Clinical Trial Results:
Effect of adipose tissue derived mesenchymal stromal cells on autism and
leaky gut syndrome. A phase I pilot study.
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Summary
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EudraCT number |
2022-002940-27 |
Trial protocol |
DK |
Global end of trial date |
20 Sep 2024
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Results information
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Results version number |
v1(current) |
This version publication date |
09 Jul 2025
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First version publication date |
09 Jul 2025
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
C2C1-1
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05602116 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Cell2Cure ApS
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Sponsor organisation address |
Kajerødgård 9, Birkerød, Denmark, 3460
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Public contact |
Jens Kastrup, Cell2Cure ApS, 45 21202994, jk@cell2cure.com
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Scientific contact |
Jens Kastrup, Cell2Cure ApS, 45 21202994, jk@cell2cure.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
22 Apr 2025
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
20 Sep 2024
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Global end of trial reached? |
Yes
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Global end of trial date |
20 Sep 2024
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The purpose of this dose titrating study was to evaluate the safety and efficacy of an allogeneic adipose tissue-derived mesenchymal stromal/stem cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms. Results from this study will be used to support the feasibility and safety of C2C_ASC treatment in children. And further to support the hypothesis of a connection between gastrointestinal symptoms, increased local gastrointestinal and systemic elevated immunological and inflammatory activity, bacterial toxins in the blood and symptoms of autism spectrum disorder that can be reduced or normalized by modulating the immunological activity and inflammation by treatment with mesenchymal stromal/stem cells.
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Protection of trial subjects |
Treatment visits was planned at least one week apart, to observe for serious adverse events related to the cell treatment and procedure, before treatment of a new trial subject.
Data monitoring was done for all enrolled trial subjects.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
09 Jun 2023
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Denmark: 10
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Worldwide total number of subjects |
10
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EEA total number of subjects |
10
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
3
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Adolescents (12-17 years) |
7
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
The trial subjects was recruited through the Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark. A total number of 10 subjects was planned and enrolled. All 10 subjects received the study intervention and completed the study follow-up. | |||||||||
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Pre-assignment
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Screening details |
Date of enrolment (first participant’s first visit): 05 September 2023 Date of completion (last participant’s last visit): 20 September 2024 Key inclusion criteria were: • Children aged 6 – 14 years • Diagnosis of autism spectrum disorder (ASD) • Gastrointestinal symptoms or previous gastrointestinal symptoms | |||||||||
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Period 1
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Period 1 title |
Baseline (overall period)
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Is this the baseline period? |
Yes | |||||||||
Allocation method |
Non-randomised - controlled
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Blinding used |
Not blinded | |||||||||
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Arms
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Are arms mutually exclusive |
Yes
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Arm title
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1 x 1 million ASCs/kg body weight | |||||||||
Arm description |
Trial subject 1-5 was allocated to this arm with one treatment of 1 x 1 million ASCs/kg body weight. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
CSCC_ASC5010
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Investigational medicinal product code |
CSCC_ASC5010
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Other name |
Allogeneic adipose tissue-derived mesenchymal stromal/stem cells
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Pharmaceutical forms |
Infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
The investigational product (IP) was an advanced therapy investigational medicinal product (ATIMP) manufactured from allogeneic adipose tissue-derived mesenchymal stromal/stem cells (ASCs). The active substance is in vitro expanded ASCs. The IP, CSCC_ASC5010, was manufactured as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial.
In chronological order subjects was randomized to a low dose or a high dose where the first 5 enrolled subjects received 1 x 1 million ASCs/kg body weight (low dose), and the last 5 enrolled subjects received 2 x
1 million ASCs/kg body weight (high dose). The IP dose was calculated from the subject’s body weight. The calculated dose was extracted from one or more vials and was diluted in 50 ml isotonic saline and infused in a vein in the hand.
