Overview
Sponsor-declared trial summary
Phase
Phase III and Phase IV (Integrated)
Status
Ongoing, recruiting
Participants planned
80
Countries
1
Sites
1
Autism spectrum disorder (ASD)
To evaluate the efficacy of multiple-dose oxytocin treatment on core autism symptoms using standardized assessments in children with ASD and comorbid ID. Change from baseline after oxytocin administration on: -Behavioral measurements of core autism (social) symptoms -Cardio electrophysiological measurements of stress
Key facts
- Sponsor
- UZ Leuven
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Psychiatry and Psychology [F] - Behavior and Behavior Mechanisms [F01]
- Trial duration
- 30 May 2023 → ongoing
- Decision date (initial)
- 2024-09-20
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-513436-14-00
- EudraCT number
- 2022-002423-36
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy
To evaluate the efficacy of multiple-dose oxytocin treatment on core autism symptoms using standardized assessments in children with ASD and comorbid ID.
Change from baseline after oxytocin administration on:
-Behavioral measurements of core autism (social) symptoms
-Cardio electrophysiological measurements of stress
Secondary objectives 2
- Change from baseline after oxytocin administration on other behavioral outcomes (e.g. sleep quality, parental stome assessments)
- The clinical trial also includes exploratory outcome measures examining treatment-mechanistic aspects of the oxytocin treatment: 1. Oxytocin and cortisol hormonal levels 2. Other neurophysiological measurements of stress 3. Microbiome assessments
Conditions and MedDRA coding
Autism spectrum disorder (ASD)
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 2
- Children within an age-range of 4 to 13 years old with a clinical multidisciplinary diagnosis of ASD; only premenstrual girls will be included (girls with onset of menstruation during the course of the trial are allowed to continue the treatment).
- Children must be enrolled in a special need school, more specific a class comprising children with an intellectual disability or meet intellectual disability criteria; a total IQ below 75 and an ABAS score below 70
Exclusion criteria 6
- Patient activelytakes anti-epileptic medication or has an active medical problem like cardiac pathology or serious liver, renal problems which influence the metabolism of oxytocin (IMP)
- Patient has a known syndrome that interacts with the reproductive hormonal system (e.g. Prader-Willi or Angelman syndrome)
- Significant hearing or vision impairments
- Subjects who have had previous chronic treatment with oxytocin
- Participation in another clinical trial with IMP
- Known hypersensitivity to active substance or ingredients of the nasal spray
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 3
- The Brief Observation of Social Communication Change (BOSCC) (clinician-rated)
- The Autism Treatment Evaluation Checklist (ATEC) (parent (caregiver) and teacher-rated)
- Recordings of electrocardiography (ECG) for the assessment of heart rate variability (HRV) during rest
Secondary endpoints 5
- The Repetitive Behavior Scale - Revised (RBS-R) (parent (caregiver) and teacher-rated)
- The Adaptive Behavior Assessment System (ABAS-3) (parent (caregiver)-rated)
- The Developmental Behaviour Checklist (DBC) (parent (caregiver)-rated)
- The Perceived Stress Scale (PSS) (parent (caregiver)-rated)
- The Child’s Sleep Habits Questionnaire Abbreviated (CSHQ-A) (parent (caregiver)-rated)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 2
Syntocinon 40 IE/ml neusspray, oplossing
PRD5383601 · Product
- Active substance
- Oxytocin
- Pharmaceutical form
- NASAL SPRAY, SOLUTION
- Route of administration
- NASAL SPRAY
- Max daily dose
- 24 IU international unit(s)
- Max total dose
- 288 IU international unit(s)
- Max treatment duration
- 12 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01BB02 — OXYTOCIN
- Marketing authorisation
- RVG 03716
- MA holder
- ALFASIGMA S.P.A.
- MA country
- Netherlands
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Oxytocine CD Pharma 40 IE/ml neusspray, oplossing
PRD11616267 · Product
- Active substance
- Oxytocin
- Substance synonyms
- GR121619
- Pharmaceutical form
- NASAL SPRAY, SOLUTION
- Route of administration
- INTRANASAL USE
- Max daily dose
- 24 IU international unit(s)
- Max total dose
- 288 IU international unit(s)
- Max treatment duration
- 12 Day(s)
- Authorisation status
- Authorised
- ATC code
- H01BB02 — OXYTOCIN
- Marketing authorisation
- RVG 03716
- MA holder
- CD PHARMACEUTICALS AB
- MA country
- Netherlands
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 1
sodium chloride nose drops 0.9%
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
UZ Leuven
- Sponsor organisation
- UZ Leuven
- Address
- Herestraat 49
- City
- Leuven
- Postcode
- 3000
- Country
- Belgium
Scientific contact point
- Organisation
- UZ Leuven
- Contact name
- Kaat Alaerts
Public contact point
- Organisation
- UZ Leuven
- Contact name
- Kaat Alaerts
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ongoing, recruiting | 80 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2023-05-30 | 2023-05-30 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 27 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-513436-14 For publication | 8 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements - For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Brochure_qualitative_EN For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Brochure_qualitative_NL For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Brochure_quantitative_EN For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Brochure_quantitative_FR For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Brochure_quantitative_NL For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Flyer_qualitative_EN For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Flyer_qualitative_NL For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Flyer_quantitative_EN For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Flyer_quantitative_FR For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Flyer_quantitative_NL For publication | 1 |
| Recruitment arrangements (for publication) | K2_Recruitment material - Tekst website For publication | 1 |
| Recruitment arrangements (for publication) | L2_Other subject information material description_informatiebrief_EN For publication | 1 |
| Recruitment arrangements (for publication) | L2_Other subject information material description_informatiebrief_kwalitatief_NL For publication | 1 |
| Recruitment arrangements (for publication) | L2_Other subject information material description_informatiebrief_kwantitatief_EN For publication | 1 |
| Recruitment arrangements (for publication) | L2_Other subject information material description_informatiebrief_kwantitatief_FR For publication | 1 |
| Recruitment arrangements (for publication) | L2_Other subject information material description_informatiebrief_kwantitatief_NL For publication | 1 |
| Subject information and informed consent form (for publication) | L1_ICF_qualitative_EN For publication | 6 |
| Subject information and informed consent form (for publication) | L1_ICF_qualitative_NL For publication | 8 |
| Subject information and informed consent form (for publication) | L1_ICF_quantitative_EN For publication | 6 |
| Subject information and informed consent form (for publication) | L1_ICF_quantitative_FR For publication | 6 |
| Subject information and informed consent form (for publication) | L1_ICF_quantitative_NL For publication | 8 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Oxytocin CD Pharma For publication | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Oxytocin CD Pharma For publication | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Syntocinon_Alfasigma For publication | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis MS 2024-513436-14 | 1 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-09-12 | Belgium | Acceptable with conditions 2024-09-20
|
2024-09-20 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-01-24 | Belgium | Acceptable 2025-04-04
|
2025-04-04 |