NCT01182662

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for the therapy of severe aplastic anemia (SAA).

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Aug 2010

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2010

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

August 11, 2010

Completed
6 days until next milestone

First Posted

Study publicly available on registry

August 17, 2010

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2013

Completed
Last Updated

August 31, 2010

Status Verified

August 1, 2010

Enrollment Period

3 years

First QC Date

August 11, 2010

Last Update Submit

August 30, 2010

Conditions

Keywords

Bone Marrow DiseaseAplastic AnemiaUmbilical Cord/placenta-Derived MSCTransplantation

Outcome Measures

Primary Outcomes (3)

  • SAA clinical symptoms

    Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.

    1 year

  • The number of blood cells

    The number of blood cells, which contains WBC, Neu, RBC, Hb,PLT and reticulocyte, will be mainly tested monthly after transplantion of MSCs for one year

    1 year

  • Bone borrow hemocytology

    Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.

    1 year

Secondary Outcomes (1)

  • Percentage of systemic T regulatory cell population and T lymphocyte subsets

    1 year

Study Arms (2)

Human umbilical cord-derived MSCs and cyclosporin

EXPERIMENTAL

Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle and CsA 5mg/kg po for 12 months

Other: Human umbilical cord-derived MSCs and cyclosporin A

cyclosporine A

ACTIVE COMPARATOR

cyclosporine A at a dose of 5 mg CsA/kg

Other: cyclosporin A

Interventions

1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months

Human umbilical cord-derived MSCs and cyclosporin

cyclosporin A 5mg/kg po for 12 months

cyclosporine A

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patient age 18\~80 years old with plan to infuse MSCs.
  • Standard of diagnosis of aplastic anemia is according to Chinese domestic classification of AA for 1987.
  • Patients must have an ECOG 0\~2.
  • No moderate or sever organ dysfunction: Ejection fraction\>45%; Creatinine \<176 umol/L.
  • No active severe viral or fungus infection.
  • Each patient must sign written informed consent.

You may not qualify if:

  • Psychiatric condition that would limit informed consent.
  • HIV positive
  • Positive Pregnancy Test
  • Patient has enrolled another clinical trial study within last 4 weeks.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Department of Hematology of the 2nd Hospital of Shandong University

Jinan, Shandong, 250033, China

RECRUITING

MeSH Terms

Conditions

Anemia, AplasticBone Marrow Diseases

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure Disorders

Intervention Hierarchy (Ancestors)

CyclosporinsPeptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • chengyun zheng, Ph. D

    Department of Hematology of The 2nd Hospital of Shandong University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

chengyun zheng, Ph. D

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER

Study Record Dates

First Submitted

August 11, 2010

First Posted

August 17, 2010

Study Start

August 1, 2010

Primary Completion

August 1, 2013

Study Completion

August 1, 2013

Last Updated

August 31, 2010

Record last verified: 2010-08

Locations