ASC - Autism Study

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 6 Years - 14 Years
Gender All
More Inclusion & Exclusion 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).

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05602116
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Cell2Cure ApS
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Not yet recruiting
Countries
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome, Mesenchymal Stromal Cells, Inflammatory Bowel Diseases
Additional Details

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.

Arms & Interventions

Arms

Experimental: 1 x 1000000 C2C_ASCs/kg body weight

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

Experimental: 2 x 1000000 C2C_ASCs/kg body weight

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

Interventions

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.

Contact Information

This trial has no sites locations listed at this time. If you are interested in learning more, you can contact the trial's primary contact:

Jens Kastrup

jk@cell2cure.com

+45 21202994

For additional contact information, you can also visit the trial on clinicaltrials.gov.