Cord Blood Transplantation in Severe Aplastic Anemia (APCORD)

June 13, 2017 updated by: Assistance Publique - Hôpitaux de Paris

A Prospective Phase II Study to Evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse After Immunosuppressive Therapy

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;

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Anticipated)

26

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75010
        • Saint Louis

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 55 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Overall survival
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
overall survival
Time Frame: 5 years
5 years
adverse events
Time Frame: 1 year
1 year
Graft versus host disease
Time Frame: 2 years
2 years
Infectious episodes
Time Frame: 1 year
1 year
treatment related mortality
Time Frame: 100 days
100 days
karnofsky scale
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Principal Investigator: Regis Peffault de la Tour, MD, PhD, Assistance Publique - Hôpitaux de Paris

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2011

Primary Completion (Actual)

June 1, 2017

Study Completion (Actual)

June 1, 2017

Study Registration Dates

First Submitted

April 27, 2011

First Submitted That Met QC Criteria

April 27, 2011

First Posted (Estimate)

April 28, 2011

Study Record Updates

Last Update Posted (Actual)

June 15, 2017

Last Update Submitted That Met QC Criteria

June 13, 2017

Last Verified

June 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P090201

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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