Identifying Oxytocin Deficiency in Pediatric Patients With Pituitary Disease

NCT07568509 | Recruiting Early Phase 1 FDA Drug

• 1 other identifier: 2025P001958
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Arginine Vasopressin Deficiency; Oxytocin Deficiency; Pediatric Disease; Hypopituitarism

Outcome Measures

Primary Outcomes (1)

• Change in neurophysin-1 from baseline

  Change in neurophysin-1 levels from baseline to 24 hours

  0 minutes (Baseline) and 24 hours

Secondary Outcomes (1)

• Change in oxytocin from baseline

  0 minutes (Baseline) and 24 hours

Study Arms (2)

Individuals with hypopituitary disease with Arginine-vasopressin deficiency (AVP-D).

EXPERIMENTAL

Norethindrone Acetate-Ethinyl Estradiol will be given to participants with arginine-vasopressin deficiency.

Drug: Norethindrone Acetate-Ethinyl Estradiol

Individuals with hypopituitary disease (without AVP-D)

EXPERIMENTAL

Norethindrone Acetate-Ethinyl Estradiol will be given to control subjects.

Drug: Norethindrone Acetate-Ethinyl Estradiol

Interventions

Norethindrone Acetate-Ethinyl Estradiol DRUG

Norethindrone Acetate-Ethinyl Estradiol will be given to participants in both cohorts, arginine-vasopressin deficiency cohort and control cohort.

Also known as: Estrogen-progestin

Individuals with hypopituitary disease (without AVP-D); Individuals with hypopituitary disease with Arginine-vasopressin deficiency (AVP-D).

Eligibility Criteria

• Age: 7 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Diabetes Insipidus, Neurogenic; Hypopituitarism

Interventions

norethindrone acetate, ethinyl estradiol, ferrous fumarate drug combination

Condition Hierarchy (Ancestors)

Diabetes Insipidus; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Pituitary Diseases; Endocrine System Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Elizabeth A Lawson, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hannah Lamont, PhD

CONTACT

617-726-1347; MGHHypopit@MGH.HARVARD.EDU

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor of Medicine

Study Record Dates

• First Submitted: April 28, 2026
• First Posted: May 5, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): April 1, 2027
• Last Updated: May 5, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)