Acute LeukemiaPhase 1/2NCT01509300
HLA-haploidentical Hematopoietic Stem Cell Transplantation for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors
A Phase 1/2 study testing anti-thymocyte globulin, filgrastim and Total body irradiation in people with Acute Leukemia and Myelodysplastic Syndrome, run by Asan Medical Center at 1 site in South Korea.
Trial summary
RATIONALE: Conditioning with total body irradiation (TBI) and fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Acute Leukemia, Myelodysplastic Syndrome, Solid Tumors
- What is being tested?
- anti-thymocyte globulin, filgrastim and Total body irradiation
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jan 2012to Mar 2014
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Disease characteristics
- Acute lymphoblastic leukemia (first remission, high risk; beyond first remission; refractory)
- Acute myeloblastic leukemia (first remission, high risk; beyond first remission; refractory)
- Myelodysplastic syndrome
- Solid tumors (Refractory/relapse)
- No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
- HLA-haploidentical related donor available
Who cannot join
- Active fungal infections
- HIV positive
- Pregnant or nursing
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Asan Medical Center
Seoul, 138-736
Ho Joon Im, MD & PhD, 82-2-3010-3371, hojim@amc.seoul.kr
Ho Joon Im, MD & PhD
About this study
Official title: HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3 Depletion for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors After Conditioning of TBI, Fludarabine, Cyclophosphamide and Antithymocyte Globulin
RATIONALE: Conditioning with total body irradiation (TBI) and fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, T-cell depletion may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.
PURPOSE: This phase I/II trial is to evaluate the safety and efficacy of TBI, fludarabine, cyclophosphamide and antithymocyte globulin with T-cell depleted graft from haploidentical donors in treating patients with acute leukemia and myelodysplastic syndrome.
- Conditions
- Acute Leukemia, Myelodysplastic Syndrome, Solid Tumors
- Being tested
- anti-thymocyte globulin, filgrastim, Total body irradiation, Fludarabine, cyclophosphamide
- Keywords
- TBI, Fludarabine, CD3 depletion, Children and adolescents, Haploidentical hematopoietic stem cell transplantation
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anti-thymocyte globulinBiological
On days -10 to -9
filgrastimBiological
Beginning on day 4 and continuing until blood counts recover
Total body irradiationRadiation
2Gy D-6 to D-4
FludarabineDrug
30mg/M2 once daily IV on days -8 to -4
cyclophosphamideDrug
60 mg/kg IV on day-3 and -2
TacrolimusDrug
begin on 0
begin on 0
RituximabDrug
375mg/m2 on day +21
| Study group | Role | What they receive |
|---|---|---|
| HAPLO | Experimental | Not described Receives: Biological: anti-thymocyte globulin; Biological: filgrastim; Radiation: Total body irradiation; Drug: Fludarabine; Drug: cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells.
Measured: 2 years post-transplant
Secondary outcomes (4)
Engraftment and graft failure rates
Number of patients who failed to stable engraftment by 28 days
Measured: 28 days engraftment and graft failure
Incidence of acute GVHD
Number of patients with acute GVHD.
Measured: 100 days post-transplant
Treatment related mortality
Number of death after transplantation
Measured: 100 days post-transplant
Relapse rate and overall survival
Measured: 2 year after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2012 (estimated)
- Study startJan 2012
- Primary completionDec 2013 (estimated)
- Study completionMar 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Acute LeukemiaStudies of the same condition, open and under way.
- More trials run by Asan Medical CenterThe sponsor's other registered studies.
- 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Asan Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ho Joon Im, Asan Medical Center
- Investigators
- Ho Joon Im, MD & PhD, Principal investigator, Asan Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01509300
- Sponsor's protocol ID
- AMCPHO-SCT0902
- Status last verified
- Jan 2012
- First submitted
- 5 Jan 2012
- Last updated
- 13 Jan 2012
- Results posted
- No
Publications
Koh KN, Im HJ, Kim BE, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Haploidentical haematopoietic stem cell transplantation using CD3 or CD3/CD19 depletion and conditioning with fludarabine, cyclophosphamide and antithymocyte globulin for acquired severe aplastic anaemia. Br J Haematol. 2012 Apr;157(1):139-42. doi: 10.1111/j.1365-2141.2011.08924.x. Epub 2011 Nov 5. No abstract available.
PubMed 22055111Lang P, Handgretinger R. Haploidentical SCT in children: an update and future perspectives. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S54-9. doi: 10.1038/bmt.2008.285.
PubMed 18978746Im HJ, Koh KN, Suh JK, Lee SW, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients. Bone Marrow Transplant. 2015 Feb;50(2):225-31. doi: 10.1038/bmt.2014.232. Epub 2014 Oct 13.
PubMed 25310303
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3 Depletion for Children and Adolescents With Acute Leukemia, Myelodysplastic Syndrome and Solid Tumors After Conditioning of TBI, Fludarabine, Cyclophosphamide and Antithymocyte Globulin
- ClinicalTrials.gov ID
- NCT01509300
- Acronym / sponsor ID
- AMCPHO-SCT0902
- Conditions
- Acute Leukemia, Myelodysplastic Syndrome, Solid Tumors
- Keywords
- TBI, Fludarabine, CD3 depletion, Children and adolescents, Haploidentical hematopoietic stem cell transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Mar 2014 (estimated)
- First posted
- 13 Jan 2012 (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.
RATIONALE: Conditioning with total body irradiation (TBI) and fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, T-cell depletion may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.
PURPOSE: This phase I/II trial is to evaluate the safety and efficacy of TBI, fludarabine, cyclophosphamide and antithymocyte globulin with T-cell depleted graft from haploidentical donors in treating patients with acute leukemia and myelodysplastic syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HAPLO | Experimental | Biological: anti-thymocyte globulin, Biological: filgrastim, Radiation: Total body irradiation, Drug: Fludarabine, Drug: cyclophosphamide, Drug: Tacrolimus, Drug: Mycophenolate mofetil, Drug: Rituximab |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Disease characteristics
- No HLA-identical family member or closely matched (8 or 7 of 8 HLA-locus match) unrelated marrow donor available
- HLA-haploidentical related donor available
Key exclusion criteria (3 listed)
- Active fungal infections
- HIV positive
- Pregnant or nursing
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 |
|---|---|---|
| anti-thymocyte globulinOn days -10 to -9 | Biological | HAPLO |
| filgrastimBeginning on day 4 and continuing until blood counts recover | Biological | HAPLO |
| Total body irradiation2Gy D-6 to D-4 | Radiation | HAPLO |
| Fludarabine30mg/M2 once daily IV on days -8 to -4 | Drug | HAPLO |
| cyclophosphamide60 mg/kg IV on day-3 and -2 | Drug | HAPLO |
| Tacrolimusbegin on 0 | Drug | HAPLO |
| Mycophenolate mofetilbegin on 0 | Drug | HAPLO |
| Rituximab375mg/m2 on day +21 | Drug | HAPLO |
Primary outcome
Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells.
Time frame: 2 years post-transplant
4 secondary outcomes
Engraftment and graft failure rates
Time frame: 28 days engraftment and graft failure
Incidence of acute GVHD
Time frame: 100 days post-transplant
Treatment related mortality
Time frame: 100 days post-transplant
Relapse rate and overall survival
Time frame: 2 year after transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2012.
- Background: PubMed 22055111Koh KN, Im HJ, Kim BE, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Haploidentical haematopoietic stem cell transplantation using CD3 or CD3/CD19 depletion and conditioning with fludarabine, cyclophosphamide and antithymocyte globulin for acquired severe aplastic anaemia. Br J Haematol. 2012 Apr;157(1):139-42. doi: 10.1111/j.1365-2141.2011.08924.x. Epub 2011 Nov 5. No abstract available.
- Background: PubMed 18978746Lang P, Handgretinger R. Haploidentical SCT in children: an update and future perspectives. Bone Marrow Transplant. 2008 Oct;42 Suppl 2:S54-9. doi: 10.1038/bmt.2008.285.
- Derived from this study: PubMed 25310303Im HJ, Koh KN, Suh JK, Lee SW, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ. Refinement of treatment strategies in ex vivo T-cell-depleted haploidentical SCT for pediatric patients. Bone Marrow Transplant. 2015 Feb;50(2):225-31. doi: 10.1038/bmt.2014.232. Epub 2014 Oct 13.
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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.
Ho Joon Im, MD & PhD principal investigator
Asan Medical Center
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 anti-thymocyte globulin, filgrastim and Total body irradiation in people with Acute Leukemia and Myelodysplastic Syndrome, 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?
The sponsor lists 7 inclusion and 3 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 13 Jan 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 Ho Joon Im, MD & PhD (82-2-3010-3371, hojim@amc.seoul.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Transplantation-related mortality and overall survival of TBI, Fludarabine, Cyclophosphamide and anti-thymocyte globulin for engraftment of CD3 depleted haploidentical peripheral blood stem cells. (time frame: 2 years post-transplant). 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 NCT01509300, which also lists 3 linked publications.
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 13 Jan 2012.