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Cord Blood Transplantation in Severe Aplastic Anemia (APCORD)

This study is currently recruiting participants.
Verified November 2012 by Assistance Publique - Hôpitaux de Paris
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01343953
First received: April 27, 2011
Last updated: November 19, 2012
Last verified: November 2012
History of Changes
  Purpose

This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;


Condition Intervention Phase
Hereditary Diseases
Acquired Aplastic Anemia
Relapse
Absence of an HLA Identical Donor
 Procedure: Cord Blood Transplantation
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Prospective Phase II Study to Evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse After Immunosuppressive Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Assistance Publique - Hôpitaux de Paris:

Primary Outcome Measures:
  • Overall survival [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Graft versus host disease [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Infectious episodes [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • treatment related mortality [ Time Frame: 100 days ] [ Designated as safety issue: Yes ]
  • overall survival [ Time Frame: 5 years ] [ Designated as safety issue: No ]
  • adverse events [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
  • karnofsky scale [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 26
Study Start Date: May 2011
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cord Blood Transplantation Procedure: Cord Blood Transplantation
One or 2 cord blood units containing alone or both together more than 4x107 frozen nucleated cells/Kg with no more than 2 out of 6 HLA mismatches between them and with the patients for acquired Severe Aplastic Anemia
Other Name: Cord Blood Transplantation

Detailed Description:

This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an Human Leucocyte Antigen (HLA) identical donor; 26 patients will be recruited, according to a one step Fleming design

  Eligibility

Ages Eligible for Study:   3 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age: 3-55 years old
  • inherited aplastic anemia (with severe aplastic anemia criteria) or acquired aplastic anemia in relapse or treatment failure after immunosuppressive therapy without clonal evolution
  • Karnovsky Index >= 60%
  • Informed consent

Exclusion Criteria:

  • HLA identical donor
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01343953

Contacts
Contact: Regis Peffault de la Tour, MD, PhD 33(1)42385073 regis.peffaultdelatour@sls.aphp.fr

Locations
France
saint Louis Not yet recruiting
Paris, France, 75010
Contact: Regis Peffault de la tour, MD, PhD     33(1)42385073     regis.peffaultdelatour@sls.aphp.fr    
Principal Investigator: Regis Peffault de la tour, MD, PhD            
saint Louis Recruiting
Paris, France, 75010
Contact: R Peffault de la Tour, MD     33(1)42494949        
Principal Investigator: Regis Peffault de la Tour, MD            
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Regis Peffault de la Tour, MD, PhD Assistance Publique - Hôpitaux de Paris
  More Information

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01343953     History of Changes
Other Study ID Numbers: P090201
Study First Received: April 27, 2011
Last Updated: November 19, 2012
Health Authority: France: Ministry of Health

Keywords provided by Assistance Publique - Hôpitaux de Paris:
severe aplastic anemia
cord blood transplantation

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Genetic Diseases, Inborn
Recurrence
 Hematologic Diseases
Bone Marrow Diseases
Disease Attributes
Pathologic Processes

ClinicalTrials.gov processed this record on May 19, 2013