Clonal Fetal Mesenchymal Stem Cells (cfMSCs) for the Control of Immune-related Disorders

Shenzhen Geno-Immune Medical Institute

Status and phase: Invitation-only; Phase 1

Conditions: Tissue Damage; Immune Related Disorder

Treatments: Biological: clonal fetal MSCs

Study type: Interventional

Funder types: Other

Identifiers

NCT03123458

GIMI-IRB-17001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 1 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Single arm, cfMSC to treat immune disorders

Experimental group

Description:

cfMSCs treatment

Treatment:

Biological: clonal fetal MSCs