Clinical trial NCT04203381: Skeletal Health and Bone Marrow Composition Among Youth
Official title: Skeletal Health and Bone Marrow Composition Among Youth
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Gender Dysphoria in Children
Puberty
Bone Development
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
Collaborators: name: Children's Hospital Medical Center, Cincinnati
First posted: 2019-12-18
Enrollment: 80
Sex: ALL
Minimum age (years): 9
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Children and adolescents experiencing gender dysphoria feel increased distress with the onset of puberty. Gender clinics treat these young adolescents by "blocking" puberty using gonadotropin-releasing hormone (GnRH) agonist medications. This has the possibility of impacting bone development as sex steroids are important to bone mass development. In this multi-site study, the investigators will examine bone marrow composition (by MRI) in 40 transgender youth and bone density and body composition before/after pubertal blockade compared to healthy participants.

Detailed description: The investigators propose to identify the effects of pubertal blockade on bone density and size in pediatric transgender individuals. Information is limited regarding bone health and metabolism for this group, and especially for children who are transgender. Further, the team's expertise in magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), dual-energy x-ray absorptiometry (DXA), and peripheral quantitative computed tomography (pQCT), examining the natal female skeleton in previous NIH funded studies, and extensive clinical expertise, provide an ideal foundation to apply this protocol to examine transgender female and transgender male skeleton. Findings from this study will allow us to identify potential preventative strategies to counter the long-term effects of puberty blockade such as osteoporosis and raise awareness of this medical consequence to medical care providers of transgender patients. The proposed project seeks to answer the clinically relevant question of how bone marrow composition relates to body composition, and its relation to both bone density and skeletal strength, in transgender patients who are undergoing puberty blockade, the first phase of therapy preceding gender-affirming hormone therapy.

Eligibility criteria: Inclusion Criteria:

1. Be willing to give informed consent/assent (parent or legal guardian able to provide informed consent)
2. Tanner Stage II/III
3. Age 9-14 years old for assigned male at birth (AMAB) and 9-13 for assigned female at birth (AFAB)
4. Current patient at the Boston Children's Hospital Transgender Clinic or Cincinnati Children's Hospital Medical Center Clinic, within six weeks of initiating pubertal blockade treatment (e.g., GnRH agonist- Lupron or Vantas)
5. Controls are matched on age within 2 years, race/ethnicity, sex assigned at birth and BMI within 25%

Exclusion Criteria:

The participant must not:

1. Have chronic disease known to affect skeletal metabolism (e.g. cystic fibrosis, celiac disease, sickle cell disease, inflammatory bowel disease etc.)
2. Receipt of other medications within previous 3 months known to affect skeletal metabolism (e.g., glucocorticoids, anticonvulsants, etc.)
3. Have metal implants or hardware in their body that would not allow them to get an MRI
4. Have a diagnosis of a developmental disorder or claustrophobia that would prevent them from undergoing an MRI

Primary outcomes:
measure: Change from Bone marrow adiposity by magnetic resonance imaging (MRI) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from magnetic resonance spectroscopy (MRS) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
Secondary outcomes:
measure: Change from total body bone mineral density Z-score by Dual-energy X-ray absorptiometry (DXA) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from spine BMD Z-score by DXA from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from hip BMD Z-score by DXA from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from volumetric bone mineral density (vBMD) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from section modulus (bone bending strength) by quantitative computed tomography (pQCT) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
Other outcomes:
measure: Change from health-related quality of life (HRQL) from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from anxiety from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
measure: Change from depression from baseline compared to 24 months
timeFrame: Change from baseline compared to 24 months
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
References: