Puberty Suppressing Hormones: Clinical Trials

During scientific dialogue, the age of entry was a key area of consideration between the sponsors and the Medicine and Healthcare products Regulatory Agency (MHRA) assessment team. This scientific dialogue has resulted in strengthened safeguards including the introduction of minimum ages of entry to the trial and more clearly defined discontinuation measures for safety around bone health, cognition, and vaginal bleeding as well as more detailed information for participants on fertility preservation.

It was recognised that an appropriate balance needed to be struck between the maturity level of participants and providing the treatment at the most clinically appropriate point in puberty. In particular, it was noted that delaying treatment until late puberty would reduce the effectiveness of puberty suppressing hormones, or medicines that pause puberty, and hence the trial’s ability to explore how this treatment impacts the physical, social, and emotional wellbeing of young people with gender incongruence.

It is important to note that the ages set are the minimum ages and young people would only enter the trial once the clinical national multidisciplinary team (NMDT) have reviewed and confirmed clinical eligibility. The NMDT's recommendations are based on a holistic assessment framework, taking into account the child's capacity to assent or consent, parental capacity to consent, and the overall impact on the young person's development and wellbeing.

Following this dialogue, the trial sponsor proposed a minimum entry age of 11 years old for girls and 12 years old for boys, which was agreed by the MHRA. This reflects a balanced approach to ensure the treatment is studied at the point it is most clinically relevant while maintaining appropriate safeguards for participant safety.