REGN4461 for Generalized Lipodystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to evaluate how REGN4461, an experimental treatment, affects blood sugar and fat levels in individuals with generalized lipodystrophy, a rare condition where the body cannot store fat properly. Researchers are testing different doses to assess the treatment's effectiveness and safety. Suitable candidates have generalized lipodystrophy and either high blood sugar (HbA1c of 7% or more) or high triglycerides (250 mg/dL or more). Participants should maintain a stable diet and medication plan for at least three months before joining. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it requires that your medication regimen has been stable for at least 3 months before the screening visit. You should discuss your specific medications with the trial team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that REGN4461, a treatment under study for conditions like generalized lipodystrophy, has promising safety results. In earlier studies with patients, REGN4461 was generally well-tolerated, with most participants not experiencing serious side effects.
In studies using mice, REGN4461 improved issues like high blood sugar and insulin resistance without major safety concerns. While these results are encouraging, ongoing research in humans aims to confirm the safety of REGN4461 for people.
Since this treatment is in a Phase 2 trial, initial safety tests in humans have already been completed, but researchers continue to gather more information to ensure its safety and effectiveness for a broader population.12345Why are researchers excited about this trial's treatments?
REGN4461 is unique because it targets the leptin receptor pathway, which is different from most treatments for generalized lipodystrophy that primarily focus on managing symptoms like insulin resistance and high triglyceride levels. Leptin, a hormone involved in regulating energy balance, is often deficient in people with this condition. By specifically activating the leptin receptor, REGN4461 aims to address the underlying hormonal imbalance, offering the potential for more comprehensive management of the disease. Researchers are excited about REGN4461 because it could provide a more direct and effective way to tackle the root causes of generalized lipodystrophy, rather than just alleviating symptoms.
What evidence suggests that REGN4461 might be an effective treatment for generalized lipodystrophy?
Research has shown that REGN4461 may help treat generalized lipodystrophy, a condition where the body can't store fat properly, leading to metabolic problems. In studies with mice, REGN4461 reduced issues like high blood sugar, insulin resistance, and high levels of triglycerides (a type of fat in the blood). The treatment targets the leptin receptor, which is important for controlling metabolism. Early studies in humans also suggested improvements in blood sugar levels and fat profiles. These initial findings offer hope that REGN4461 could effectively manage metabolic issues related to generalized lipodystrophy. Participants in this trial will join one of two experimental treatment arms to further evaluate the effects of REGN4461.12367
Who Is on the Research Team?
Clinical Trial Management
Principal Investigator
Regeneron Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for individuals with Generalized Lipodystrophy who have been on a stable diet and medication regimen for at least 3 months. They must have high blood sugar (HbA1c ≥7%) or high fasting triglycerides (TG ≥250 mg/dL). Those with recent weight loss treatments, metreleptin use, uncontrolled hepatitis B/C or tuberculosis, HIV, or participation in other drug trials recently are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive REGN4461 to evaluate its effects on glycemic parameters and fasting triglyceride levels
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- REGN4461
Find a Clinic Near You
Who Is Running the Clinical Trial?
Regeneron Pharmaceuticals
Lead Sponsor
Leonard Schleifer
Regeneron Pharmaceuticals
Chief Executive Officer since 1988
MD and PhD in Medicine
George Yancopoulos
Regeneron Pharmaceuticals
Chief Medical Officer since 1997
MD from Harvard Medical School