Overview

This trial is active, not recruiting.

Conditions hereditary diseases, acquired aplastic anemia, relapse, absence of an hla identical donor
Treatment cord blood transplantation
Phase phase 2
Sponsor Assistance Publique - Hôpitaux de Paris
Start date May 2011
End date November 2016
Trial size 26 participants
Trial identifier NCT01343953, P090201

Summary

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;

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
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

Primary Outcomes

Measure
Overall survival
time frame: 1 year

Secondary Outcomes

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

Eligibility Criteria

Male or female participants from 3 years up to 55 years old.

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

Additional Information

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
Principal investigator Regis Peffault de la Tour, MD, PhD
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
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Assistance Publique - Hôpitaux de Paris.