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ASC - Autism Study

Primary Purpose

Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome

Status
Recruiting
Phase
Phase 1
Locations
Denmark
Study Type
Interventional
Intervention
Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)
Sponsored by
Cell2Cure ApS
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Gastrointestinal microbiota, Gut-brain axis, Immunomodulation

Eligibility Criteria

6 Years - 14 Years (Child)All SexesDoes not accept 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).

Sites / Locations

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

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

1 x 1000000 C2C_ASCs/kg body weight

2 x 1000000 C2C_ASCs/kg body weight

Arm Description

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

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

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

Changes in autism spectrum disorder
Difference from baseline to follow-up within the group treated with C2C_ASC: Changes in autism spectrum disorder score test (questionnaire). [Raw scores converting to standard scores and then generates five major domain composite scores: communication, daily living skills, socialization, adaptive behavior composite and motor. For these domains a standard score of 100 is the mean with a standard deviation of 15. This means that a score of 100 is similar to the typical population of the same age. Scores in the range of 86 and above are considered adequate. Scores in the range of 85 and below indicates a significant skill deficit when comparing with the typical population of the same age. High scores: standard scores of 130-140 Moderately high scores: standard scores of 115-129 Adequate scores: standard scores of 86-114 Moderately low scores: standard scores of 71-85 Low scores: standard scores of 20-70]
Changes in gastrointestinal symptoms
Difference from baseline to follow-up within the group treated with C2C_ASC: Changes in The Infant Gastrointestinal Symptom Questionnaire (questionnaire). [A 0-100 scale where higher scores indicate better irritable bowel syndrome specific quality of life.]

Full Information

First Posted
September 23, 2022
Last Updated
June 12, 2023
Sponsor
Cell2Cure ApS
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1. Study Identification

Unique Protocol Identification Number
NCT05602116
Brief Title
ASC - Autism Study
Official Title
Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 9, 2023 (Actual)
Primary Completion Date
February 1, 2026 (Anticipated)
Study Completion Date
February 1, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cell2Cure ApS

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome, Mesenchymal Stromal Cells, Inflammatory Bowel Diseases
Keywords
Gastrointestinal microbiota, Gut-brain axis, Immunomodulation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Model Description
Open dose titrating with two groups in extension of each other (thus not categorically parallel).
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
1 x 1000000 C2C_ASCs/kg body weight
Arm Type
Experimental
Arm Description
Trial participant 1 - 5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
Arm Title
2 x 1000000 C2C_ASCs/kg body weight
Arm Type
Experimental
Arm Description
Trial participant 6 - 10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight
Intervention Type
Drug
Intervention Name(s)
Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)
Intervention Description
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.
Primary Outcome Measure Information:
Title
Safety of C2C_ASC (Adverse Events)
Description
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).
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Changes in autism spectrum disorder
Description
Difference from baseline to follow-up within the group treated with C2C_ASC: Changes in autism spectrum disorder score test (questionnaire). [Raw scores converting to standard scores and then generates five major domain composite scores: communication, daily living skills, socialization, adaptive behavior composite and motor. For these domains a standard score of 100 is the mean with a standard deviation of 15. This means that a score of 100 is similar to the typical population of the same age. Scores in the range of 86 and above are considered adequate. Scores in the range of 85 and below indicates a significant skill deficit when comparing with the typical population of the same age. High scores: standard scores of 130-140 Moderately high scores: standard scores of 115-129 Adequate scores: standard scores of 86-114 Moderately low scores: standard scores of 71-85 Low scores: standard scores of 20-70]
Time Frame
12 weeks
Title
Changes in gastrointestinal symptoms
Description
Difference from baseline to follow-up within the group treated with C2C_ASC: Changes in The Infant Gastrointestinal Symptom Questionnaire (questionnaire). [A 0-100 scale where higher scores indicate better irritable bowel syndrome specific quality of life.]
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jens Kastrup
Phone
+45 21202994
Email
jk@cell2cure.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jens Kastrup
Organizational Affiliation
Cell2Cure ApS
Official's Role
Study Director
Facility Information:
Facility Name
Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark
City
Aalborg
Country
Denmark
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marlene Briciet Lauritsen, MD, DMSc
Email
marlene.lauritsen@rn.dk

12. IPD Sharing Statement

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ASC - Autism Study

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