E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Healthy transgender persons (the effect of differences in sex hormones on the amount of bone marrow fat) |
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E.1.1.1 | Medical condition in easily understood language |
Healthy transgender persons (the effect of differences in sex hormones on the amount of bone marrow fat) |
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E.1.1.2 | Therapeutic area | Body processes [G] - Physiological processes [G07] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the effect of sex hormones on bone marrow fat fat |
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E.2.2 | Secondary objectives of the trial |
To determine the effect of sex hormones on visceral and liver fat. And to test whether DXA can be used to calculate the amount of visceral fat in transgender patients and if so, what algorithm to use. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Diagnosed with gender dysphoria according to DSM V (transmen or transwomen) - Age between 18 and 50 years - Transmen need to be premenopausal - Starting cross-sex hormone treatment |
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E.4 | Principal exclusion criteria |
• Previous use of cross-sex hormones • Contraindications to MRI scanning as determined by the standard VUmc checklist (See F4. Checklist MRI) • Use of bone-modifying or adipose tissue-modifying drugs, current or in history o Bisphosphonates (alendronic acid, clodronic acid, ibandronic acid, pamidronic acid, risedronic acid, zoledronic acid, etidronate) o Estrogen receptor modulators (raloxifene, bazedoxifene) o Calcium regulating agents (denosumab, calcitonin, teriparatide, strontium ranelate, cinacalcet, etelcalcetine) o Corticosteroids ( • Bone or bone marrow diseases, current or in history o Metabolic (osteroporosis, osteomalacia, dystosis, osteodystrophia, Pagets disease, osteogenesis imperfecta) o Malignancy (primary, metastatic) o Infectious (osteomyelitis, periostitis) o Mechanic (lumbal vertebral fracture) • Bone marrow diseases (leukemia, myelodysplastic syndrome, myeloproliferative disorders) • Platelet count <120*109/l • History of non-traumatic major bleeding • Known bleeding diathesis • Conditions which require antiplatelet therapy • Usage of antiplatelet therapy • Chronic usage of medication known to influence platelet function (e.g. DOAC’s, NSAIDs, warfarin)
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E.5 End points |
E.5.1 | Primary end point(s) |
The main study parameter is the change in vertebral bone marrow fat fraction, measured by MRI quantitative chemical shift imaging (QCSI) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline (before start of hormones), week 6 (after start GnRH analogues), week 8 (after start cross-sex hormones), week 18, week 58 |
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E.5.2 | Secondary end point(s) |
- Changes in visceral and liver fat in cm2, measured by MRI and DXA - Bone mineral density in g/cm2, measured by DXA - Bone turnover markers (Ctx, P1NP, osteocalcin) - Leptin and adiponectine level - Inflammation markers (e.g. IL-6, HsCRP, G-CSF) - Platelet activation (e.g. PFA-200 paremeters, plasma thromboxane B2, flow-cytometry) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline (before start of hormones), week 6 (after start GnRH analogues), week 8 (after start cross-sex hormones), week 18, week 58 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The last visit of the last subject undergoing the trial |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |