Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)
This study is currently recruiting participants.
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology
Sponsor:
The Korean Society of Pediatric Hematology Oncology
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
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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
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 Anemia, Aplastic 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 May 19, 2013