Effects of Intranasal Oxytocin in Patients With Arginine-vasopressin Deficiency

NCT04789148 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 2020P003071
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This is a randomized, double-blind, placebo-controlled crossover pilot study of single-dose intranasal oxytocin (6 IU and 24 IU) vs. placebo in adult men and women (aged 18 years and above) with arginine-vasopressin deficiency to evaluate the effect of oxytocin on anxiety, depression, and socioemotional functioning (Part A), with an optional randomized, double-blind, placebo-controlled 2-week repeated dose substudy of intranasal oxytocin 6 IU or placebo (Part B). Following a screening visit to determine eligibility, participants will return for three main study visits in Part A. During the main study visits, study participants will receive either oxytocin or placebo, followed by assessments of emotional behavior. In Part A, thirty participants will be equally randomized to one of six possible groups:

1. 1.6 IU oxytocin - 24 IU oxytocin - placebo
2. 2.6 IU oxytocin - placebo - 24 IU oxytocin
3. 3.24 IU oxytocin - 6 IU oxytocin - placebo
4. 4.24 IU oxytocin - placebo - 6 IU oxytocin
5. 5.placebo - 6 IU oxytocin - 24 IU oxytocin
6. 6.placebo - 24 IU oxytocin - 6 IU oxytocin

Conditions

Vasopressin Deficiency

Keywords

Hypopituitarism; posterior pituitary; oxytocin; psychopathology; anxiety; depressive symptoms; socioemotional functioning

Outcome Measures

Primary Outcomes (1)

• Dot-probe task - anxious behavior between low dose oxytocin and placebo

  Difference in response times (in milliseconds) to dots appearing in the location of the previously shown negative versus the neutral face between 6 IU oxytocin vs placebo in the dot-probe task.

  20 minutes following intervention at each main visit

Secondary Outcomes (3)

• Dot-probe task - anxious behavior between all three interventions

  20 minutes following intervention

• Depressive behavior - probabilistic reward task between all three interventions

  30 minutes following intervention at each main visit

• Socioemotional functioning - Emotion recognition task between all three interventions

  40 minutes following intervention at each main visit

Study Arms (8)

Part A Arm 1

EXPERIMENTAL

Main visit 1: 6 IU intranasal oxytocin; Main visit 2: 24 IU intranasal oxytocin; Main visit 3: intranasal placebo

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part A Arm 2

EXPERIMENTAL

Main visit 1: 6 IU intranasal oxytocin; Main visit 2: intranasal placebo; Main visit 3: 24 IU intranasal oxytocin

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part A Arm 3

EXPERIMENTAL

Main visit 1: 24 IU intranasal oxytocin; Main visit 2: 6 IU intranasal oxytocin; Main visit 3: intranasal placebo

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part A Arm 4

EXPERIMENTAL

Main visit 1: 24 IU intranasal oxytocin; Main visit 2: intranasal placebo; Main visit 3: 6 IU intranasal oxytocin

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part A Arm 5

EXPERIMENTAL

Main visit 1: intranasal placebo; Main visit 2: 6 IU intranasal oxytocin; Main visit 3: 24 IU intranasal oxytocin

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part A Arm 6

EXPERIMENTAL

Main visit 1: intranasal placebo; Main visit 2: 24 IU intranasal oxytocin; Main visit 3: 6 IU intranasal oxytocin

Drug: Intranasal Oxytocin (IN-OXT); Drug: Placebo

Part B Arm 1

ACTIVE COMPARATOR

Intranasal oxytocin 6 IU three times a day for 14 days

Drug: Intranasal Oxytocin (IN-OXT)

Part B Arm 2

EXPERIMENTAL

Intranasal placebo three times a day for 14 days

Drug: Placebo

Interventions

Intranasal Oxytocin (IN-OXT) DRUG

6 IU single dose

Part A Arm 1; Part A Arm 2; Part A Arm 3; Part A Arm 4; Part A Arm 5; Part A Arm 6

Placebo DRUG

Intranasal placebo three times a day for 2 weeks

Part A Arm 1; Part A Arm 2; Part A Arm 3; Part A Arm 4; Part A Arm 5; Part A Arm 6

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 and above
• Arginine-vasopressin deficiency
• Normal FT4 or T4
• Normal serum/plasma sodium
• Stable hormone replacement

You may not qualify if:

• Active substance use disorder within the last 6 months
• History of psychosis
• Suicidal behavior and/or active suicidal ideation with plan and/or intent, e.g., suicidal ideation of type 4 or type 5 as assessed by the Columbia Suicide Severity Rating Scale (C-SSRS), in the last month
• Medication changes within 4 weeks of enrollment or planned medication changes during the study
• History of chronic nasal obstruction or local pathology in nostril pathway which, in the opinion of the investigator, would prevent appropriate nasal administration of the study drug.
• History of chronic kidney disease stage III and above
• History of liver cirrhosis
• Pregnancy or breastfeeding within the last 8 weeks
• Unwilling to use a medically acceptable form of contraception throughout the study period (female of child-bearing potential only)
• Any significant illness, condition, drug or medical device that the Investigator determines could interfere with study participation, data collection, or safety

Sponsors & Collaborators

• Elizabeth Austen Lawson: lead
• Tonix Pharmaceuticals, Inc.: collaborator

Study Sites (1)

Massachusetts General Hospital, Neuroendocrine Unit

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Diabetes Insipidus, Neurogenic; Hypopituitarism; Anxiety Disorders; Depression

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; Mental Disorders; Behavioral Symptoms; Behavior

Study Officials

• Elizabeth A Lawson, MD, MMSc

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Francesca Galbiati, MD

CONTACT

(617) 726-3870; FGALBIATI@BWH.HARVARD.EDU

Elisa Asanza, MSN, MPH

CONTACT

617-726-3870; easanza@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: BASIC SCIENCE
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate Professor of Medicine, Harvard Medical School; Director, Interdisciplinary Oxytocin Research Program, Neuroendocrine Unit, Massachusetts General Hospital

Model Details: Randomized, double-blind, placebo-controlled, single dose crossover study evaluating 2 different dose levels of intranasal oxytocin vs placebo (Part A), followed by Part B, an optional 2-week randomized, double-blind, placebo-controlled substudy investigating intranasal oxytocin 6 IU vs. placebo three times a day.

Study Record Dates

• First Submitted: March 5, 2021
• First Posted: March 9, 2021
• Study Start: September 10, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)