Haploidentical Hematopoietic Stem Cell TransplantationPhase 1/2NCT02014506
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents
A Phase 1/2 study testing Fludarabine, Cyclophosphamide and anti-thymocyte globulin in people with Haploidentical Hematopoietic Stem Cell Transplantation and Malignant Disease, run by Asan Medical Center at 1 site in South Korea.
Trial summary
Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Haploidentical Hematopoietic Stem Cell Transplantation, Malignant Disease, Non-malignant Disease
- What is being tested?
- Fludarabine, Cyclophosphamide and anti-thymocyte globulin
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jan 2017to Dec 2018
- 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 inclusions
- Hematologic malignancy:
- Acute lymphoblastic leukemia including induction failure, CR1 (Ph+, t(4:11), hypodiploid and other very high risk features), ≥ CR2, infant ALL with MLL or other unfavorable features
- Acute myeloid leukemia excluding CR1 with t(8:21), inv(16), t(15:17), and Down syndrome
- Myelodysplastic syndrome: RCC with -7 or RCC in need of transfusion
- Chronic myeloid leukemia in AP
Show all 23Show fewer
- Juvenile myelomonocytic leukemia
- Malignant lymphoma, NHL or HD, after failed autologous HSCT
- Other
- Non-hematologic malignancy
- Relapsed or refractory solid tumors including neuroblastoma, rhabdomyosarcoma and so on
- Non-malignant hematologic disease
- Acquired severe and very severe aplastic anemia
- Fanconi anemia
- Paroxysmal nocturnal hemoglobinuria
- Congenital dyserythropoietic anemia
- Others
- Inherited or metabolic disease
- Hemophagocytic lymphohistiocytosis
- Malignant osteopetrosis
- Storage diseases
- Others B. Recipient inclusions
- 1. Age < 21 years 2. No HLA-identical stem cell donor available 3. Lansky-Play performance score >60 4. No active infection at the time of transplantation
Who cannot join
- HIV-infection
- Presence of active and serious infection
- Cardiac ejection fraction <35% on echocardiography
- Severe pulmonary dysfunction (DLCO <30%)
- Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L
- Concurrent severe or uncontrolled medical disease
- Patients who are pregnant
- Patients unwilling or unable to comply with the protocol or unable to give informed consent
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
Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.
- Conditions
- Haploidentical Hematopoietic Stem Cell Transplantation, Malignant Disease, Non-malignant Disease
- Being tested
- Fludarabine, Cyclophosphamide, anti-thymocyte globulin, filgrastim, Total body irradiation
- Keywords
- Children and adolescents, Malignant disease, Non-malignant disease, TCRαβ depletion, Haploidentical hematopoietic stem cell transplantation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
40mg/M2 once daily IV on days -7 to -2
CyclophosphamideDrug
50 mg/kg IV on day -3 and -2
anti-thymocyte globulinBiological
filgrastimBiological
Beginning on day 4 and continuing until blood counts recover
Total body irradiationRadiation
200 cGy per day on D-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 \& -4 (severe aplastic anemia)
TCRαβ-depleted hematopoietic cell transplantationProcedure or surgery
CliniMACSDevice
Immunogenetic depletion of TCRαβ cells
| Study group | Role | What they receive |
|---|---|---|
| HAPLO | Experimental | Not described Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Biological: anti-thymocyte globulin; Biological: filgrastim; Radiation: Total body irradiation; Procedure: TCRαβ-depleted hematopoietic cell transplantation; Device: CliniMACS |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft
Measured: 1 year posttransplant
Secondary outcomes (10)
To assess engraftment and graft failure
Measured: 28 days posttransplant
To estimate the risk of acute GVHD
Measured: 100 days posttransplant
To estimate the incidence of relapse
Measured: 100 days and 1 year post-transplant
To estimate the incidence and severity of chronic GVHD
Measured: 1 year posttransplant
To estimate the overall survival
Measured: 1 year posttransplant
To estimate the incidence of bacterial, fungal and viral infection
Measured: 100 days and 1 year posttransplant
To estimate the reactivation rate of CMV, EBV
Measured: 100 days and 1 year posttransplant
To evaluate the immune reconstitution of T, B, and NK cells
Measured: days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant
To evaluate the lineage-specific chimerism using flow cytomery of CD3+, CD19, CD56, TCR αβ, and TCRγδ at pre-transplant
Measured: days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant
To assess event free survival
Measured: 1 year posttransplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2013 (estimated)
- Study startJan 2017
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (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 Haploidentical Hematopoietic Stem Cell TransplantationStudies 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
- 30 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
- NCT02014506
- Sponsor's protocol ID
- AMCPHO-SCT1303
- Status last verified
- Mar 2017
- First submitted
- 4 Dec 2013
- Last updated
- 28 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Park JA, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ, Im HJ. Successful rescue of early graft failure in pediatric patients using T-cell-depleted haploidentical hematopoietic SCT. Bone Marrow Transplant. 2014 Feb;49(2):270-5. doi: 10.1038/bmt.2013.163. Epub 2013 Oct 21.
PubMed 24141651Im HJ, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Chi HS, Seo JJ. Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2013 May;19(5):754-9. doi: 10.1016/j.bbmt.2013.01.023. Epub 2013 Feb 1.
