Navepegritide for Achondroplasia

(teACH Trial)

This trial tests a new treatment called navepegritide for adolescents with achondroplasia, a genetic condition affecting bone growth. The main goal is to determine if this treatment can increase growth speed over a year compared to a placebo (an inactive substance). Participants will receive weekly injections for 52 weeks. Eligible participants must have a confirmed diagnosis of achondroplasia and be between the ages of 12 and 18. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

The trial does not specify if you need to stop taking your current medications, but it does exclude those who have taken medications affecting growth or require chronic use of certain steroids. It's best to discuss your specific medications with the trial team.

Research has shown that navepegritide is generally safe for children with achondroplasia. Studies have found that the treatment is well-tolerated, with only a few minor reactions at the injection site. In one study, the safety of navepegritide matched that of a placebo, indicating no increase in side effects compared to a non-active treatment.

Unlike the standard treatments for achondroplasia, which primarily focus on symptom management, Navepegritide represents a novel approach by targeting the underlying growth pathways. Researchers are excited about Navepegritide because it works by specifically modulating the signaling pathways involved in bone growth, potentially addressing the root cause of the condition rather than just alleviating symptoms. This treatment is administered through a once-weekly subcutaneous injection, which might offer a more convenient and less invasive option compared to current therapies requiring daily administration. By offering a different mechanism and a more patient-friendly delivery method, Navepegritide could significantly improve the quality of life for those with achondroplasia.

Research has shown that navepegritide, which participants in this trial may receive, can help children with achondroplasia grow taller. Specifically, studies found that children taking navepegritide grew an average of 5.89 cm per year, exceeding the growth of those on a placebo. This indicates the treatment can effectively increase growth rates. Additionally, the treatment has demonstrated safety and benefits for bone health. Overall, the evidence supports its potential to improve growth in adolescents with this condition.¹²³⁴⁵