Clinical trial NCT05602116: ASC - Autism Study
Official title: Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Autism Spectrum Disorder
Gastrointestinal Dysfunction
Leaky Gut Syndrome
Mesenchymal Stromal Cells
Inflammatory Bowel Diseases
Keywords: Gastrointestinal microbiota
Gut-brain axis
Immunomodulation
Lead sponsor: Cell2Cure ApS
Sponsor class: INDUSTRY
First posted: 2022-11-01
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Open dose titrating with two groups in extension of each other (thus not categorically parallel).
Primary purpose: TREATMENT

Brief summary: The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms.

Detailed description: The ASC - Autism Pilot Study will be performed at the Child and Adolescent Psychiatry, Aalborg University Hospital, Denmark. 10 children aged 6-14 years with ASD and gastrointestinal symptoms will be enrolled through the Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark.

The participants will randomly be divied into two treatment groups:

1. participant 1-5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
2. participant 6-10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight

C2C_ASC treatment:

The C2C_ASCs product will be diluted in 50 ml isotonic saline and infused in an arm vein within 15 min. In both groups there will for each participant be a week observation for SAE's related to the cell treatment before the next participant will be treated, if no SAE's have been detected.

The investigators hypothesize that the connection between gastrointestinal symptoms/leaky gut syndrome, increase local gastrointestinal and systemic elevated immunological and inflammatory activity, bacterial toxins in the blood and symptoms of ASD in children with symptoms of leaky gut syndrome can be reduced or normalized by modulating the immunological activity and inflammation by treatment with mesenchymal stromal cells.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of autism spectrum disorder.
* Ongoing gastrointestinal symptoms or previous gastrointestinal symptoms which disappeared or were reduced after dietary changes.
* Age 6 to 14 years.

Exclusion Criteria:

* Known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder.
* Known CNS-infection (now or previously) and/or HIV positivity.
* Primary immunodeficiency disorder or autoimmune cytopenia.
* Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other antiinflammatory medication (except nonsteroidal anti-inflammatory drugs).
* Epilepsy or known seizure disorder (now or previously).

Primary outcomes:
measure: Safety of C2C_ASC (Adverse Events)
timeFrame: 12 weeks
Secondary outcomes:
measure: Changes in autism spectrum disorder
timeFrame: 12 weeks
measure: Changes in gastrointestinal symptoms
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Aalborg
country: Denmark
facility: Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark
Study officials:
affiliation: Cell2Cure ApS
name: Jens Kastrup
role: STUDY_DIRECTOR
References: