Partial lipodystrophy

This study tests a new drug called mibavademab for people with generalized lipodystrophy, a rare condition where the body lacks fat tissue. The goal is to see if the drug can improve blood sugar, lower blood fats, and reduce liver fat. About 28 children and adults will take part …

This registry monitors the safety and effectiveness of metreleptin in up to 100 people with generalized or partial lipodystrophy who are already taking the drug in routine care. Researchers will track serious events like pancreatitis, liver problems, infections, and cancers. The …

This study looks at the long-term safety and effectiveness of a daily injection called metreleptin for people with familial partial lipodystrophy, a rare condition where fat is distributed abnormally. About 24 participants who completed a previous study will receive the drug for …

This study tests a drug called pegvisomant in 25 adults with severe insulin resistance caused by lipodystrophy or insulin receptor gene changes. Participants give themselves daily shots for one month and have two hospital stays for tests. The goal is to see if blocking growth hor…

This program provides expanded access to REGN4461 (mibavademab) for people with rare diseases caused by deficient leptin signaling, such as generalized lipodystrophy and monogenic obesity. The drug aims to help control metabolic issues linked to these conditions. Participants rec…

This expanded access program provides metreleptin (MyaLept) to patients with familial partial lipodystrophy who also have diabetes or very high triglycerides. Metreleptin is already approved for generalized lipodystrophy, but this program aims to help those with the partial form.…

This phase 2 trial tests whether tirzepatide (Mounjaro), a drug already approved for diabetes and weight loss, can improve blood sugar and lower triglycerides in people with partial lipodystrophy—a rare condition where the body loses fat tissue. Researchers will compare tirzepati…

This study aims to find out whether some women diagnosed with polycystic ovary syndrome (PCOS) actually have a rare genetic condition causing severe insulin resistance. Researchers will compare 25 women with a known LMNA gene mutation (linked to lipodystrophy) to 50 women with cl…

This study looks at how fat is stored in people with familial partial lipodystrophy (FPLD), a condition that can lead to diabetes and heart disease. Researchers will compare fat tissue from people with FPLD and healthy volunteers. Participants drink special water and get infusion…

This study looks at whether people with vascular Ehlers-Danlos syndrome (vEDS), a rare genetic disorder that weakens blood vessels, also have problems with how their body stores and uses fat. Researchers will compare 12-17 adults with vEDS to similar adults without the condition …

This study is a European registry for people with lipodystrophy, a rare condition where the body loses or lacks fat tissue. Researchers will follow up to 5,000 patients over time, collecting health data and genetic information. The goal is to better understand the disease, its pr…