Expanded access treatment for leptin deficiency conditions
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This program provides access to an experimental drug (REGN4461) for people with rare conditions related to a problem with the hormone leptin, which helps control appetite and body weight. It is an expanded access program, meaning it's a pathway for patients who have no other treatment options.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed condition linked to problems with how your body uses the hormone leptin.
- Standard treatments are either not available or have not worked for you.
- Your doctor must be willing to work with the study sponsor to get the medication for you.
- This program is for individual patients and requires special approval before you can receive the drug.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study is for people with a rare genetic condition that causes severe obesity and high appetite, called leptin receptor deficiency or related leptin problems. The trial tests a new approach that targets the liver to help with weight control, and it aims to see if this can improve their condition.
This trial tests a new drug called mibavademab for people with generalized lipodystrophy, a condition where the body can't store fat properly, leading to problems like diabetes and high triglycerides. It aims to see if the drug safely improves these metabolic issues.
This trial tests a new drug called mibavademab for people with severe obesity caused by changes in the leptin (LEP) gene. It aims to see if the drug can help with weight loss in children, teens, and adults who have this rare genetic condition.
This registry follows people who are already taking metreleptin (a medicine for certain rare conditions) to better understand its long-term safety and how well it works in everyday use. It may help you and other patients by building real-world evidence about this treatment.
This trial tests an open-label study of metreleptin in people with partial lipodystrophy, a condition where fat is lost from some areas of the body. The goal is to see if the drug can help manage related health problems.
This Phase 2 study tests pegvisomant, a medicine that blocks growth hormone, to see if it can improve severe insulin resistance in people with a specific genetic cause or a rare fat distribution condition. If you fit the rare diagnosis, the study may help clarify whether growth-hormone blocking can improve blood sugar and related health markers.
Hear when a new Lipodystrophy trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.