Clinical trial NCT03123458: Clonal Fetal Mesenchymal Stem Cells (cfMSCs) for the Control of Immune-related Disorders
Official title: Clonal Fetal Mesenchymal Stem Cells (cfMSCs) for the Control of Immune-related Disorders
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Immune Related Disorder
Tissue Damage
Keywords: MSC
GVHDs
transplantation
Lead sponsor: Shenzhen Geno-Immune Medical Institute
Sponsor class: OTHER
First posted: 2017-04-21
Enrollment: 100
Sex: ALL
Minimum age (years): 1
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objectives are to evaluate the safety and efficacy of infusion of the third party fully-characterized clonally derived fetal MSCs (cfMSCs) for the control of severe symptoms associated with acute and chronic immune-related disorders and tissue damage.

Detailed description: MSCs have been extensively studied and clinically evaluated for the treatment of autoimmune diseases and graft versus host disease (GVHD) after hematopoietic stem cell transplantation (HSCT). The variable source of MSCs and the lack of consistency of primary tissue-derived MSCs are major obstacles to reliable translational applications of such therapeutic cell products. Fetal tissue-derived clonal MSCs (cfMSCs) have extended expansion potential and express rich levels of various growth factors, and thus can achieve quality consistency. Careful evaluation of fMSCs in clinical studies has not been conducted. Autoimmune diseases involve aberrant immune responses that harm tissues and organs. GVHD is a serious and often fatal problem associated with HSCT. MSCs have immunomodulatory and immunosuppressive effects. In many studies, MSCs have demonstrated promising beneficial effects that reduce severe autoimmune reactions, diminish symptoms of chronic GvHD and therapy-resistant acute GvHD including steroid-resistant GVHD. The safety and therapeutic effects of phenotype and functionally characterized fMSCs still require extensive clinical evaluation. This study aims to assess the safety and the potential beneficial effects of infusion of various dosages of third party fMSCs for the control of severe symptoms associated with acute and chronic immune-related disorders.

Eligibility criteria: Inclusion Criteria:

1. Informed consent.
2. No available alternative treatment that can reduce the symptoms
3. Patients are required to meet the following inclusion criteria:

   * Any patient that has clinically documented abnormal immune or age-related disorders including acute and chronic GVHD. Patients may receive best available treatment for the control of disease symptoms.
   * Patients with symptoms associated with genetic defects or infectious diseases are not eligible.

Exclusion Criteria:

1. Inability to give informed consent.
2. Patients with ongoing infection or history of cancer.
3. Patients with poor clinical conditions with the life expectancy of less than 14 days.
4. Pregnancy.

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: up to one month
Secondary outcomes:
measure: Number of participants with reduced symptoms or stabilized conditions after treatment
timeFrame: after 1 month from fMSC infusion
Other outcomes:
Locations:
city: Beijing
country: China
facility: Capital Institute of Pediatrics affiliated Children's hospital
state: Beijing
city: Shenzhen
country: China
facility: Shenzhen Geno-immune Medical Institute
state: Guangdong
city: Beijing
country: China
facility: Aerospace Center Hospital
Study officials:
References: