Primary lymphedema
MONDO:0019175A congenital condition that results in swelling in the arms or legs, and can occur during adolescence or adulthood. Loss of motion and pain may also accompany the swelling. Protein-rich lymphatic fluid accumulates in tissues, engorging and enlarging vessels and often causing visible swelling, tenderness, and pain. Left untreated, the affected tissues may continue to swell, and can become hardened or fibrotic and susceptible to infection.
Also known as: Troncular lymphatic malformation, primary lymphedema
69 clinical trials for this condition and its sub-types.
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Broader categories
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Weekly shot could replace daily needles for kids with growth disorders
Disease control OngoingThis study tests if a new growth hormone medicine (somapacitan) given once a week works as well as the standard daily growth hormone (Norditropin) for children who are very short due to being born small, or having Turner syndrome, Noonan syndrome, or unknown causes. About 412 chi…
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Jun 27, 2026 13:08 UTC
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New pill may replace chemo for kids with brain tumors
Disease control OngoingThis study tests a new drug called DAY101 (tovorafenib) against standard chemotherapy for children and young adults with a type of brain tumor called low-grade glioma that has a specific gene change (RAF alteration). The goal is to see if the new drug works better at shrinking tu…
Phase: PHASE3 • Sponsor: Day One Biopharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
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New drug combo targets Hard-to-Treat cancers in early trial
Disease control OngoingThis early-phase study tests a new drug called ERAS-601, alone or with other cancer treatments, in adults with advanced solid tumors that have not responded to standard therapies. The main goals are to find safe doses and check for side effects. About 90 participants will take pa…
Phase: PHASE1 • Sponsor: Erasca, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New drug aims to help kids with rare genetic short stature grow taller
Disease control OngoingThis study tests a drug called vosoritide in 56 children with short stature caused by certain genetic conditions. The drug targets the growth plate to help children grow faster. Participants are observed for 6 months, then treated with daily injections for 12 months to check safe…
Phase: PHASE2 • Sponsor: Andrew Dauber • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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Weekly shot may help short kids grow – new study underway
Disease control OngoingThis study tests a new growth hormone medicine called somapacitan, given once a week, in 47 children who are very short due to being born small for gestational age, or having Turner syndrome, Noonan syndrome, or idiopathic short stature. The main goal is to see if it is safe and …
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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New program aims to ease burden on families of kids with rare diseases
Symptom relief ENROLLING_BY_INVITATIONThis study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …
Phase: NA • Sponsor: Children's National Research Institute • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC
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Forgotten side effect: new study tracks leg swelling in men with prostate cancer
Knowledge-focused OngoingThis study follows 214 men treated for prostate cancer to see how many develop lower limb lymphedema (leg swelling) and who is most at risk. Participants attend check-ups every three months for 18 months. The goal is to better understand this side effect and its impact on quality…
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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New registry to monitor growth hormone treatment in kids with rare condition
Knowledge-focused ENROLLING_BY_INVITATIONThis study is a registry that will follow up to 221 children with Noonan Syndrome who are already taking or starting Norditropin® for short stature. Researchers will collect information on growth, side effects, and quality of life over time. No new treatment is being tested—the g…
Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC