Leuprolide for Precocious Puberty

This trial tests whether the drug Leuprolide Mesylate, a hormone therapy, is safe and effective for children with central precocious puberty (early puberty due to hormone issues). Participants will receive two injections of the drug, six months apart, to determine its effectiveness in managing their condition. Suitable candidates are children aged 2 to 8 for girls and 2 to 9 for boys, who show early signs of puberty, such as breast development or increased testicle size, and have not started other treatments for this condition. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

The trial does not specify if you need to stop taking your current medications. However, if you are currently using medications associated with seizures or convulsions, you may need to stop them as they are excluded from the trial.

Research has shown that Leuprolide Mesylate, a treatment for early puberty, is generally safe. Studies have found that its six-month injection has a safety record similar to other treatments, with manageable and expected side effects. For instance, a long-term study found that Leuprolide Mesylate does not affect body weight or cause early puberty to return later. It can also help children grow taller as adults. Overall, children with early puberty tolerate Leuprolide Mesylate well.¹²³⁴⁵

Leuprolide Mesylate is unique because it offers a potentially more convenient dosing schedule for managing central precocious puberty. Unlike standard treatments that often require monthly injections, this new formulation only needs to be administered twice in a year, with doses given six months apart. This can significantly reduce the treatment burden for young patients and their families, making it an exciting advancement in treatment options. Researchers are optimistic that this less frequent dosing will maintain effectiveness while improving patient compliance and quality of life.

Research shows that Leuprolide Mesylate effectively treats early puberty, known as central precocious puberty (CPP). Studies on similar versions, such as the 1-month depot, have demonstrated positive effects on growth and help maintain normal adult height. After treatment, patients typically return to a normal puberty process, and girls often have regular periods by age 12. In this trial, participants will receive a 6-month depot version of Leuprolide, which other studies have found to effectively control hormone levels and growth in children with CPP. Overall, leuprolide treatments have safely managed early puberty symptoms, supporting their use for this condition.³⁴⁶⁷⁸