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Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia

NCT02407470 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2015-04-03

No results posted yet for this study

Summary

RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in the alternative donor (matched unrelated, partially matched family member) transplantation. Although an overall and disease free survival of 85% to 100%, can be obtained in allogeneic blood or bone marrow stem cell transplantation using an human leukocyte antigen (HLA) matched sibling donor, only about 25% of patients have such a donor.

PURPOSE: In an attempt to avoid GVHD, reduce earlier infection rate and decrease regimen-related toxicity while maintaining better engraftment, this study is to evaluate the effectiveness and safety of patient's own adipose-derived mesenchymal stem cell (AD-MSC) or AD-MSC transdifferentiated HSC (AD-HSC) transplant after an immunosuppressive regimen in treating patients who have severe aplastic anemia.

The patient will be in the study for one year for observation and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely. The principle measures of safety and efficacy will be :

1. Patient survival probability at 3 months, 6 months and 1 year.
2. Engraftment at 3 months, 6 months and 1 year
3. Incidence of graft versus host disease (GVHD), incidence of acute and chronic GVHD and Incidence of earlier infection rate as well as other complications within 6 months and 1 years.

Conditions

  • Severe Aplastic Anemia

Interventions

DRUG

Rabbit antithymoglobulin (ATG)

Rabbit ATG at 3.5 mg/kg/dose IV is given from day -6 to -2.

PROCEDURE

Adipose derived mesenchymal stem cells ( AD-MSCs)

Participants will receive rabbit ATG at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-MSCs at a dose of 3000000 cells/kg/d on day 1-3.

PROCEDURE

AD-MSC transdifferentiated HSCs (AD-HSCs)

Participants will receive rabbit anti-thymocyte globulin at 3.5 mg/kg/dose IV from day -6 to -2, and then patient's own AD-HSCs at a dose of 3000000 cells/kg/d from day 1 to 4.

Sponsors & Collaborators

  • Peking Union Medical College Hospital

    collaborator OTHER

  • General Hospital of Beijing PLA Military Region

    collaborator OTHER

  • Chinese Academy of Medical Sciences

    collaborator OTHER

  • Navy General Hospital, Beijing

    lead OTHER

Principal Investigators

  • james Q Yin, M.D.,Ph.D. · The military general hospital of Beijing

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
14 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-31
Primary Completion
2017-01-31
Completion
2017-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02407470 on ClinicalTrials.gov