Primary lymphedema

This study tests alpelisib, a drug that targets a specific gene mutation (PIK3CA), in people with lymphatic malformations—abnormal growths of lymph vessels. The trial includes children and adults and compares alpelisib to a placebo to see if it can shrink the growths and ease sym…

This phase 3 trial compares a once-weekly growth hormone injection (lonapegsomatropin) to a daily one (somatropin) in 186 prepubertal children with growth failure due to Turner syndrome, SHOX deficiency, being small for gestational age, or idiopathic short stature. The goal is to…

This Phase 2 study tests an oral drug called RLY-2608 in adults and children with overgrowth conditions (like CLOVES or Klippel-Trenaunay syndrome) caused by PIK3CA gene mutations. The drug is designed to block the faulty protein driving abnormal tissue growth. The trial will enr…

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

This study tests whether the drug rapamycin can shrink large, hard-to-treat lymphatic malformations in the head and neck area of children. Over 6 months, 28 kids will take oral rapamycin, and doctors will measure changes using MRI scans and quality-of-life surveys. The goal is to…

This study tests an injected medicine called TARA-002 for children aged 6 months to 18 years who have lymphatic malformations (fluid-filled cysts). The goal is to see if the injections can safely shrink or eliminate the cysts. Up to 38 children will receive up to 4 injections, ea…

This study tests vosoritide, a protein that stimulates bone growth, in 30 children with Noonan syndrome who are not growing well despite growth hormone treatment. The goal is to see if vosoritide can safely increase their growth rate over six months. Participants will receive one…

This phase 2 trial is testing two targeted drugs—alpelisib and mirdametinib—in 50 people with slow-flow or fast-flow vascular malformations that haven't responded to standard treatments. The goal is to see if these drugs can improve each person's most bothersome symptom over 48 w…

This study tests whether taking the drug sirolimus just once a week can safely treat venous and lymphatic malformations—abnormal clusters of blood vessels or lymph vessels that can cause pain and swelling. Current daily dosing works but often causes side effects like low white bl…

This study tests whether a special dye (indocyanine green) that glows under a special camera can help surgeons better see and remove cystic lymphatic malformations in children. About 110 children will be randomly assigned to either standard surgery or surgery guided by this dye. …

This study tests a systematic approach for treating orbital lymphatic malformation in children aged 1-18. Participants are split into three groups: one gets sclerotherapy (injections to shrink the lesion), one gets the drug sirolimus, and one gets both. The goal is to see if siro…

This study tests a new type of ultrasound called super-resolution ultrasound (SRUS) to see if it can better tell the difference between benign (non-cancerous) and malignant (cancerous) swollen lymph nodes. About 779 adults with abnormal lymph nodes will receive an injection of ul…

This study compares a newer painkiller called oliceridine with two standard drugs, morphine and sufentanil, for pain relief after lumbar spine surgery. Ninety adults will receive one of the three drugs through a patient-controlled pump. The main goal is to see how well each drug …

This study looks at whether doing resistance exercises (like leg presses) can reduce swelling in the lower legs of people with lymphedema. It also checks if wearing compression garments during exercise makes a difference. Thirty-six adults with lower limb lymphedema will exercise…

This study tests whether a supplement called MyCondro can help people with knee osteoarthritis move better and feel less joint discomfort. Researchers will give two different doses to 240 adults aged 45 and older who have had knee problems for at least six months. The main goal i…

This study tests whether adding virtual reality (VR) exercises to standard therapy can improve walking, reduce leg swelling, and boost quality of life in people with lower limb lymphedema. Forty adults with stage 2 or 3 lymphedema after cancer surgery will either receive standard…

This study tests whether adding aerobic exercise to standard lymphedema therapy (complex decongestive physiotherapy) can improve walking, balance, leg strength, and daily function. Twenty-four adults with bilateral lower limb lymphedema will be randomly assigned to either standar…

This study tests whether Acceptance and Commitment Therapy (ACT), delivered through a smartphone app, can help caregivers of children with RASopathies (like Neurofibromatosis type 1 and Noonan syndrome) cope with parenting stress. The trial is fully remote and involves 70 adult c…

This study tests whether adding kinesio tape to standard therapy helps women with long-term breast or trunk swelling after breast cancer treatment. About 128 participants will receive either standard care alone or standard care plus kinesio tape for four weeks. Researchers will m…

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

This natural history study aims to collect data from 1,200 people with lymphatic anomalies—rare conditions affecting the body's lymph system—over many years. Researchers will track symptoms, perform genetic testing, and gather biospecimens to better understand why these condition…

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

This study 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 looks at how to reduce the long and frustrating journey to a diagnosis for people with developmental abnormalities. Researchers will review past cases, collect new blood or skin samples, and use advanced genetic testing. The goal is to understand why some people remain…

This study aims to learn more about RASopathies, a group of genetic conditions that can cause developmental issues, birth defects, and increased cancer risk. Researchers will follow up to 500 people of any age who have or may have a RASopathy, along with their family members, for…

This study is creating a registry of up to 1,000 people with rare lymphatic system disorders. Researchers will collect information from medical records and yearly surveys to understand how these diseases progress, what treatments are used, and how they affect quality of life. The…

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

This study aims to create a collection of blood and urine samples from 100 people with Noonan syndrome. Researchers will use these samples to study how the disease works and look for factors that predict how it will progress. The study does not test any treatment, but may help gu…

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 looks at cholesterol and blood sugar levels in 200 people with Noonan syndrome and related conditions, aged 2 to 35. Researchers want to see if these levels differ by age, gender, or genetic type. The goal is to improve long-term care by identifying who might be at hig…

This study looks at the emergency and disaster plans of about 319 German hospitals to see how well they are prepared for crises. Researchers will compare the plans to find common strengths, weaknesses, and best practices. The goal is to help hospitals improve their emergency plan…

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

This study is testing advanced heart MRI scans to better diagnose and predict risks for people with rare heart muscle diseases. Researchers will scan 1000 participants to see if these new imaging methods can identify conditions like Fabry disease and cardiac amyloidosis more accu…

This study is a large European registry that will observe up to 500 patients with lymphatic disorders related to congenital heart disease. Researchers will collect data on current diagnostic imaging and treatments like lymphatic embolization to see which approaches improve sympto…

This study aims to understand why lymphedema develops by comparing gene activity in swollen versus healthy limbs. Researchers will take tiny tissue samples from 60 adults with primary or secondary lymphedema. The goal is to identify molecular differences that could point toward f…

This study is collecting blood, tissue, and medical information from up to 1,000 people with RASopathies—a group of genetic conditions that affect development and raise cancer risk. Researchers will store these samples and data in a database for future studies. The goal is to lea…

This study looks at 48 people with chronic leg swelling to find out why some respond to compression therapy and others don't. Participants will have an MRI and genetic testing to measure fat, muscle, and fluid in their legs. The goal is to better understand the condition, not to …