Clinical trial NCT04850469: Study of MSC-Exo on the Therapy for Intensively Ill Children
Official title: Study of Exosomes Derived From Mesenchymal Stem Cells on the Therapy for Children With Severe Infection
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Sepsis
Critical Illness
Keywords: Sepsis
Critical Illness
Mesenchymal Stem Cell Derived Exosomes
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2021-04-20
Enrollment: 0
Sex: ALL
Minimum age (years): 29
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All children hospitalized in PICU of Children's Hospital of Fudan University from January 2022 to December 2023

Brief summary: At present, a number of projects related to MSCs have been approved for graft-versus-host disease, myocardial infarction, Crohn's disease and other diseases, indicating a strong therapeutic potential of MSCs. However, the efficacy of MSC-Exo for severely infected children is not fully evaluated. In our study, patients who met the inclusion criteria will be divided into trial group and control group. Clinical and demographic data, as well as treatment outcome will be collected from the electronic health record. This study will evaluate the application and therapeutic effect of MSC-Exo in severely infected children, and determine the Optimal dosage and infusion.

Detailed description: Mesenchymal stem cells (MSCs) are pluripotent stem cells with high self-renewal ability and multidirectional differentiation potential. studies revealed that local or systemic administration of mesenchymal stem cell-derivrd exosomes (MSC-Exo) efficiently suppressed detrimental immune response in inflamed tissues and promoted survival and regeneration of injured parenchymal cells. European Union countries have written MSCs into treatment guidelines for refractory graft-versus-host disease.

In China, the technology for isolation, in vitro culture and expansion of MSCs is already mature. With further research on the therapeutic efficacy of MSC-Exo and the development of clinical trials, it is expected to become a treatment for severe illness.

A predictable infection plan is going to be widely used in clinics to provide individualized treatment for patients, improve the prognosis of patients and improve the quality of life.

The investigators intend to enroll all children who were hospitalized in pediatric intensive care unit PICU of Children's Hospital of Fudan University from January 2022 to December 2023. Children with a PICU length of day less than 48h will be excluded.

Eligibility criteria: Inclusion Criteria:

* children hospitalized in PICU of Children's Hospital of Fudan University

Exclusion Criteria:

* discharge within 48 hours
* patients without informed consent
* incomplete clinical and demographic data

Primary outcomes:
measure: The death rate of children
timeFrame: within 28 days after they discharged from PICU
measure: Marshall Multiple Organ Dysfunction Score
timeFrame: Within 3 to 15 days after the start of treatment
measure: the APPS score
timeFrame: Within 3 to 15 days after the start of treatment
Secondary outcomes:
measure: length of stay in PICU
timeFrame: up to 28 days
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Guoping Lu, Doctor
role: PRINCIPAL_INVESTIGATOR
References: