An open label safety and efficacy study of allogenic adipose tissue-derived mesenchymal stromal cells on autism and gastrointestinal symptoms

The primary objective of this study is to assess the safety of intravenous infusion of in vitro expanded adipose tissue-derived mesenchymal stromal cells (ASCs) used for allogenic treatment of children with autism spectrum disorder (ASD) and gastrointestinal symptoms. This objective addresses the clinical need to establish the safety profile of this advanced therapy medicinal product in a pediatric population presenting with both neurodevelopmental and gastrointestinal manifestations, which frequently co-occur in ASD patients.

The secondary objectives evaluate:

• Behavioral outcomes in autism spectrum disorder through standardized behavioral testing

• Changes in gastrointestinal symptoms, which represent a common comorbidity affecting quality of life in this patient population

The sponsor did not provide information regarding the total number of participants enrolled in this clinical trial. The study population consisted of children and adolescents aged **6 to 18 years** (both inclusive), including both **male** and **female** subjects. Participants were required to have a confirmed diagnosis of **autism spectrum disorder** and to be experiencing ongoing **gastrointestinal symptoms** at the time of enrollment. The trial population was selected based on these specific medical conditions, representing a pediatric cohort with concurrent neurodevelopmental and digestive system manifestations. No information was provided regarding the general health status, lifestyle considerations, or additional selection criteria for this study population.

This clinical trial investigates the safety and efficacy of **allogenic adipose-tissue-derived mesenchymal stromal cells** administered via **intravenous infusion** in pediatric participants diagnosed with **autism spectrum disorder** and concurrent **gastrointestinal symptoms**. The study employs an **open-label** design, meaning both participants and investigators are aware of the treatment being administered. Two investigational products, C2C_ASC110 and C2C_ASC66, are utilized as test interventions, both classified as **advanced therapy medicinal products** containing **mesenchymal stem cells** derived from adipose tissue of allogeneic origin. The trial is designated as a **Phase Ib clinical trial** with an estimated recruitment start date in January 2026 and an anticipated completion date in January 2029, resulting in an overall trial duration of approximately three years.

The primary objective is to assess the safety profile of the investigational cell therapy by monitoring the number of **adverse reactions** and **serious adverse reactions** occurring during the study period. Secondary objectives include evaluating changes in adaptive behavior, communication skills, and quality of life related to gastrointestinal symptoms through validated assessment tools. These include the **Vineland Adaptive Behavior Scales**, the **Children Communication Checklist-2**, and the **IBS-QoL** (Irritable Bowel Syndrome Quality of Life) questionnaire. These endpoints will provide comprehensive data on both the tolerability and potential therapeutic benefits of the intervention.

Eligible participants must meet specific inclusion criteria, including an age range of 6 to 18 years inclusive, a confirmed diagnosis of autism spectrum disorder, and the presence of ongoing gastrointestinal symptoms. The study visit schedule will include an initial **screening visit** to determine eligibility and establish baseline assessments, followed by treatment administration visits and subsequent **follow-up visits** to monitor safety parameters and assess efficacy outcomes through questionnaire-based evaluations. An **end-of-study visit** will be conducted to complete final assessments and document the participant's overall response to treatment. The expected length of participant involvement will extend throughout the duration of the treatment protocol and follow-up period as defined in the study design.

Conditions that may lead to **early termination** from the study include the occurrence of unacceptable adverse events, withdrawal of consent by the participant or legal guardian, protocol violations, loss to follow-up, or any other circumstances that may compromise participant safety or data integrity as determined by the investigator. All safety data will be systematically collected and evaluated throughout the trial to ensure appropriate monitoring of the investigational cell therapy products.

The experimental treatment consists of two investigational **advanced therapy medicinal products** containing **allogenic adipose-tissue-derived mesenchymal stem cells** as the active substance. Both products are classified as **cell therapy** products utilizing structurally diverse substances derived from allogeneic sources. The mesenchymal stem cells are obtained from adipose tissue and expanded in vitro prior to administration. The products are also known by the synonym **CellReady**.

The first investigational product, designated as **C2C_ASC110**, is formulated for **intravenous infusion** and contains allogenic adipose-tissue-derived mesenchymal stem cells. The product is manufactured by CELL2CURE APS and is administered via the intravenous route. Specific dosage amounts, frequency of administration, and treatment duration are not provided in the available clinical trial documentation.

The second investigational product, designated as **C2C_ASC66**, is also formulated for intravenous infusion and contains the same active substance of allogenic adipose-tissue-derived mesenchymal stem cells. This product is manufactured by CELL TO CURE APS and is similarly administered via intravenous infusion. As with C2C_ASC110, detailed information regarding specific dosage, administration frequency, and treatment period is not specified in the source documentation.

Both investigational products are classified as test products within the trial design and represent advanced therapy medicinal products specifically categorized as mesenchymal stem cell therapies. Neither product is formulated as a **paediatric formulation**, nor are they designated as **orphan drugs**. The products are characterized as somatic cell therapy products with cells of allogeneic origin. Information regarding **placebo**, **comparator treatments**, standard-of-care therapies, dosing schedules, maximum daily or total dose amounts, and **participant compliance monitoring** procedures is not available in the provided clinical trial data.

Efficacy will be assessed through the evaluation of secondary endpoints measuring changes in adaptive behavior, communication skills, and quality of life related to gastrointestinal symptoms. The Vineland Adaptive Behavior Scales will be utilized as a questionnaire-based tool to evaluate changes in adaptive functioning. The Children Communication Checklist-2 will be employed as a questionnaire to assess changes in communication abilities. Additionally, the IBS-QoL (Irritable Bowel Syndrome Quality of Life) questionnaire will be used to measure changes in quality of life associated with gastrointestinal symptoms. These validated instruments will provide quantitative measures of treatment efficacy in children with autism spectrum disorder and gastrointestinal symptoms receiving allogenic adipose tissue-derived mesenchymal stromal cells.