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Cord Blood Transplantation in Severe Aplastic Anemia (APCORD)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01343953
First received: April 27, 2011
Last updated: June 23, 2016
Last verified: June 2016
  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: 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:


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 2017
Estimated Primary Completion Date: November 2016 (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

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   (Child, Adult)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01343953

Locations
France
saint Louis
Paris, France, 75010
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

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: June 23, 2016
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
Recurrence
Anemia, Aplastic
Genetic Diseases, Inborn
Hematologic Diseases
Disease Attributes
Pathologic Processes
Bone Marrow Diseases

ClinicalTrials.gov processed this record on September 27, 2016