LEAP - Familial Partial Lipodystrophy Research Study

STUDY BASICS

Have you been diagnosed with familial partial lipodystrophy (FPLD)? You may be able to participate in a study to see if the investigational study drug called REGN4461 can help in improving some of the symptoms and additional medical challenges experienced by people who have FPLD.

STUDY PURPOSE

The purpose of the study is to see if an investigational drug REGN4461 can help in improving some of the symptoms and additional medical challenges experienced by people who have FPLD.

COULD THIS STUDY BE RIGHT FOR YOU?

Eligibility:

• At least 18 years of age
• Diagnosis of having familiar partial lipodystrophy
• High fasting triglycerides (fat in the blood) with or without diabetes (high amount of sugar in the blood)
• Have not taken metreleptin in the last 3 months

WHAT PARTICIPANTS CAN EXPECT

This study involves a screening period to be sure you are eligible to participate. This period will last up to 10 weeks and will include up to 3 visits to the study clinic. If eligible after screening, you will be enrolled into the study and randomly chosen (like the flip of a coin) to receive either 24 weeks of the study drug REGN4461 or placebo for 12 weeks and REGN4461 for 12 weeks. You will not know which treatment you are receiving. During the treatment period, you will be asked to attend three study center visits where you will be given REGN4461 or placebo into a vein using a needle. This is called an intravenous (IV) infusion. Following these visits, you will receive weekly subcutaneous injections; you will either be given these injections at the study center or at your home (if you have opted for the home nursing visits). At the end of the treatment period, you will have 5 more visits to the study clinic 4 weeks apart for the main purpose of following up with you for safety. Participants will receive $75 for each completed visit.