PubMed 23380343Schumm M, Lang P, Bethge W, Faul C, Feuchtinger T, Pfeiffer M, Vogel W, Huppert V, Handgretinger R. Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy. 2013 Oct;15(10):1253-8. doi: 10.1016/j.jcyt.2013.05.014.
PubMed 23993299
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation in Children and Adolescents
- ClinicalTrials.gov ID
- NCT02014506
- Acronym / sponsor ID
- AMCPHO-SCT1303
- Conditions
- Haploidentical Hematopoietic Stem Cell Transplantation, Malignant Disease, Non-malignant Disease
- Keywords
- Children and adolescents, Malignant disease, Non-malignant disease, TCRαβ depletion, Haploidentical hematopoietic stem cell transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- Jan 2017 (actual)
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 18 Dec 2013 (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.
Purpose of study: This phase I/II trial is to evaluate the safety and feasibility of TCRαβ-depleted graft from haploidentical family donors in treating children and adolescents with malignant or non-malignant diseases.
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 | Drug: Fludarabine, Drug: Cyclophosphamide, Biological: anti-thymocyte globulin, Biological: filgrastim, Radiation: Total body irradiation, Procedure: TCRαβ-depleted hematopoietic cell transplantation, Device: CliniMACS |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (23 listed)
- Disease inclusions
- Hematologic malignancy:
- Non-hematologic malignancy
- Non-malignant hematologic disease
- Inherited or metabolic disease
Key exclusion criteria (8 listed)
- HIV-infection
- Presence of active and serious infection
- Cardiac ejection fraction <35% on echocardiography
- Severe pulmonary dysfunction (DLCO <30%)
- Liver function abnormalities with bilirubin >4mg/dL and elevation of transaminases > 400U/L
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 |
|---|---|---|
| Fludarabine40mg/M2 once daily IV on days -7 to -2 | Drug | HAPLO |
| Cyclophosphamide50 mg/kg IV on day -3 and -2 | Drug | HAPLO |
| anti-thymocyte globulin | Biological | HAPLO |
| filgrastimBeginning on day 4 and continuing until blood counts recover | Biological | HAPLO |
| Total body irradiation200 cGy per day on D-6 to -4 (eligible disease except aplastic anemia) 200 cGy per day on D-5 \& -4 (severe aplastic anemia) | Radiation | HAPLO |
| TCRαβ-depleted hematopoietic cell transplantation | Procedure or surgery | HAPLO |
| CliniMACSImmunogenetic depletion of TCRαβ cells | Device | HAPLO |
Primary outcome
To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft
Time frame: 1 year posttransplant
10 secondary outcomes
To assess engraftment and graft failure
Time frame: 28 days posttransplant
To estimate the risk of acute GVHD
Time frame: 100 days posttransplant
To estimate the incidence of relapse
Time frame: 100 days and 1 year post-transplant
To estimate the incidence and severity of chronic GVHD
Time frame: 1 year posttransplant
To estimate the overall survival
Time frame: 1 year posttransplant
To estimate the incidence of bacterial, fungal and viral infection
Time frame: 100 days and 1 year posttransplant
To estimate the reactivation rate of CMV, EBV
Time frame: 100 days and 1 year posttransplant
To evaluate the immune reconstitution of T, B, and NK cells
Time frame: days 7, 14, 21, 28, 60, 90, 180, 270, and 365 days post-transplant
To evaluate the lineage-specific chimerism using flow cytomery of CD3+, CD19, CD56, TCR αβ, and TCRγδ at pre-transplant
Time frame: days 7, 10, 14, 21, 28, 60, 90, 180, 270 and 365 post-transplant
To assess event free survival
Time frame: 1 year posttransplant
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Mar 2017.
- Background: PubMed 24141651Park JA, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Seo JJ, Im HJ. Successful rescue of early graft failure in pediatric patients using T-cell-depleted haploidentical hematopoietic SCT. Bone Marrow Transplant. 2014 Feb;49(2):270-5. doi: 10.1038/bmt.2013.163. Epub 2013 Oct 21.
- Background: PubMed 23380343Im HJ, Koh KN, Choi ES, Jang S, Kwon SW, Park CJ, Chi HS, Seo JJ. Excellent outcome of haploidentical hematopoietic stem cell transplantation in children and adolescents with acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2013 May;19(5):754-9. doi: 10.1016/j.bbmt.2013.01.023. Epub 2013 Feb 1.
- Background: PubMed 23993299Schumm M, Lang P, Bethge W, Faul C, Feuchtinger T, Pfeiffer M, Vogel W, Huppert V, Handgretinger R. Depletion of T-cell receptor alpha/beta and CD19 positive cells from apheresis products with the CliniMACS device. Cytotherapy. 2013 Oct;15(10):1253-8. doi: 10.1016/j.jcyt.2013.05.014.
- fludarabineLink provided by the sponsor.
- cyclophosphamideLink provided by the sponsor.
- leukemiaLink provided by the sponsor.
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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 Fludarabine, Cyclophosphamide and anti-thymocyte globulin in people with Haploidentical Hematopoietic Stem Cell Transplantation and Malignant Disease, 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 23 inclusion and 8 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 28 Mar 2017. 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?
To evaluate tralsplant-related mortality after haploidentical hematopoietic stem cell transplantation using TCRαβ-depleted graft (time frame: 1 year posttransplant). 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 NCT02014506, 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 28 Mar 2017.