to 600 people, including patients and their family members. The long-term goal is to understand the disease…

This study is building a large registry and biorepository of 3,500 adults with systemic lupus erythematosus (SLE), including those with lupus nephritis and neuropsychiatric lupus. Participants will provide clinical data and biospecimens over time, creating a resource for future r…

This study aims to understand exactly how the drug interferon attacks cancer stem cells in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will analyze blood samples from 80 adults to see why some patients respond well while others do not. T…

This study is building a database of 700 people with cutaneous lupus erythematosus (CLE) to track how often it occurs and how severe it gets. Researchers at the University of Pennsylvania and UT Southwestern are collecting information over time. No new treatments are being tested…

This study looks at the immune system of 200 people with desmoid tumors, either under observation or receiving treatment. Researchers will analyze blood and tumor samples to find markers that may predict whether the tumor shrinks, stays stable, or grows. The goal is to better und…

This study looks at how multiple osteochondromas (non-cancerous bone growths) affect movement and body shape in children and adults. Researchers will use wearable sensors, pressure mats, and 3D body scans to measure walking, balance, and joint motion. The goal is to find simple w…

This study looks at whether a new way of scoring whole-body MRI scans can reliably measure bone inflammation in children with CRMO, a rare condition causing repeated bone pain and swelling. Researchers will compare the MRI scores from different doctors and also check if changes i…

This study aims to create a collection of blood and urine samples from 300 people with Marfan syndrome or related conditions. Researchers will use these samples to study how the disease works and find clues that might predict how it will progress. Participants provide extra sampl…

This study follows 2500 people who have had hip replacement surgery with Corin implants. Researchers will check how long the implants last and how well they work over 10, 15, and 20 years. The goal is to gather safety and performance data, not to test a new treatment.

This study aims to collect blood samples and medical data from 3000 people with autoimmune diseases like lupus, scleroderma, and myositis. Researchers will use these samples to find new biomarkers and better understand these conditions. The goal is to help develop new treatments …

This study is gathering information from 15 people with CACP syndrome, a rare genetic condition that causes joint problems and sometimes heart issues. Researchers want to better understand how the disease develops and changes over time. The goal is to improve diagnosis and care f…

This study looks back at the past 10 years to understand how a life-support machine called ECMO has been used in children across China. Researchers will gather information from 500 children, from newborns to 18-year-olds, to see how many survive after treatment, how long they nee…

This study follows 400 pregnant women with rare blood vessel diseases (like HHT, Marfan syndrome, or lymphedema) to learn about serious pregnancy complications. Researchers will ask participants about their health during pregnancy and for 12 months after birth. The goal is to gat…

This study explores whether a person's personality traits influence how well they take their daily leukemia pills. Researchers will interview 75 adults with chronic myeloid leukemia and use questionnaires to measure medication adherence, personality, and quality of life. The goal…

This study is testing whether giving the COVID-19 booster and flu vaccine at the same time is as good as giving them a month apart for people with weakened immune systems. It also compares getting a COVID-19 booster every 3 months versus every 6 months. The trial involves 660 adu…

This study is for adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis who are starting their first treatment. Researchers want to find out why some patients respond well to standard drugs while others do not. They will look at genetic and immune system …

This study looks at whether using a uterine manipulator during hysterectomy changes sexual function three months after surgery. Sixty women having a hysterectomy for non-cancer reasons will be randomly assigned to have the surgery with or without the tool. The main goal is to com…

This study looks at whether people with CAPS (a rare genetic inflammatory condition) have disrupted circadian rhythms—the body's natural day-night cycle. Researchers will measure melatonin levels and track activity with a watch in 30 participants (patients and healthy household m…

This study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

This study is building a large registry and biorepository of 5,000 adults with various rheumatic diseases like rheumatoid arthritis, lupus, and scleroderma. Researchers at Yale University will collect medical data and biological samples over time to better understand these condit…

This study looks at how changes in the TREX1 gene are linked to lupus, especially skin problems. Researchers will check disease activity in 90 adults with lupus using a standard scoring tool. No new treatment is given; the goal is to better understand the disease.

This study looks at whether using 3D printed models of a patient's bones helps surgeons plan and perform tumor removal more effectively. About 150 people with bone tumors will take part, and half will have their surgery planned with both 3D models and regular scans, while the oth…

This study looks at how well bone cancer patients follow a personalized home exercise program in the first six months after lower leg surgery. Researchers will track daily exercise logs from 50 patients and explore what factors help or hinder adherence. The goal is to learn how t…

This study reviews past medical records of 12 people with Familial Mediterranean Fever to see how well different drugs control symptoms in everyday practice. Researchers will measure symptom improvement using patient ratings. The goal is to understand which treatments work best o…

This long-term observational study is enrolling 5,000 adults with myeloproliferative neoplasms (MPNs)—rare blood cancers like polycythemia vera and myelofibrosis. Researchers will collect health records, patient surveys, and insurance data over at least five years to track sympto…

This study is a fully online registry for adults and children with lupus. Participants fill out surveys every six months about their symptoms, treatments, and quality of life. The goal is to collect real-world data to improve care and guide future research. No travel or clinic vi…

This study investigates whether higher lifetime exposure to estrogen—from sources like birth control, pregnancy, or hormone therapy—is linked to the development of larger prolactin-secreting pituitary tumors (macroprolactinomas) compared to smaller ones (microprolactinomas). Rese…

This study is observing children with childhood-onset lupus who are receiving an experimental therapy called a bispecific T-cell engager. Researchers will take blood samples over time to track how the immune system changes. The goal is to find patterns that could help monitor the…

