NCT01297972

Brief Summary

Mesenchymal stem cells have been tested in many autoimmune disorders with encouraging results and may be an alternative to the treatment of immune-mediated severe acquired aplastic anemia.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2011

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

February 16, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 17, 2011

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2013

Completed
Last Updated

February 4, 2014

Status Verified

February 1, 2014

Enrollment Period

2.8 years

First QC Date

February 16, 2011

Last Update Submit

February 2, 2014

Conditions

Aplastic Anemia

Keywords

Aplastic AnemiaMesenchymal Stem Cells

Outcome Measures

Primary Outcomes (1)

  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability of intravenous allogeneic unrelated mesenchymal stem cells infusion in patients with severe acquired aplastic anemia.

    Adverse events like allergic reactions,infectious diseases,organ dysfunction or other related to mesenchymal stem cells infusion will be assessed.

    After the mesenchymal stem cells infusion up to 6 mounth after

Secondary Outcomes (3)

  • Level of cytopenias

    weekly until 6 months

  • Transfusional requirements

    weekly until 6 months

  • Incidence of infections and febrile neutropenia

    weekly until 6 months

Interventions

Intravenous bone marrow mesenchymal stem cells infusionBIOLOGICAL

After standard immunosuppressive therapy with rabbit antithymocyte globulin 3,5 mg/Kg/day during 5 days, allogeneic unrelated bone marrow derived mesenchymal stem cells will be infused intravenously. Oral cyclosporine 5 mg/Kg/day (with dose correction weekly to keep serum cyclosporine level between 150-250 mg/dl) up to 6 months will be added.

Eligibility Criteria

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

You may qualify if:

  • Diagnosis of Acquired Aplastic Anemia
  • Relapse/refractory to at least 1 immunosuppressive first line treatment
  • Not eligible to allogeneic bone marrow transplantation

You may not qualify if:

  • Previous or current malignancy
  • Active or latent infectious disease
  • Positive serologic tests for HIV, HCV, HBV, HTLV-1 and 2, Syphilis or Chagas disease
  • Previous drug reaction for antithymocyte globulin, cyclosporin or corticosteroids
  • Severe organic impairment (renal, hepatic, cardiac, pulmonary)
  • Uncontrolled hypertension or diabetes
  • Pregnancy
  • Previous history of allergic reaction to penicillin or streptomycin
  • Severe psychiatric disorder

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo

Ribeirão Preto, São Paulo, 14048900, Brazil

Location

Related Publications (3)

  • Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006 Oct 15;108(8):2509-19. doi: 10.1182/blood-2006-03-010777. Epub 2006 Jun 15.

    PMID: 16778145BACKGROUND

  • Fouillard L, Bensidhoum M, Bories D, Bonte H, Lopez M, Moseley AM, Smith A, Lesage S, Beaujean F, Thierry D, Gourmelon P, Najman A, Gorin NC. Engraftment of allogeneic mesenchymal stem cells in the bone marrow of a patient with severe idiopathic aplastic anemia improves stroma. Leukemia. 2003 Feb;17(2):474-6. doi: 10.1038/sj.leu.2402786. No abstract available.

    PMID: 12592355BACKGROUND

  • Cle DV, Santana-Lemos B, Tellechea MF, Prata KL, Orellana MD, Covas DT, Calado RT. Intravenous infusion of allogeneic mesenchymal stromal cells in refractory or relapsed aplastic anemia. Cytotherapy. 2015 Dec;17(12):1696-705. doi: 10.1016/j.jcyt.2015.09.006.

    PMID: 26589752DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Diego V Clé, MD

    University of Sao Paulo

    PRINCIPAL INVESTIGATOR

  • Rodrigo T Calado, MD

    University of Sao Paulo

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

February 16, 2011

First Posted

February 17, 2011

Study Start

February 1, 2011

Primary Completion

November 1, 2013

Study Completion

November 1, 2013

Last Updated

February 4, 2014

Record last verified: 2014-02

Locations

BR(1)