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Oxytocin Substitution Therapy in Patients With Central Diabetes Insipidus

Phase 2
Recruiting
Conditions
Central Diabetes Insipidus (cDI)
Interventions
Drug: Intranasal OXT
Other: Placebo nasal spray
Registration Number
NCT06036004
Lead Sponsor
University Hospital, Basel, Switzerland
Brief Summary

This randomized, placebo-controlled, double-blind trial aims to investigate intranasal OXT as a novel therapeutical option in central diabetes insipidus (cDI) to improve psychological symptoms and socio-emotional functioning.

Optionally, patients can present for additional assessments in sub-studies:

* fMRI sub-study at day 14 (± 2 days) (one additional visit)

* Social-stress sub-study at day 14 (± 2 days) (one additional visit)

Detailed Description

Arginine vasopressin (AVP) and oxytocin (OXT) are hormones released into circulation from the posterior pituitary. While AVP acts mainly in the kidneys and causes reuptake of free water, OXT is well-known for its key role in the regulation of complex social-emotional functioning including attachment and pair bonding, fear possessing, emotion recognition, and empathy.

Disruption of the hypothalamic-pituitary axis can cause AVP deficiency

- known as central diabetes insipidus (cDI) - characterized by polyuria and polydipsia. Once diagnosed, desmopressin (an AVP receptor analogue) can be effectively used to treat diabetes insipidus. However, despite treatment with desmopressin, patients often report residual psychological symptoms, particularly heightened anxiety levels, depressed mood, impairment in social interactions, leading to an overall reduced quality of life.

Due to the anatomical proximity, local disruptions of the AVP system could also disturb the OXT system leading to an additional OXT deficiency. The additional OXT deficiency could explain (at least partially) the residual psychological deficits in patients with cDI. OXT replacement therapy to improve psychological symptoms would have great clinical implications. This randomized, placebo-controlled, double-blind trial aims to investigate intranasal OXT as a novel therapeutical option in cDI to improve psychological symptoms and socio-emotional functioning.

Optional fMRI sub-study: Participants will undergo a structural sequence to investigate grey and white matter anatomy (T1- weighted). Functional neuronal responses will be assessed through the surrogate of blood oxygenated level dependent (BOLD) signal, an indirect measure of neural activity. Three functional sequences (echo planar imaging, EPI) will investigate group differences in various aspects of brain activity: a resting state sequence and the EFMT.

Optional Social-stress sub-study: The three main components are an anticipation phase, a 5-minute interview, and a surprise mental arithmetic task. Acute stress is measured by cortisol increase.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
112
Inclusion Criteria
  • Adult patients with a confirmed diagnosis of central diabetes insipidus based on accepted criteria
  • Heightened anxiety levels (STAI - Trait subscale ≥ 39 score points) or alexithymia levels (impaired ability to identify and describe feelings; TAS-20 total ≥ 52 score points)
  • Stable hormone replacement therapy for at least three months with desmopressin and, in case of additional anterior pituitary deficiencies, with the respective substitution therapies.
Exclusion Criteria
  • Participation in a trial with investigational drugs within 30 days
  • Active substance use disorder within the last six months
  • Consumption of alcoholic beverages >15 drinks/week
  • Current or previous psychotic disorder (e.g., schizophrenia spectrum disorder)
  • Pregnancy and breastfeeding within the last eight weeks
  • Unwilling to use a medically acceptable form of contraception throughout the study period (female of childbearing potential only)
  • Prolonged QTc-time >470 ms assessed with a 12-lead electrocardiogram.
  • Regular use (> 3 times per week) of sympathomimetic drugs (e.g., bronchodilators)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Study Product Intervention: intranasal OXTIntranasal OXTIntranasal OXT spray of 40 IU per ml (Syntocinon®).
Control Intervention: placebo nasal sprayPlacebo nasal sprayThe placebo nasal spray will be identical in volume, labelling, container system, and other features.
Primary Outcome Measures
NameTimeMethod
Change in State-Trait Anxiety Inventory (STAI) questionnaire to assess general anxiety levelsDay 0, day 1, day 14, day 28

Questionnaire with scores ranging from 1 ("almost never") to 4 ("almost always"). The STAI has two sub-scales, the State-Anxiety Scale (STAI-S; 20 items) and the Trait-Anxiety Scale (STAI-T; 20 items). The STAI-S evaluates the current state of anxiety, asking how respondents feel "right now," using items that measure subjective feelings of apprehension, tension, nervousness, worry, and activation/arousal of the autonomic nervous system. The STAI-T evaluates relatively stable aspects of "anxiety proneness," including general states of calmness, confidence, and security. The total scores range from 20 to 80, with higher scores indicating more pronounced anxiety. A score above 39/80 indicates clinically significant anxiety symptoms.

