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

Phase 1

• Conditions: Immune Related Disorder; Tissue Damage
• Interventions: Biological: clonal fetal MSCs

• Registration Number: NCT03123458
• Lead Sponsor: Shenzhen Geno-Immune Medical Institute

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.

Study Timeline

• Study Start: 2016-01-01
• Study First Submitted: 2017-03-13
• Study First Submitted QC: 2017-04-20
• Study First Posted: 2017-04-21
• Status Verified: 2018-02
• Last Update Submitted: 2018-02-23
• Last Update Posted: 2018-02-26
• Primary Completion: 2019-12-31
• Study Completion: 2020-12-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 100

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single arm, cfMSC to treat immune disorders	clonal fetal MSCs	cfMSCs treatment

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (3)

Shenzhen Geno-immune Medical Institute; 🇨🇳 Shenzhen, Guangdong, China

Capital Institute of Pediatrics affiliated Children's hospital; 🇨🇳 Beijing, Beijing, China

Aerospace Center Hospital; 🇨🇳 Beijing, China