NCT01343953

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;

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
26

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2011

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 27, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 28, 2011

Completed
3 days until next milestone

Study Start

First participant enrolled

May 1, 2011

Completed
6.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2017

Completed
Last Updated

June 15, 2017

Status Verified

June 1, 2017

Enrollment Period

6.1 years

First QC Date

April 27, 2011

Last Update Submit

June 13, 2017

Conditions

Hereditary DiseasesAcquired Aplastic AnemiaRelapseAbsence of an HLA Identical Donor

Keywords

severe aplastic anemiacord blood transplantation

Outcome Measures

Primary Outcomes (1)

  • Overall survival

    1 year

Secondary Outcomes (6)

  • Graft versus host disease

    2 years

  • Infectious episodes

    1 year

  • treatment related mortality

    100 days

  • overall survival

    5 years

  • adverse events

    1 year

  • +1 more secondary outcomes

Study Arms (1)

Cord Blood Transplantation

EXPERIMENTAL
Procedure: Cord Blood Transplantation

Interventions

Cord Blood TransplantationPROCEDURE

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

Cord Blood Transplantation

Eligibility Criteria

Age3 Years - 55 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • HLA identical donor

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

saint Louis

Paris, 75010, France

Location

Related Publications (1)

  • 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.

    PMID: 29760162DERIVED

MeSH Terms

Conditions

Genetic Diseases, InbornRecurrenceAnemia, Aplastic

Interventions

Cord Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsAnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Stem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Regis Peffault de la Tour, MD, PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 27, 2011

First Posted

April 28, 2011

Study Start

May 1, 2011

Primary Completion

June 1, 2017

Study Completion

June 1, 2017

Last Updated

June 15, 2017

Record last verified: 2017-06

Locations

FR(1)