Aplastic AnemiaPhase 2NCT00882323
Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)
A Phase 2 study testing Cyclophosphamide, Fludarabine, Thymoglobulin in people with Aplastic Anemia, run by The Korean Society of Pediatric Hematology Oncology at 1 site in South Korea.
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 graft versus host disease (GVHD) and rejection of organ transplants. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Aplastic Anemia
- 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
- 33 participants
- Where
- 1 siteSouth Korea
- Study dates
- Nov 2008to Oct 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 1 year to 21 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%
- Bone marrow
Show all 18Show fewer
- 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% and ejection fraction > 45%.
- Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper
- 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, 1 recruiting
- Recruiting
Seoul National University Hospital
Seoul, 110-744
Hyo Seop Ahn, M.D, Ph.D, 82 2 2072 3625, hsahn@snu.ac.kr
About this study
Official title: Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor 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 graft versus host disease (GVHD) and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for transplantation from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in unrelated donor transplantation. Our previous phase II study of fludarabine, cyclophosphamide plus thymoglobulin conditioning resulted in good engraftment (100%) and survival rate (74%). But grade III/IV toxicities occurred in 25% of patients and all events were treatment related mortalities. As cyclophosphamide is more toxic agent than fludarabine, we plan a new phase II study re; 'reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated donor transplantation in severe aplastic anemia' by reducing dosage of cyclophosphamide and increasing dosage of fludarabine.
- Conditions
- Aplastic Anemia
- Being tested
- Cyclophosphamide, Fludarabine, Thymoglobulin
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphamide, Fludarabine, ThymoglobulinDrug
cyclophosphamide (60 mg/kg once daily i.v. on days -8, -7)
fludarabine (40 mg/m2 once daily i.v. on days -6, -5, -4, -3, -2)
thymoglobulin (2.5 mg/kg once daily i.v. on days -4, -3, -2)
| Study group | Role | What they receive |
|---|---|---|
| Fludarabine | Experimental | Not described Receives: Drug: Cyclophosphamide, Fludarabine, Thymoglobulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia.
Measured: From Nov. 2008 to Oct. 2012
Secondary outcomes (3)
To evaluate toxicities of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for UBMT/PBSCT in SAA.
Measured: From Nov. 2008 to Oct. 2012
To evaluate overall and EFS rate after UBMT/PBSCT.
Measured: From Nov. 2008 to Oct. 2012
To evaluate GVHD and immunologic recovery after UBMT/PBSCT.
Measured: From Nov. 2008 to Oct. 2012
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2009 (estimated)
- Study startNov 2008
- Primary completionOct 2012 (estimated)
- Study completionOct 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Korean Society of Pediatric Hematology Oncology
- Sponsor type
- Research network
- Investigators
- Hyo seop Ahn, M.D, Ph. D, Principal investigator, The Korean Society of Pediatric Hematology Oncology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00882323
- Sponsor's protocol ID
- KSPHO-SCT 0802
- Status last verified
- Mar 2012
- First submitted
- 15 Apr 2009
- Last updated
- 26 Mar 2012
- Results posted
- No
- Data monitoring committee
- Yes
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
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00882323
- Acronym / sponsor ID
- KSPHO-SCT 0802
- Conditions
- Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- The Korean Society of Pediatric Hematology OncologyResearch network
- Study start
- Nov 2008
- Primary completion
- Oct 2012 (estimated)
- Study completion
- Oct 2012 (estimated)
- First posted
- 16 Apr 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 graft versus host disease (GVHD) and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for transplantation from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in unrelated donor transplantation. Our previous phase II study of fludarabine, cyclophosphamide plus thymoglobulin conditioning resulted in good engraftment (100%) and survival rate (74%). But grade III/IV toxicities occurred in 25% of patients and all events were treatment related mortalities. As cyclophosphamide is more toxic agent than fludarabine, we plan a new phase II study re; 'reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated donor transplantation in severe aplastic anemia' by reducing dosage of cyclophosphamide and increasing dosage of fludarabine.
Study design
- Primary purpose
- Treatment
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fludarabine | Experimental | Drug: Cyclophosphamide, Fludarabine, Thymoglobulin |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
- 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:
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cyclophosphamide, Fludarabine, Thymoglobulincyclophosphamide (60 mg/kg once daily i.v. on days -8, -7) fludarabine (40 mg/m2 once daily i.v. on days -6, -5, -4, -3, -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -4, -3, -2) | Drug | Fludarabine |
Primary outcome
To evaluate engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia.
Time frame: From Nov. 2008 to Oct. 2012
3 secondary outcomes
To evaluate toxicities of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for UBMT/PBSCT in SAA.
Time frame: From Nov. 2008 to Oct. 2012
To evaluate overall and EFS rate after UBMT/PBSCT.
Time frame: From Nov. 2008 to Oct. 2012
To evaluate GVHD and immunologic recovery after UBMT/PBSCT.
Time frame: From Nov. 2008 to Oct. 2012
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2012.
- Derived from this study: PubMed 20685256Kang 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hyo seop Ahn, M.D, Ph. D principal investigator
The Korean Society of Pediatric Hematology Oncology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Fludarabine, Thymoglobulin in people with Aplastic Anemia, 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 18 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?
Contact Hyoung Jin Kang, M.D, Ph.D (82 2 2072 3304, kanghj@snu.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia. (time frame: From Nov. 2008 to Oct. 2012). 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 NCT00882323, 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; AzTrials last refreshed its copy on 7 Nov 2026.