Arginine Vasopressin DeficiencyEarly Phase 1Pedi-EVOLVENCT07568509
Identifying Oxytocin Deficiency in Pediatric Patients With Pituitary Disease
A Early Phase 1 study testing Norethindrone Acetate-Ethinyl Estradiol in people with Arginine Vasopressin Deficiency and Oxytocin Deficiency, run by Massachusetts General Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 21 years
- Conditions the study is for
- Arginine Vasopressin Deficiency, Oxytocin Deficiency, Pediatric Disease
- What is being tested?
- Norethindrone Acetate-Ethinyl Estradiol
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Aug 2026to Apr 2027
- Phase
- Early Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Hannah Lamont, PhD, 617-726-1347, MGHHypopit@MGH.HARVARD.EDU
About this study
Official title: Identifying a Provocation Test for Diagnosis of Oxytocin Deficiency in Youth With Hypopituitarism
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.
- Being tested
- Norethindrone Acetate-Ethinyl Estradiol
- Medical subject headings
- Diabetes Insipidus, Neurogenic, Hypopituitarism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Norethindrone Acetate-Ethinyl EstradiolDrug
Also called Estrogen-progestin
Norethindrone Acetate-Ethinyl Estradiol will be given to participants in both cohorts, arginine-vasopressin deficiency cohort and control cohort.
| Study group | Role | What they receive |
|---|---|---|
| Individuals with hypopituitary disease with Arginine-vasopressin deficiency (AVP-D). | Experimental | Norethindrone Acetate-Ethinyl Estradiol will be given to participants with arginine-vasopressin deficiency. Receives: Drug: Norethindrone Acetate-Ethinyl Estradiol |
| Individuals with hypopituitary disease (without AVP-D) | Experimental | Norethindrone Acetate-Ethinyl Estradiol will be given to control subjects. Receives: Drug: Norethindrone Acetate-Ethinyl Estradiol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in neurophysin-1 from baseline
Change in neurophysin-1 levels from baseline to 24 hours
Measured: 0 minutes (Baseline) and 24 hours
Secondary outcomes (1)
Change in oxytocin from baseline
Change in oxytocin from baseline to 24 hours
Measured: 0 minutes (Baseline) and 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2026
- Study start17 Aug 2026
- Primary completionApr 2027 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Arginine Vasopressin DeficiencyStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Elizabeth Austen Lawson, Massachusetts General Hospital
- Investigators
- Elizabeth A Lawson, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07568509
- Sponsor's protocol ID
- 2025P001958
- Acronym
- Pedi-EVOLVE
- Status last verified
- Aug 2026
- First submitted
- 28 Apr 2026
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No