Researchers from King's College London have announced details of a new clinical trial to evaluate puberty-blocking drugs in children with gender incongruence, marking the first such study since the UK government banned these medications for gender treatment in 2023.
The trial, called Pathway, will involve approximately 220 children under age 16 who are experiencing gender incongruence and have reached puberty. The study aims to provide crucial evidence on the safety and efficacy of puberty suppressing hormones (PSH) following concerns raised by the Cass review, which highlighted the absence of robust clinical data supporting their use in young people.
Study Design and Participant Selection
The randomized controlled trial will divide participants into two groups: one receiving puberty blockers immediately and another starting treatment 12 months later. All participants must meet strict eligibility criteria and undergo intensive medical and psychological screening before enrollment.
Prof Emily Simonoff, the study leader and professor of child and adolescent psychiatry at King's College London, emphasized the research focus: "We know there are ongoing societal discussions about gender transition, but this research is focused solely on informing and improving healthcare by better understanding how to support the physical and mental health of young people with gender incongruence."
Participants must demonstrate sufficient understanding of the treatment's potential impacts to provide informed consent, with additional consent required from parents or legal guardians. The research team has established that there will be no minimum age requirement, though puberty typically begins around age 11 for girls and 12 for boys.
Comprehensive Outcome Measures
The trial will examine multiple aspects of participants' health and development over a four-year period. Key outcome measures include bone density, brain development, and mental health and wellbeing. A specialist team of NHS doctors must conduct thorough assessments of each young person's overall wellbeing before determining treatment suitability.
Alongside the main trial, researchers plan to conduct a larger observational study involving 3,000 children to evaluate different types of support and their effectiveness. A second study, PATHWAYS CONNECT, will specifically examine brain health in those receiving puberty suppression compared to those receiving only psychosocial and non-medical care.
Regulatory Context and Timeline
The trial emerges from recommendations in the Cass review of gender identity services for children and young people in the UK, which identified a critical need for evidence-based research in this area. Following the review's findings, the UK government implemented an indefinite ban on puberty blockers for gender treatment, restricting their use to research settings only.
The study has received ethical approval and is expected to begin in January, with recruitment of five to six children per month. First results are anticipated in approximately four years, providing the medical community with long-awaited data on these treatments' safety and efficacy profile.
Controversy and Opposition
The trial has generated significant controversy within the medical and advocacy communities. Keira Bell, who previously challenged the Tavistock gender clinic in court after receiving puberty blockers and cross-sex hormones as a teenager, has threatened judicial review proceedings if the trial proceeds. She described the study as "disgusting," arguing that children should not receive drugs that have been banned due to safety concerns.
Some clinicians from the Clinical Advisory Network on Sex and Gender have also questioned the trial's ethical framework, raising concerns about testing treatments on children when safety data remains insufficient.
However, supporters argue the research is essential for evidence-based care. A Stonewall spokesperson stated that all young people should have access to excellent medical care guided by evidence, urging investment in delivering quality healthcare for transgender youth while ensuring their voices remain central to policy decisions.
The trial represents a significant step toward addressing the evidence gap identified by the Cass review, potentially providing the scientific foundation needed to inform future treatment guidelines for young people with gender incongruence.
