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

• ClinicalTrials.gov Identifier: NCT03123458
• Recruitment Status: Unknown
• First Posted: April 21, 2017
• Last Update Posted: February 26, 2018
• Sponsor: Shenzhen Geno-Immune Medical Institute
• Information provided by (Responsible Party): Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute

Tracking Information

• First Submitted Date: March 13, 2017
• First Posted Date: April 21, 2017
• Last Update Posted Date: February 26, 2018
• Actual Study Start Date: January 1, 2016
• Estimated Primary Completion Date: December 31, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 20, 2017)

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: up to one month ]

Safety of cfMSC infusion acute and prolonged

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 10, 2017)

Number of participants with reduced symptoms or stabilized conditions after treatment [ Time Frame: 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.

Original Secondary Outcome Measures (submitted: April 20, 2017)

Number of participants with reduced symptoms or stabilized conditions after treatment assessed by doctors [ Time Frame: 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.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Immune Related Disorder
• Tissue Damage

• Intervention: Biological: clonal fetal MSCs

Study Arms

Experimental: Single arm, cfMSC to treat immune disorders

cfMSCs treatment

Intervention: Biological: clonal fetal MSCs

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: April 20, 2017): 100
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 31, 2020
• Estimated Primary Completion Date: December 31, 2019 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 1 Year to 80 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: China

Administrative Information

• NCT Number: NCT03123458
• Other Study ID Numbers: GIMI-IRB-17001
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute
• Original Responsible Party: Same as current
• Current Study Sponsor: Shenzhen Geno-Immune Medical Institute
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Shenzhen Geno-Immune Medical Institute
• Verification Date: February 2018