This study looks at why some lupus patients get false positive results on tests for HLA antibodies, which can make it harder to get an organ transplant. Researchers will study 30 adults with lupus who have no known risk factors for these antibodies. The goal is to find better way…

This study is testing whether artificial intelligence can help doctors better classify and predict the behavior of rare smooth muscle tumors of the uterus, including those that are hard to diagnose (called STUMP). Researchers will train AI models on digital images of tumor tissue…

This study is looking for better ways to predict blood clots in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will measure specific markers in the blood of 300 participants to see if they can tell who is at higher risk for clots. The goal …

This registry enrolls pregnant women or those planning pregnancy who have autoimmune diseases like lupus or rheumatoid arthritis. Researchers at Duke University will track health data to find ways to improve outcomes for mothers and babies. The goal is to better understand risk f…

This study is creating a registry of 1,000 people with lupus (systemic lupus erythematosus or cutaneous lupus) to collect health information and blood samples over time. The goal is to learn how lupus changes and find better ways to treat it. Participants must be patients at Duke…

This study is for people under 50 with a type of bone cancer called Ewing sarcoma. Researchers will collect information about patients' health, treatments, and results over time. The goal is to learn more about the disease and how it is treated, not to test a new drug or therapy.

This study looks at whether women with lipedema have flatter feet and looser joints compared to women without the condition. Researchers will use foot X-rays and flexibility tests to measure these differences. The goal is to better understand how lipedema affects foot function an…

This study compares a continuous heart-monitoring patch with standard electrocardiograms (ECGs) in 60 adults receiving cancer drugs that can cause dangerous heart rhythm changes. The goal is to see if the patch detects problems more accurately and consistently. Participants will …

This study looks at pregnancy outcomes in 50 women who had at least one pregnancy before being diagnosed with lupus. Researchers will collect data from medical records and interviews to see how often pregnancy complications occurred before the lupus diagnosis. The goal is to bett…

This study looks at how natural cannabis compounds (phytocannabinoids) affect immune cells from 100 people with chronic inflammatory diseases like rheumatoid arthritis, lupus, and Crohn's disease. Researchers will take a blood sample and expose the cells to different cannabis ext…

This early-phase trial is testing two new antibody fragments that are designed to attach to a sugar molecule found on many solid tumors. The fragments are labeled with a radioactive tracer so they can be seen on PET/CT scans. The study aims to see where these fragments go in the …

This study looks at people with a mechanical heart valve who hear a clicking noise from it. Researchers want to know if remembering information given before surgery about this noise affects their quality of life afterward. About 180 participants will answer questionnaires over th…

This study is not a treatment trial. It aims to collect blood samples from 400 children and their relatives who have rare autoimmune or autoinflammatory diseases (like lupus or juvenile arthritis). Researchers will analyze the samples to find genetic mutations that cause these co…

This study aims to learn more about rare genetic diseases that affect both the immune system and the nervous system. Researchers will collect medical information and samples from 1,000 participants, including patients, their relatives, and healthy volunteers. The goal is to bette…

This study at Johns Hopkins University is looking at how aging cells (senescent cells) contribute to uterine fibroids, which are non-cancerous growths in the uterus. Researchers will analyze tissue samples from 150 women with fibroids to map these cells and their signals. The goa…

This study creates a registry of 5,000 adults aged 50 and older with blood cancers like multiple myeloma and lymphoma. Researchers will track frailty, muscle loss, and other age-related health issues to better understand this group's needs. The goal is to gather information that …

This study looks at whether a special eye scan can detect early blood flow changes in the retina of people with pseudoxanthoma elasticum (PXE), a rare genetic disease that can cause vision loss. Researchers will compare 30 PXE patients with 30 healthy volunteers matched by age an…

This study is testing whether a special ultrasound technique called VExUS can help doctors manage fluids better during chest surgery. The goal is to see if it lowers the chance of lung and kidney problems after surgery. About 230 adults having video-assisted lung surgery will tak…

This study aims to find a way to predict which cancer patients will experience side effects from immunotherapy. These side effects happen when the immune system attacks healthy tissues, similar to autoimmune diseases. Researchers will collect blood samples from 300 cancer patient…

This study is building a large registry of people with multiple osteochondromas, a condition that causes benign bone tumors. Researchers will collect medical history, genetic data, and treatment information from up to 10,000 participants. The goal is to better understand the dise…

This study looks at how immune cells and inflammatory molecules differ in people with migraine, especially those who also have autoimmune diseases like multiple sclerosis, rheumatoid arthritis, or endometriosis. Researchers will compare blood samples from 396 women to measure reg…

This study looks at how thick the blood is in 160 people with polycythemia (a condition with too many red blood cells). Researchers want to see if thicker blood causes more symptoms like headaches or dizziness, and how it differs between types of polycythemia. No new treatments a…

This study is building a registry of up to 400 people with Ollier disease or Maffucci syndrome. Researchers will collect medical history, genetic data, imaging, and quality-of-life information to better understand how these conditions progress and how they are best managed. The g…

This study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…

This study looks at whether lupus makes the immune system age faster. Researchers will take blood samples from 75 lupus patients to measure changes in immune cells and telomeres (a marker of aging). The goal is to better understand how lupus affects the body over time.

This study looks at whether doing a repeat kidney biopsy one year after starting treatment for lupus nephritis can help doctors decide if more treatment is needed. Half of the 206 participants will get the repeat biopsy, and the other half will follow standard care. The goal is t…