ASC - Autism Study

NCT05602116 | Completed Phase 1

• 1 other identifier: ASC II
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Gastrointestinal microbiota; Gut-brain axis; Immunomodulation

Outcome Measures

Primary Outcomes (1)

• Safety of C2C_ASC (Adverse Events)

  Safety of C2C\_ASC within the 12 weeks follow-up period: Adverse event (AE), serious adverse event (SAE), suspected unexpected serious adverse reaction (SUSAR) and development safety update report (DSUR).

  12 weeks

Secondary Outcomes (2)

• Changes in autism spectrum disorder

  12 weeks

• Changes in gastrointestinal symptoms

  12 weeks

Study Arms (2)

1 x 1000000 C2C_ASCs/kg body weight

EXPERIMENTAL

Trial participant 1 - 5 will be treated with 1 x 1000000 C2C\_ASCs/kg body weight

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

2 x 1000000 C2C_ASCs/kg body weight

EXPERIMENTAL

Trial participant 6 - 10 will be treated with 2 x 1000000 C2C\_ASCs/kg body weight

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

Interventions

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

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.

1 x 1000000 C2C_ASCs/kg body weight; 2 x 1000000 C2C_ASCs/kg body weight

Eligibility Criteria

• Age: 6 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Cell to Cure ApS: lead

Study Sites (1)

Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark

Aalborg, Denmark

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Inflammatory Bowel Diseases

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Jens Kastrup

  Cell to Cure ApS

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open dose titrating with two groups in extension of each other (thus not categorically parallel).

Study Record Dates

• First Submitted: September 23, 2022
• First Posted: November 1, 2022
• Study Start: June 9, 2023
• Primary Completion: June 28, 2024
• Study Completion: September 20, 2024
• Last Updated: September 26, 2024

Record last verified: 2024-09

Locations

DK (1)