Change in EmBody/EmFace to assess recognition of facial and body expressionsDay 0, day 1, day 28

The EmBody and EmFace subtasks comprise each of 42 stimuli showing body or facial expressions of angry, happy, or neutral affect. Stimuli last 1.5 seconds at 24 frames per second and are geometrically and optically standardised to prevent biases induced by ethnic cues. Each trial consists of one point-light display (PLD), followed by a response window during which participants are asked to indicate via mouse input which emotion they believe was portrayed in the PLD in a three-option forced-choice format (angry-neutral-happy). The total correct classification scores range from 0 to 42 (for each sub-task), with higher scores indicating more correct recognition of facial \& body expressions.

Secondary Outcome Measures
NameTimeMethod
Change in Autism-Spectrum Quotient Test (AQ) to assess the expression of ASD traits in an individual by his or her subjective self-assessmentDay 0, day 28

The AQ consists of 50 items, with four choices for each item from "definitely agree" to "definitely disagree" and a total score from 0 to 50. A score above the proposed cut-off of 29 highlights significant traits of autism

Change in Beck's Depression Inventory II (BDI-II) to assess depressionDay 0, day 28

It uses 21 items ranked from 0 (symptom absent) to 3 (severe symptoms) to measure the severity of depression. The minimum score is 0, and the maximum score is 63. In non-clinical populations, scores above 20 indicate depression. In those diagnosed with depression, the score ≤16, mild mood disturbance; 17 to 20, borderline clinical depression, 21 to 30, moderate depression, and score ≥31, severe depression.

Change in Facial emotion recognition task (FERT) to assess emotion recognition and empathyDay 0, day 28

The FERT assesses the recognition of basic emotions. The task includes ten neutral faces and 160 faces that express one of four basic emotions (i.e., happiness, sadness, anger, and fear), with pictures morphed between 0% (i.e., neutral) and 100% in 10% steps. Stimuli are shown in random order for 500 ms, followed by the rating screen, where participants have to indicate the correct emotion. The outcome measure is accuracy (proportion correct).

Change in Multifaceted Empathy Test (MET) to assess the cognitive and emotional aspects of empathyDay 0, day 1, day 28

The computer-assisted test consists of 40 photographs that showed people in emotionally charged situations. To assess cognitive empathy, the participants are required to infer the mental state of the subject in each scene and indicate the correct mental state from a list of four responses. Cognitive empathy is the percentage of correct responses in the total responses. To measure emotional empathy, the subjects are asked to rate how much they feel for an individual in each scene (i.e., explicit emotional empathy) and how much they are aroused by each scene (i.e., implicit emotional empathy) on a 1-9 point scale.

Change in Hamilton Anxiety Rating Scale (HAM) to assess the severity of anxiety symptomsDay 0, day 28

The scale consists of 14 items, each defined by a series of symptoms, and measures both psychic anxiety (mental agitation and psychological distress) and somatic anxiety (physical complaints related to anxiety). The physician/psychologist is instructed to assess the extent to which the patient displays the given criterion. Each item is independently scored on a scale of 0 (not present) to 4 (severe), with a total score range of 0-56 (\<17 indicates mild severity, 18-24 mild to moderate severity, and 25-30 moderate to severe).

Change in Toronto Alexithymia Scale 20 (TAS-20) to assess emotions experienced by oneself or othersDay 0, day 28

The TAS 20 has a three- factor structure: difficulty identifying and describing feelings and externally oriented thinking. It includes 20 items with scores ranging from 1 (strongly disagree) to 5 (strongly agree). The total scores range from 20 to 100, with higher scores indicating worse alexithymia. A score of ≥61 indicates alexithymia (difficulty understanding one's emotions), a score of 52 to 60 indicates possible alexithymia, and a ≤51 non-alexithymia.

Trial Locations

Locations (2)

Erasmus University Medical Center Rotterdam

🇳🇱

Rotterdam, Netherlands

University Hospital Basel, Department of Endocrinology, Diabetes & Metabolism

🇨🇭

Basel, Switzerland

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