Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
The Korean Society of Pediatric Hematology Oncology
ClinicalTrials.gov Identifier:
NCT00881933
First received: April 14, 2009
Last updated: March 23, 2012
Last verified: March 2012
  Purpose

Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.


Condition Intervention Phase
Aplastic Anemia
Other: Cyclophosphosphamide, TBI
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia

Resource links provided by NLM:


Further study details as provided by The Korean Society of Pediatric Hematology Oncology:

Primary Outcome Measures:
  • To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia. [ Time Frame: From Oct 2008. to Sep 2011 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To evaluate the incidence and severity of toxicity and treatment related mortality [ Time Frame: From Oct 2008. to Sep 2011 ] [ Designated as safety issue: No ]
  • To evaluate overall and event free survival rate [ Time Frame: From Oct 2008. to Sep 2011 ] [ Designated as safety issue: No ]
  • To evaluate acute and chronic GVHD [ Time Frame: From Oct 2008. to Sep 2011 ] [ Designated as safety issue: No ]
  • To evaluate immunologic recovery after CBT [ Time Frame: From Oct 2008. to Sep 2011 ] [ Designated as safety issue: No ]

Estimated Enrollment: 5
Study Start Date: November 2008
Estimated Study Completion Date: September 2012
Estimated Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fludarabine Other: Cyclophosphosphamide, TBI
fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1)

  Eligibility

Ages Eligible for Study:   1 Year to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
  • Peripheral blood

    • Neutrophils < 0.5 x 109/l
    • Platelets < 20 x 109/l
    • Corrected reticulocytes < 1%
  • Bone marrow

    • Severe hypocellularity (< 25%)
    • Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells
  • No prior hematopoietic stem cell transplantation.
  • Age: no limits.
  • Performance status: ECOG 0-2.
  • Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

    • Heart: a shortening fraction > 30%, ejection fraction > 45%.
    • Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.
    • Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.
  • Patients must lack any active viral infections or active fungal infection.
  • No appropriate donor
  • Appropriate cord blood is available: matched at least in 4/6 of A, B, DR loci.
  • Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion Criteria:

  • Pregnant or nursing women.
  • Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
  • Psychiatric disorder that would preclude compliance.
  • Congenital aplastic anemia including Fanconi anemia.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00881933

Contacts
Contact: Hyoung Jin Kang, M.D, Ph.D 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Ji Won Lee, M.D 82 2 2072 0177 agnesjw@hanmail.net

Locations
Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Korea, Republic of, 110-744
Contact: Hyo Seop Ahn, M.D, Ph.D    82 2 2072 3625    hsahn@snu.ac.kr   
Sponsors and Collaborators
The Korean Society of Pediatric Hematology Oncology
Investigators
Principal Investigator: Hyo Seop Ahn, M.D, Ph.D The Korean Society of Pediatric Hematology Oncology
  More Information

No publications provided

Responsible Party: The Korean Society of Hematology, The Korean Society of Pediatric Hematology Oncology
ClinicalTrials.gov Identifier: NCT00881933     History of Changes
Other Study ID Numbers: KSPHO-SCT0804
Study First Received: April 14, 2009
Last Updated: March 23, 2012
Health Authority: Korea: Food and Drug Administration

Keywords provided by The Korean Society of Pediatric Hematology Oncology:
Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,
cord blood transplantation, pediatric

Additional relevant MeSH terms:
Anemia, Aplastic
Anemia
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Fludarabine monophosphate
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antimetabolites, Antineoplastic
Antimetabolites

ClinicalTrials.gov processed this record on July 28, 2014