Use of low doses of interleukin-2 in autism spectrum disorders

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Mental Disorders [F03]

Decision date (initial)

2025-11-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

DGOS - Ministry of Health

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the stimulation of the Tregs of 6 to 8 -year-old children with ASD whose mothers had ASI during pregnancy, by low doses of interleukin-2 (ILT-101) on day 8 versus placebo.

Secondary objectives 6

1. To evaluate in children aged 6 to 8 years with ASD whose mother presented with ASI during pregnancy
2. Clinical effect: - To assess the effect at D85 and D169 of low doses of interleukin-2 (ILT-101) versus placebo on the Vineland II global score and the persistent effect at D275.
3. Evaluate the effect at D85 and D169 of low-dose interleukin-2 (ILT-101) versus placebo on the "communication", "socialisation" and "life" sub-scores. of the Vineland II and the residual effect at D275
4. Effect at D85 and D169 of ILT-101 versus placebo on the patient's other clinical dimensions (social cognition, repetitive behaviour and stereotypies, hyperactivity) and the residual effect at D275
5. Biological effect: measurement of Tregs,Th17 and CD25 at D0, D8, D29) then at , D8 and D169 and the residual effect at D275; as well as the correlation between the biological response and socio-communicative symptoms at D85 and D169 and the residual effect at D275;
6. Tolerance at D0, D8, D85, 169

Conditions and MedDRA coding

Autism Spectrum disorder ASD

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Age 6 to 8 years
2. Severity of ASD considered moderate or severe on the ADOS
3. Meeting DSM-5 criteria for autism spectrum disorder
4. Mother with : (i) an autoimmune disease (as listed by the American Autoimmune Related Diseases Association: https://www.aarda.org/diseaselist/) that began during the first and second trimesters of pregnancy, or that was present prior to pregnancy and experienced a relapse (defined as a change in disease activity leading to a change/modification of treatment) during pregnancy; (ii) a maternal infection (viral or bacterial) during pregnancy, defined as a fever greater than 38.5°C for at least 48 hours and documented (medical consultation, biological sample, prescription of antipyretic and/or antibiotic). Infections by a pathogen with a well-documented direct cerebral effect (CMV) will be excluded
5. Consent of parental authority and social security affiliation
6. One of whose parents lives in the HAD pediatric intervention area

Exclusion criteria 8

1. Recent change in ASD management (behavioral therapy within 6 weeks, introduction of psychotropic molecules within 2 weeks)
2. Contraindication to IL2 use (hypersensitivity, cancer history, active infection, obesity, transplant history, vaccination with live attenuated vaccine within 4 weeks)
3. Participation in another therapeutic trial within the last 3 months
4. BMI >95th percentile or BMI <5th percentile
5. Participants who have already received a genetic diagnosis of ASD of the ‘syndromic’ type by DNA chip chromosome analysis
6. Participants with hyperchloremia or hypernatremia
7. Participants who are related to a person involved in the study at the investigating centre, the clinical research organisation (CRO) or the sponsor.
8. Participant with uncontrolled epilepsy.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in Tregs (in % of CD4+ cells and absolute value) between baseline and D8, compared with ILT-101 and placebo.

Secondary endpoints 16

1. Clinical:
2. Socio-adaptive symptoms : Vineland II Adaptive Behavior Composite- Total Score [at D0, D85, D169 and D275].
3. Social Cognition: Vineland II Adaptive Behavior Composite- Score Socialization Domain [at D0, D85, D169 and D275].
4. Communication: Vineland II Adaptive Behavior Composite - Communication Domain Score [at D0, D85, D169 and D275].
5. Global functional impact: Vineland Adaptive Behavior Composite - Daily Life Domain Score [at D0, D85, D169 and D275].
6. Social Communication: Brief Observation of Social - Communication Change (BOSCC) [at D0, D85, D169 and D275] Social Cognition
7. Social cognition: Social Responsiveness Scale - total score [at D0, D85, D169 and D275].
8. Social cognition: Autism Diagnostic observation schedule-2 [at D0, 169 and D275].
9. Repetitive behaviour and stereotypies: Aberrant Behavior Checklist [at D0, D85, D169 and D275].
10. Hyperactivity: ADHD Rating Scale parent report- total score [at D0, 85, 169 and D275].
11. Global functional impact: Clinical Global Improvement - [at D0, D85, D169 and D275].
12. Global functional impact: Caregiver Strain Index - [at D0, D85, D169 and D275]
13. Biological:
14. Treg, Th17 and CD25 assays (in % of CD4+ and absolute value) [at D0, D8, D29, D85, 169 and D275], and AUC (D0-D29 / D29-169)
15. Tolerance:
16. Pediatric adverse event rating scale [at D0, D8, D85, D169]

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr. Pierre ELLUL

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr. Pierre ELLUL

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Results and documents

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

Documents 7 files

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

Application history

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