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Arm title
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2 x 1 million ASCs/kg body weight | |||||||||
Arm description |
Trial subject 6-10 was allocated to this arm with one treatment of 2 x 1 million ASCs/kg body weight. | |||||||||
Arm type |
Experimental | |||||||||
Investigational medicinal product name |
CSCC_ASC5010
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Investigational medicinal product code |
CSCC_ASC5010
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Other name |
Allogeneic adipose tissue-derived mesenchymal stromal/stem cells
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Pharmaceutical forms |
Infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
The investigational product (IP) was an advanced therapy investigational medicinal product (ATIMP) manufactured from allogeneic adipose tissue-derived mesenchymal stromal/stem cells (ASCs). The active substance is in vitro expanded ASCs. The IP, CSCC_ASC5010, was manufactured as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial.
In chronological order subjects was randomized to a low dose or a high dose where the first 5 enrolled subjects received 1 x 1 million ASCs/kg body weight (low dose), and the last 5 enrolled subjects received 2 x
1 million ASCs/kg body weight (high dose). The IP dose was calculated from the subject’s body weight. The calculated dose was extracted from one or more vials and was diluted in 50 ml isotonic saline and infused in a vein in the hand.
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Baseline characteristics reporting groups
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Reporting group title |
Baseline
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Reporting group description |
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Subject analysis sets
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Subject analysis set title |
Baseline data
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Subject analysis set type |
Intention-to-treat | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Age
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End points reporting groups
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Reporting group title |
1 x 1 million ASCs/kg body weight
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Reporting group description |
Trial subject 1-5 was allocated to this arm with one treatment of 1 x 1 million ASCs/kg body weight. | ||
Reporting group title |
2 x 1 million ASCs/kg body weight
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Reporting group description |
Trial subject 6-10 was allocated to this arm with one treatment of 2 x 1 million ASCs/kg body weight. | ||
Subject analysis set title |
Baseline data
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Subject analysis set type |
Intention-to-treat | ||
Subject analysis set description |
Age
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End point title |
Safety [1] | |||||||||||||||||||||
End point description |
To assess the safety and tolerability of intravenous administration of a single dose 1 x 1 million ASCs per kg body weight and 2 x 1 million ASCs per kg body weight in children with autism spectrum disorder and gastrointestinal symptoms. Endpoint defined by adverse events (AEs), serious adverse events (SARs), and suspected unexpected serious adverse events (SUSARs).
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End point type |
Primary
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End point timeframe |
From treatment to the 12-week follow-up.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The primary end point is safety registred as the number of safety events, thus no statistical analyses are needed. |
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| No statistical analyses for this end point | ||||||||||||||||||||||
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End point title |
Efficacy - changes in symptoms of autism spectrum disorder | ||||||||||||||||||
End point description |
To characterize changes in symptoms of autism spectrum disorder. Assessed by questionnaires:
• Children’s Communication Checklist (second edition, Pearson Clinical)
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End point type |
Secondary
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End point timeframe |
From baseline to the 12-week follow-up.
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Efficacy - changes in gastrointestinal symptoms | |||||||||||||||
End point description |
To characterize changes in gastrointestinal symptoms. Assessed by questionnaire:
• IBS-QOL (irritable bowel syndromequality of life) (ePROVIDE)
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End point type |
Secondary
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End point timeframe |
From baseline to the 12-week follow-up.
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| No statistical analyses for this end point | ||||||||||||||||
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End point title |
Efficacy - changes in inflammatory markers | |||||||||||||||
End point description |
To characterize changes in inflammatory markers. Assessed by plasma analysis of:
• CRP
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End point type |
Secondary
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End point timeframe |
From baseline to the 12-week follow-up.
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| No statistical analyses for this end point | ||||||||||||||||
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End point title |
Efficacy - changes in gastrointestinal symptoms | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Reported gastrointestinal symptoms.
Measurement: history of painful and hard stools.
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| No statistical analyses for this end point | |||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From treatment to the 12-week follow-up.
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Assessment type |
Systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
18
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Reporting groups
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Reporting group title |
1 x 1 million ASCs/kg body weight
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Reporting group description |
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Reporting group title |
2 x 1 million ASCs/kg body weight
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Reporting group description |
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| Frequency threshold for reporting non-serious adverse events: 1% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
