Clinical trial
Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
Name
ASC II
Description
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.
Trial arms
Trial start
2023-06-09
Estimated PCD
2026-02-01
Trial end
2026-02-01
Status
Recruiting
Phase
Early phase I
Treatment
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.
Arms:
1 x 1000000 C2C_ASCs/kg body weight, 2 x 1000000 C2C_ASCs/kg body weight
Size
10
Primary endpoint
Safety of C2C_ASC (Adverse Events)
12 weeks
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).
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Open dose titrating with two groups in extension of each other (thus not categorically parallel).', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 10, 'type': 'ESTIMATED'}}
Updated at
2023-06-15
1 organization
1 product
5 indications
Organization
Cell2CureIndication
Autism Spectrum DisorderIndication
Gastrointestinal DysfunctionIndication
Leaky Gut SyndromeIndication
Mesenchymal Stromal CellsIndication
Inflammatory Bowel Disease