NCT05602116

ASC - Autism Study

Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Cell to Cure ApS · Industry
• Sex: All
• Age: 6 Years – 14 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Autism Spectrum Disorder
• Gastrointestinal Dysfunction
• Leaky Gut Syndrome
• Mesenchymal Stromal Cells
• Inflammatory Bowel Diseases

Interventions

Type	Name	Description
DRUG	Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)	The Investigational Product is a C2C\_ASC cell product (Cell2Cure®, Cell2Cure Aps, Denmark). C2C\_ASC is an advanced therapy investigational medicinal product (ATIMP) manufactured from abdominal adipose tissue derived mesenchymal stromal cells (ASC) from healthy donors. C2C\_ASC is aseptically procured and manufactured according to tissue law and GMP by Cell2Cure ApS or Cardiology Stem Cell Center, Rigshospitalet, Copenhagen, Denmark, using manual isolation of cells from abdominal fat tissue, xeno-free cell expansion in automated closed bioreactor systems and cryopreservation of the final product. The active substance is the in vitro expanded ASCs. The final product, C2C\_ASC, is provided as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial. The excipient is CryoStor10 (Biolife Solutions), holding 10% DMSO.

Timeline

• Start date: 2023-06-09
• Primary completion: 2024-06-28
• Completion: 2024-09-20
• First posted: 2022-11-01
• Last updated: 2024-09-26

Locations

1 site across 1 country: Denmark