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Anemia, AplasticPhase 2NCT00737685

Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)

A Phase 2 study testing cyclophosphamide, fludarabine , thymoglobulin in people with Anemia, Aplastic, run by The Korean Society of Pediatric Hematology Oncology at 1 site in South Korea.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and rejection of organ transplants. Read more about this study

Who is it for?
Ages Up to 25 years
Conditions the study is for
Anemia, Aplastic
What is being tested?
cyclophosphamide, fludarabine , thymoglobulin
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteSouth Korea
Study dates
Jan 2006to Dec 2012
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 25 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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%
Show all 19Show fewer
  • 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.
  • Appropriate donor is available: Matched in 6/6 of A, B, DR loci.
  • Patients (or one of parents if patients age < 19) should sign informed consent.

Who cannot join

  • 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.
  • Manipulated bone marrow.

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • Seoul National University Hospital

    Seoul, 110-744

Other trials in South Korea

About this study

Official title: Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia

Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.

Read the detailed description

GVHD prophylaxis recommendation tacrolimus (0.03 mg/kg/day i.v. by continuous infusion from day -2 and taper with an oral form until 1 year after BMT/PBSCT) methotrexate (15 mg/m2 i.v. on days 1 and 10 mg/m2 i.v. on days 3, 6, 11)

Being tested
cyclophosphamide, fludarabine , thymoglobulin
Keywords
Pediatric, UBMT, Severe
Medical subject headings
Anemia, Aplastic, Lymphoma, Follicular

What participants receive

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

  • cyclophosphamide, fludarabine , thymoglobulinDrug

    cyclophosphamide (50 mg/kg once daily i.v. on days -9, -8, -7 \& -6) fludarabine (30 mg/m2 once daily i.v. on days -5, -4, -3 \& -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -3, -2 \& -1)

Study groups in NCT00737685
Study groupRoleWhat they receive
FludarabineExperimentalNot described
Receives: Drug: cyclophosphamide, fludarabine , thymoglobulin

What is being measured

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

  1. To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.

    Measured: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.

  2. To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.

    Measured: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years

Secondary outcomes (1)

  1. To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning

    Measured: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.

Timeline

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

  1. First posted19 Aug 2008 (estimated)
  2. Study startJan 2006
  3. Primary completionAug 2012 (estimated)
  4. Study completionDec 2012 (estimated)

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00737685
Sponsor's protocol ID
KSPHO-SCT0401
Status last verified
Mar 2012
First submitted
18 Aug 2008
Last updated
26 Mar 2012
Results posted
No
Data monitoring committee
No

Publications

  • Kang HJ, Shin HY, Park JE, Chung NG, Cho B, Kim HK, Kim SY, Lee YH, Lim YT, Yoo KH, Sung KW, Koo HH, Im HJ, Seo JJ, Park SK, Ahn HS; Korean Society of Pediatric Hematology-Oncology. Successful engraftment with fludarabine, cyclophosphamide, and thymoglobulin conditioning regimen in unrelated transplantation for severe aplastic anemia: A phase II prospective multicenter study. Biol Blood Marrow Transplant. 2010 Nov;16(11):1582-8. doi: 10.1016/j.bbmt.2010.05.010. Epub 2010 May 26.

    PubMed 20685256

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing cyclophosphamide, fludarabine , thymoglobulin in people with Anemia, Aplastic, run by The Korean Society of Pediatric Hematology Oncology 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?

The sponsor lists 19 inclusion and 5 exclusion criteria. 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: status not confirmed, as of the sponsor's last update on 26 Mar 2012. 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?

Results are not available in the source record.

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?

To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA. (time frame: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.). The study lists 2 primary outcomes in all. 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 NCT00737685, which also lists 1 linked publication.

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 26 Mar 2012.

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