A randomized, placebo-controlled, double-blind, 3-period cross-over study in youth with autism spectrum disorders evaluating neural plasticity and learning after a single administration of intranasal oxytocin

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Oslo University Hospital HF

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Psychological Phenomena [F02], Psychiatry and Psychology [F] - Behavior and Behavior Mechanisms [F01]

Trial duration

26 Jan 2026 → ongoing

Decision date (initial)

2024-05-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Helse Sør-Øst RHF

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Dose response

Identify effects of oxytocin delivered intranasally (8 IU and 24 IU) on neural plasticity and learning compared to placebo in youth diagnosed with ASD

Secondary objectives 2

1. To identify effects of 24 IU and 8IU oxytocin delivered intranasally on parasympathetic nervous system activity in ASD patients
2. To identify the role of trait anxiety and disorder severity on oxytocin treatment response

Conditions and MedDRA coding

Autism spectrum disorder

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Male and female participant between the ages of 12 and 25, both inclusive
2. 1. Participants and their caregivers (if applicable) must be able to communicate with the Investigator, to understand and comply with the requirements of the study, and to understand the oral and written patient information.
3. 2. Participants must have a confirmed diagnosis of autism spectrum disorder (ASD)

Exclusion criteria 13

1. Previous nasal disease, surgery, and dependence on inhaled drugs
2. Current significant nasal congestion due to common colds
3. Clinically relevant history of significant hepatic, renal, endocrine, cardiac, nervous, pulmonary, haematological or metabolic disorder
4. Epilepsy and previous history of photosensitive seizures
5. Systemic illness requiring treatment within 2 weeks prior to Study Day 1
6. Known allergic reactions or hypersensitivity to any component of the study medication in the nasal spray, such as propyl parahydroxybenzoate (E216), methyl parahydroxybenzoate (E218) and chlorobutanol hemihydrate
7. Known allergic reactions or hypersensitivity/intolerance to latex
8. New concomitant medications or formal cognitive/behavioural therapies. If a participant has been taking any medications or receiving formal cognitive/behavioural therapies for at least 4 weeks, then this is not considered a new therapy
9. Participation in any (other) clinical trial with an investigational medicinal product or medical device within 1 month prior to randomization
10. Full scale IQ below 70 (due to the prerequisite ability to complete self-report measures)
11. Pregnancy (self-reported or assessed by pregnancy test prior to the first administration at the experimental sessions for all menstruating individuals)
12. Currently breastfeeding
13. Other unspecified reasons that, in the opinion of the investigator or the sponsor make the participants unsuitable for enrolment

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Neural plasticity will be evaluated using EEG 40 minutes after intervention administration. The visual evoked potential (VEP) paradigm will be used to probe neural plasticity. An increase in neural plasticity will be measured by a statistically significant amplitude modulation when compared to placebo in the following components: C1, P1, N1, N1b and P2. H1: There is a difference between placebo and active treatments. H0: There is no significant difference between placebo and active treatments.
2. Cognitive flexibility will be evaluated behaviourally using the computerized probabilistic reversal learning task. An increase in task performance is measured as a statistically significant decrease in number of incorrect trials and total number of trials needed to reach the learning criterion. H1: There is a difference between placebo and active treatments. H0: There is no significant difference between placebo and active treatments).

Secondary endpoints 2

1. Heart rate variability (HRV), measured using electrocardiogram (ECG) 60 minutes after intervention administration. H1: There is a difference in HRV between placebo and active treatments. H0: There is no significant difference in HRV between placebo and active treatments)
2. The potential moderating effect on intervention response of symptom severity, as indexed by the Social Responsiveness ScaleSecond Edition, Repetitive Behaviour ScaleRevised, Behavioral Inflexibility scale and The Autism Behaviour Inventory. H1: Higher disorder severity will be associated with increased oxytocin treatment response. H0: H0: There is no significant positive association between disorder severity with oxytocin treatment response.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oslo University Hospital HF

Sponsor organisation

Oslo University Hospital HF

Address

Taarnbygget, Kirkeveien 166 Kirkeveien 166

City

Oslo

Postcode

0450

Country

Norway

Scientific contact point

Organisation

Oslo University Hospital HF

Contact name

Daniel S. Quintana

Public contact point

Organisation

Oslo University Hospital HF

Contact name

Daniel S. Quintana

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 15 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications