Aplastic AnemiaPhase 1/2NCT00881933
Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)
A Phase 1/2 study testing Cyclophosphosphamide, TBI in people with Aplastic Anemia, run by The Korean Society of Pediatric Hematology Oncology at 1 site in South Korea.
Trial summary
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. 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?
- Cyclophosphosphamide, TBI
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSouth Korea
- Study dates
- Nov 2008to Sep 2012
- Phase
- Phase 1/2
- 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 19Show 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%, 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.
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.
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: Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia
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.
- Conditions
- Aplastic Anemia
- Being tested
- Cyclophosphosphamide, TBI
- Keywords
- Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,, cord blood transplantation, pediatric
- Medical subject headings
- Anemia, Aplastic, Brain Injuries, Traumatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphosphamide, TBIOther
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)
| Study group | Role | What they receive |
|---|---|---|
| Fludarabine | Experimental | Not described Receives: Other: Cyclophosphosphamide, TBI |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.
Measured: From Oct 2008. to Sep 2011
Secondary outcomes (4)
To evaluate the incidence and severity of toxicity and treatment related mortality
Measured: From Oct 2008. to Sep 2011
To evaluate overall and event free survival rate
Measured: From Oct 2008. to Sep 2011
To evaluate acute and chronic GVHD
Measured: From Oct 2008. to Sep 2011
To evaluate immunologic recovery after CBT
Measured: From Oct 2008. to Sep 2011
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2009 (estimated)
- Study startNov 2008
- Primary completionSep 2012 (estimated)
- Study completionSep 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1/2
- Primary purpose
- Treatment
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 5 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
- NCT00881933
- Sponsor's protocol ID
- KSPHO-SCT0804
- Status last verified
- Mar 2012
- First submitted
- 14 Apr 2009
- Last updated
- 26 Mar 2012
- Results posted
- No
- Data monitoring committee
- Yes
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
- Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00881933
- Acronym / sponsor ID
- KSPHO-SCT0804
- Conditions
- Aplastic Anemia
- Keywords
- Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,, cord blood transplantation, pediatric
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- The Korean Society of Pediatric Hematology OncologyResearch network
- Study start
- Nov 2008
- Primary completion
- Sep 2012 (estimated)
- Study completion
- Sep 2012 (estimated)
- First posted
- 15 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.
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.
Study design
- Primary purpose
- Treatment
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fludarabine | Experimental | Other: Cyclophosphosphamide, TBI |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
- Peripheral blood
- Bone marrow
- No prior hematopoietic stem cell transplantation.
- Age: no limits.
Key exclusion criteria (4 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.
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 |
|---|---|---|
| Cyclophosphosphamide, TBIfludarabine (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) | Other | Fludarabine |
Primary outcome
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
4 secondary outcomes
To evaluate the incidence and severity of toxicity and treatment related mortality
Time frame: From Oct 2008. to Sep 2011
To evaluate overall and event free survival rate
Time frame: From Oct 2008. to Sep 2011
To evaluate acute and chronic GVHD
Time frame: From Oct 2008. to Sep 2011
To evaluate immunologic recovery after CBT
Time frame: From Oct 2008. to Sep 2011
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.
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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 1/2 study testing Cyclophosphosphamide, TBI 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 19 inclusion and 4 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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.