Cord Blood Transplantation in Severe Aplastic Anemia

Phase 2

Completed

• Conditions: Absence of an HLA Identical Donor; Acquired Aplastic Anemia; Hereditary Diseases; Relapse
• Interventions: Procedure: Cord Blood Transplantation

• Registration Number: NCT01343953
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief 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;

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 Timeline

• Study First Submitted: 2011-04-27
• Study First Submitted QC: 2011-04-27
• Study First Posted: 2011-04-28
• Study Start: 2011-05
• Status Verified: 2017-06
• Primary Completion: 2017-06
• Study Completion: 2017-06
• Last Update Submitted: 2017-06-13
• Last Update Posted: 2017-06-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cord Blood Transplantation	Cord Blood Transplantation	-

Primary Outcome Measures

Name	Time	Method
Overall survival	1 year

Secondary Outcome Measures

Name	Time	Method
Infectious episodes	1 year
treatment related mortality	100 days
overall survival	5 years
adverse events	1 year
karnofsky scale	1 year
Graft versus host disease	2 years

Trial Locations

Locations (1)

saint Louis; 🇫🇷 Paris, France