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

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.

Study Overview

• Status: Unknown
• Conditions: Tissue Damage; Immune Related Disorder
• Intervention / Treatment: Biological: clonal fetal MSCs

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.

• Study Type: Interventional
• Enrollment (Anticipated): 100
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100049
    • Aerospace Center Hospital
  • Beijing
    • Beijing, Beijing, China, 100020
      • Capital Institute of Pediatrics affiliated Children's hospital
  • Guangdong
    • Shenzhen, Guangdong, China, 518000
      • Shenzhen Geno-Immune Medical Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 80 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Single arm, cfMSC to treat immune disorders; cfMSCs treatment	Biological: clonal fetal MSCs

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	Safety of cfMSC infusion acute and prolonged	up to one month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with reduced symptoms or stabilized conditions after treatment	Short term clinical effects measured by physiological and serological parameters related to the disease condition and symptoms using prepared study assessment table.	after 1 month from fMSC infusion

Collaborators and Investigators

• Sponsor: Shenzhen Geno-Immune Medical Institute

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 1, 2016
• Primary Completion (ANTICIPATED): December 31, 2019
• Study Completion (ANTICIPATED): December 31, 2020

Study Registration Dates

• First Submitted: March 13, 2017
• First Submitted That Met QC Criteria: April 20, 2017
• First Posted (ACTUAL): April 21, 2017

Study Record Updates

• Last Update Posted (ACTUAL): February 26, 2018
• Last Update Submitted That Met QC Criteria: February 23, 2018
• Last Verified: February 1, 2018

More Information

Terms related to this study

• Keywords: transplantation; MSC; GVHDs
• Additional Relevant MeSH Terms: Pathologic Processes; Disease
• Other Study ID Numbers: GIMI-IRB-17001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No