ASC - Autism Study

Cell2Cure

Status and phase: Completed; Phase 1

Conditions: Mesenchymal Stromal Cells; Autism Spectrum Disorder; Gastrointestinal Dysfunction; Leaky Gut Syndrome; Inflammatory Bowel Diseases

Treatments: Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)

Study type: Interventional

Funder types: Industry

Identifiers

NCT05602116

ASC II

Details and patient eligibility

About

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.

Full 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.

Enrollment: 10 patients

Sex: All

Ages: 6 to 14 years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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10 participants in 2 patient groups

1 x 1000000 C2C_ASCs/kg body weight

Experimental group

Description:

Trial participant 1 - 5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight

Treatment:

Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)

2 x 1000000 C2C_ASCs/kg body weight

Experimental group

Description:

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

Treatment:

Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)