An open label safety and efficacy study of allogenic adipose tissue-derived mesenchymal stromal cells on autism and gastrointestinal symptoms

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Cell to Cure ApS

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Psychiatry and Psychology [F] - Mental Disorders [F03], Diseases [C] - Digestive System Diseases [C06]

Decision date (initial)

2026-01-23

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess safety of intravenous infusion of in vitro expanded adipose tissue-derived mesenchymal stromal cells (ASCs) used for allogenic treatment of children with autism spectrum disorder (ASD) and gastrointestinal symptoms.

Secondary objectives 2

1. Autism spectrum disorder behavioral test
2. Gastrointestinal symptoms

Conditions and MedDRA coding

Gastrointestinal symptoms

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Aged 6-18 years (both inclusive)
2. Diagnosis of autism spectrum disorder
3. Ongoing gastrointestinal symptoms

Exclusion criteria 5

1. If known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder
2. Known CNS-infection (now or previously) and/or HIV positivity
3. Primary immunodeficiency disorder or autoimmune cytopenia
4. Celiac disease or inflammatory bowel disease (Crohn’s disease or ulcerative colitis)
5. Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other anti-inflammatory medication (except nonsteroidal anti-inflammatory drugs)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of adverse reactions (ARs) and serious adverse reactions (SARs)

Secondary endpoints 3

1. Change in Vineland Adaptive Behavior Scales (questionnaire)
2. Change in Children Communication Checklist-2 (questionnaire)
3. Change in IBS-QoL - Irritable Bowel Syndrome Quality of Life (questionnaire)

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Cell to Cure ApS

Sponsor organisation

Cell to Cure ApS

Address

Kajeroedgaard 9

City

Birkeroed

Postcode

3460

Country

Denmark

Scientific contact point

Organisation

Cell to Cure ApS

Contact name

Tania Poulsen

Public contact point

Organisation

Cell to Cure ApS

Contact name

Tania Poulsen

Third parties 1

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Application history

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