Arginine Vasopressin Deficiency, Hypopituitarism, Oxytocin Deficiency, Pediatric Disease
Conditions
Brief summary
An open-labeled, interventional pilot trial, 10 youth with AVP-D and 10 PD matched for age, sex, and BMI will be recruited from Pediatric Endocrinology and Neuroendocrinology at Massachusetts General Hospital and in the community. This study tests the hypothesis that oral estrogen/progestin will stimulate endogenous oxytocin release in control subjects. Eligible participants will receive two tablets in a single administration containing a total of 1 mg of norethindrone acetate 70 mcg of ethinyl estradiol. Sampling for blood and saliva will take place at baseline and approximately 24 hours following study drug administration. Neuropsychological assessment (anxiety, mood and emotion regulation; impulse control; aberrant eating behaviors; social cognition and functioning; quality of life) will be assessed at baseline to characterize the study population.
Interventions
Norethindrone Acetate-Ethinyl Estradiol will be given to participants in both cohorts, arginine-vasopressin deficiency cohort and control cohort.
Sponsors
Massachusetts General Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 21 Years
Healthy volunteers
No
Inclusion criteria
(participants with AVP-D): * AVP-D diagnosed in clinic using standard of care diagnostic tools; * Stable pituitary hormone replacement (no change in dose of hormone replacement in six weeks prior to baseline); * If on estrogen/progestin, female participants agree to stop for at least 6 weeks prior to Main study visits; * English language proficiency. Inclusion criteria (participants with hypopituitary disease): * Hypopituitary disease diagnosis; * If receiving pituitary hormone replacement, no change in dose in six weeks prior to baseline); * If on estrogen/progestin, participants agree to stop for at least 6 weeks prior to Main study visits; * English language proficiency.
Exclusion criteria
(all participants): * History of pulmonary embolism or unprovoked deep venous thrombosis; * History of breast/endometrial cancer as well as current therapies on estrogen modulators/blockers (i.e., tamoxifen, raloxifene, aromatase inhibitors); * History of stroke, transient ischemic attack, myocardial infarction, angina pectoris, or peripheral arterial disease; * Pregnancy or breastfeeding within the last 8 weeks; * Medication changes within 2 weeks of enrollment or within 5 half-lives of the respective medication; * History of stage 3 chronic kidney disease or cirrhosis; * Any significant illness or condition that the investigator determines could interfere with study participation, data collection or safety; * Active tobacco smoking or nicotine patch use; * Psychosis or active suicidality.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in neurophysin-1 from baseline | 0 minutes (Baseline) and 24 hours | Change in neurophysin-1 levels from baseline to 24 hours |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in oxytocin from baseline | 0 minutes (Baseline) and 24 hours | Change in oxytocin from baseline to 24 hours |
Countries
United States
Contacts
CONTACTHannah Lamont, PhD
PRINCIPAL_INVESTIGATORElizabeth A Lawson, MD
Massachusetts General Hospital
Outcome results
None listed