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Cord Blood Transplantation in Severe Aplastic Anemia (APCORD)

Primary Purpose

Hereditary Diseases, Acquired Aplastic Anemia, Relapse

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Cord Blood Transplantation
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hereditary Diseases focused on measuring severe aplastic anemia, cord blood transplantation

Eligibility Criteria

3 Years - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • saint Louis

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cord Blood Transplantation

Arm Description

Outcomes

Primary Outcome Measures

Overall survival

Secondary Outcome Measures

Graft versus host disease
Infectious episodes
treatment related mortality
overall survival
adverse events
karnofsky scale

Full Information

First Posted
April 27, 2011
Last Updated
June 13, 2017
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT01343953
Brief Title
Cord Blood Transplantation in Severe Aplastic Anemia
Acronym
APCORD
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2017
Overall Recruitment Status
Completed
Study Start Date
May 2011 (undefined)
Primary Completion Date
June 2017 (Actual)
Study Completion Date
June 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Diseases, Acquired Aplastic Anemia, Relapse, Absence of an HLA Identical Donor
Keywords
severe aplastic anemia, cord blood transplantation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
26 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cord Blood Transplantation
Arm Type
Experimental
Intervention Type
Procedure
Intervention Name(s)
Cord Blood Transplantation
Intervention Description
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 Outcome Measure Information:
Title
Overall survival
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Graft versus host disease
Time Frame
2 years
Title
Infectious episodes
Time Frame
1 year
Title
treatment related mortality
Time Frame
100 days
Title
overall survival
Time Frame
5 years
Title
adverse events
Time Frame
1 year
Title
karnofsky scale
Time Frame
1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Regis Peffault de la Tour, MD, PhD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
saint Louis
City
Paris
ZIP/Postal Code
75010
Country
France

12. IPD Sharing Statement

Citations:
PubMed Identifier
29760162
Citation
Peffault de Latour R, Chevret S, Jubert C, Sirvent A, Galambrun C, Ruggeri A, Gandemer V, Cornillon J, Rialland F, Dalle JH, Forcade E, Bruno B, Paillard C, Rorlich PS, Salmon A, Furst S, Sicre de Fontbrune F, Rubio MT, Bay JO, Mohty M, Larghero J, Gluckman E, Socie G; Francophone Society of Bone Marrow Transplantation and Cellular Therapy. Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study. Blood. 2018 Aug 16;132(7):750-754. doi: 10.1182/blood-2018-01-829630. Epub 2018 May 14.
Results Reference
derived

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Cord Blood Transplantation in Severe Aplastic Anemia

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