Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia

Evaluation of Safety and Efficacy in Patients With Aplastic Anemia by Transplantation of Umbilical Cord Derived Mesenchymal Stem Cells

This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.

Study Overview

• Status: Unknown
• Conditions: Aplastic Anemia
• Intervention / Treatment: Biological: mesenchymal stem cells

Detailed Description

Aplastic anemia (AA) is a disorder thought to be caused by an immune-mediated bone marrow failure. Not all people with AA are eligible for today's standard treatments. One new treatment approach uses umbilical cord derived mesenchymal stem cells-specialized cells capable of developing into other types of cells-to provide the basis for clinical application. The aim of the present study is to investigate the safety and efficacy of vein infusion of allogeneic mesenchymal stem cells in patients with AA .

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 1

Contacts and Locations

Study Locations

• China
  • Hebei
    • Shijiazhuang, Hebei, China, 050031
      • Recruiting
      • The First Hospital of Hebei Medical University
      • Contact: Jun Zhang, Master; Phone Number: 86-311-85917384; Email: june_luck@126.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 56 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Diagnosis of AA according to established criteria in 2010
2. Age from 14 to 60 years
3. Suffering from AA within six months
4. No serious infection or acute hemorrhage.
5. Left ventricular ejection fraction (LVEF) ≥ 50%
6. No acute infectious diseases.
7. Understanding and willingness to sign a written informed consent document.
8. Eastern Cooperative Oncology Group(ECOG) score of 0-2pionts.

Exclusion Criteria:

- Patients with AA have to be disqualified from this study if any of the following is applicable.

1. Severe aplastic anemia(SAA) with severe infection.
2. Severe aplastic anemia(SAA) with active hemorrhage.
3. Severe heart attack, liver and kidney disease following serious complications
4. Patients with allergic constitution.
5. Pregnancy and lactation.
6. Accompanied by malignant tumors and other clonal disease.
7. Patients with active tuberculosis, acute sever hepatitis or infectious period of diseases.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: mesenchymal stem cells; According to the inclusion and exclusion criteria, selected patients were divided into a cell therapy group and a control group. Umbilical cord derived mesenchymal stem cells at a dose of 100-300 million by intravenous infusion.	Biological: mesenchymal stem cells; Patients in the cell treated group were given umbilical cord derived mesenchymal stem cells by intravenous infusion besides conventional therapy. There was only conventional treatment in the control group. All patients after treatment for 1months, 3months, 6months and 9months were evaluated respectively the therapeutic effect.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemoglobin levels in peripheral blood	The recovery of bone marrow erythrocyte is evaluated by hemoglobin levels (g/L) in peripheral blood.	Post cell transplantation: 9months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of red blood cell in peripheral blood	The improvement of bone marrow erythrocyte is evaluated by red blood cell count (×10^12/L) in peripheral blood.	Post cell transplantation: 1, 3, 6, 9months
The number of granulocyte in peripheral blood	The recovery of bone marrow granulocyte is evaluated by granulocyte count (×10^9/L) in peripheral blood.	Post cell transplantation: 1, 3, 6, 9months
The number of white blood cell in peripheral blood	The improvement of bone marrow granulocyte is evaluated by white blood cell count (×10^9/L) in peripheral blood.	Post cell transplantation: 1, 3, 6, 9months
The evaluation of bone marrow megakaryocytic series	The status of megakaryocytic series is evaluated by platelet count (×10^9/L) in peripheral blood.	Post cell transplantation: 1, 3, 6, 9months
adverse reaction	Adverse reaction includes temperature changes(℃), the change of blood pressure(mmHg) and allergic reaction.	Post cell transplantation: 1, 3, 6, 9months

Collaborators and Investigators

• Sponsor: Hebei Medical University
• Investigators: Study Director: Quanhai Li, Doctor, The First Hospital of Hebei Medical University; Principal Investigator: Xianyun Wang, Doctor, The First Hospital of Hebei Medical University; Principal Investigator: Fan Zhang, Bachelor, The First Hospital of Hebei Medical University; Principal Investigator: Xiaohui Jia, Doctor, The First Hospital of Hebei Medical University; Principal Investigator: Yang Shen, Master, The First Hospital of Hebei Medical University; Principal Investigator: Bing Ma, Master, The First Hospital of Hebei Medical University; Principal Investigator: Wanyi Yin, Master, The First Hospital of Hebei Medical University; Principal Investigator: Dan Zhao, Master, The First Hospital of Hebei Medical University; Principal Investigator: Bojian Sun, Master, The First Hospital of Hebei Medical University; Study Director: Qingchi Liu, Doctor, The First Hospital of Hebei Medical University

Study record dates

Study Major Dates

• Study Start: January 1, 2017
• Primary Completion (Anticipated): December 1, 2018
• Study Completion (Anticipated): December 1, 2019

Study Registration Dates

• First Submitted: February 4, 2017
• First Submitted That Met QC Criteria: February 13, 2017
• First Posted (Actual): February 16, 2017

Study Record Updates

• Last Update Posted (Actual): April 7, 2017
• Last Update Submitted That Met QC Criteria: April 6, 2017
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: 17277787D-AA

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No