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LeukemiaPhase 2NCT00731328

Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome

A Phase 2 study testing nonmyeloablative allogeneic hematopoietic stem cell transplantation in people with Leukemia and Myelodysplastic Syndromes, run by Asan Medical Center at 1 site in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. Read more about this study

Who is it for?
Ages Up to 75 years
What is being tested?
nonmyeloablative allogeneic hematopoietic stem cell transplantation
How many people
15 participants
Where
1 siteSouth Korea
Study dates
Apr 2008to Dec 2013
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 75 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

DISEASE CHARACTERISTICS:

  • Diagnosis of any of the following bone marrow failure syndromes:
  • Severe aplastic anemia, meeting 1 of the following criteria:
  • Not responsive to immunosuppressive therapy
  • With recurrent cytopenia after immunosuppressive therapy or allogeneic hematopoietic cell transplantation
  • Low-risk myelodysplastic syndrome, including any of the following:
  • Refractory anemia
  • Refractory anemia with ringed sideroblasts
  • Refractory cytopenia with multi-lineage dysplasia
  • Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
  • With thrombotic episodes
  • With severe cytopenia
  • No willing, suitable HLA-compatible donor in family or in donor registries
  • Related donor with HLA-haploidentical mismatch at three or less of 6 loci
  • Patients with very severe neutropenia (< 200/μL) or febrile episodes, who feel urgent need for allogeneic hematopoietic cell transplantation, are eligible without a search for HLA-matched unrelated donors

PATIENT CHARACTERISTICS:

  • Karnofsky performance status 70-100%
  • Bilirubin < 2.0 mg/dL
  • AST < 3 times upper limit of normal
  • Creatinine < 2.0 mg/dL
  • Ejection fraction > 40% by MUGA scan

PRIOR CONCURRENT THERAPY:

  • See Disease Characteristics

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • Asan Medical Center - University of Ulsan College of Medicine

    Seoul, 138-736

Other trials in South Korea

About this study

Official title: HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY

RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect).

PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.

Read the detailed description

OBJECTIVES:

  • To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell transplantation after reduced-intensity conditioning regimen comprising busulfan, fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure syndromes.

OUTLINE:

  • Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7 and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30 minutes starting 30 minutes before ATG on days -4 to -1.
  • HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.
  • Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4 hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the absence of GVHD, cyclosporine is tapered starting between days 30 to 60.

After completion of study treatment, patients are followed periodically for 1 year.

Being tested
nonmyeloablative allogeneic hematopoietic stem cell transplantation
Keywords
refractory anemia, refractory anemia with ringed sideroblasts, refractory cytopenia with multilineage dysplasia, childhood myelodysplastic syndromes, de novo myelodysplastic syndromes, previously treated myelodysplastic syndromes, secondary myelodysplastic syndromes, aplastic anemia, paroxysmal nocturnal hemoglobinuria
Medical subject headings
Leukemia, Myelodysplastic Syndromes, Neoplasms, Hemoglobinuria, Paroxysmal, Anemia, Refractory, Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • nonmyeloablative allogeneic hematopoietic stem cell transplantationProcedure or surgery

    infusion of mobilized donor hematopoietic progenitor cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Donor cell engraftment

    neutrophil count over 500/ul

    Measured: 10-35 days after transplantation

Secondary outcomes (4)

  1. Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria

    various toxicities of treatment

    Measured: 0-60 months after transplantation

  2. Acute and chronic GVHD

    ocurrence of acute or chronic GVHD after transplantation

    Measured: 15-100 days; 100 days to 4 years

  3. overall survival

    patients surviving after transplantaion

    Measured: 0-60 months

  4. event-free survival

    patients undergoing transplantation and maintaining donor hematopoiesis

    Measured: 0-60 months after transplatation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Aug 2008 (estimated)
  2. Study startApr 2008
  3. Primary completionDec 2013
  4. Study completionDec 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00731328
Sponsor's protocol ID
CDR0000600351
Other IDs
AMC-UUCM-2008-0038
Status last verified
Dec 2015
First submitted
8 Aug 2008
Last updated
31 Dec 2015
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing nonmyeloablative allogeneic hematopoietic stem cell transplantation in people with Leukemia and Myelodysplastic Syndromes, run by Asan Medical Center at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Donor cell engraftment (time frame: 10-35 days after transplantation). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00731328.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2015.

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