Hematological MalignanciesPhase 2NCT02199041
Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen
A Phase 2 study testing Cyclophosphamide, Thiotepa and Fludarabine in people with Hematological Malignancies, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical… Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Hematological Malignancies
- What is being tested?
- Cyclophosphamide, Thiotepa and Fludarabine
- Type of treatment
- Pill or oral medicine, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Jul 2014to 23 May 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Age less than or equal to 21 years old.
- Does not have a suitable matched related/sibling donor (MSD) or volunteer matched unrelated donor (MUD) available in the necessary time for stem cell donation.
- Has a suitable partially human leukocyte antigen (HLA)-matched (≥ 3 of 6) family member donor.
- Has a partially HLA-matched single umbilical cord blood (UCB) unit (≥ 4 of 6) with adequate cell dose. UCB units must fulfill eligibility as outlined in 21 CFR 1271 and agency guidance.
- High-risk hematologic malignancy.
- High risk acute lymphocytic leukemia (ALL) in complete remission-1 (CR)1. [Examples include, but not limited to t(9;22), hypodiploid,, M2 or greater marrow at the end of induction, infants with mixed lineage leukemia (MLL) fusion or t(4;11)].
Show all 35Show fewer
- ALL in High risk CR2. [Examples include but not limited to t(9;22), bone marrow (BM) relapse <36 mo CR1, T-ALL, very early (< 6mo CR1) isolated central nervous system (CNS) relapse.]
- ALL in CR3 or subsequent.
- Acute myeloid leukemia (AML) in high risk CR1. [Examples include but not limited to preceding MDS, 5q-, -5, -7, FAB M6, FAB M7 not t(1;22), minimal residual disease (MRD) ≥ 5% on day 22 (AML08), M3 marrow after induction 1, M2 marrow after two cycles of induction, FLT3-ITD.]
- AML in CR2 or subsequent.
- Therapy related AML, with prior malignancy in CR > 12mo
- Myelodysplastic syndrome (MDS), primary or secondary
- Natural killer (NK) cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
- Chronic myeloid leukemia (CML) in accelerated phase, or in chronic phase with persistent molecular positivity or intolerance to tyrosine kinase inhibitor.
- Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous hematopoietic cell transplantation (HCT), or unable to mobilize stem cells for autologous HCT.
- Non-Hodgkin lymphoma in CR2 or subsequent.
- Juvenile myelomonocytic leukemia (JMML).
- Refractory hematologic malignancies [ALL, AML, chronic myeloid leukemia (CML) in blast crisis, Hodgkin or non-Hodgkin lymphoma] due to chemoresistant relapse or primary induction failure.
- All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
- Patient must fulfill pre-transplant evaluation:
- Cardiac Function: Left ventricular ejection fraction (LVEF) ≥ 40% or shortening fraction (SF) ≥ 25%.
- Creatinine clearance (CrCL) or glomerular filtration rate (GFR) ≥ 50 ml/min/1.73m2.
- Forced vital capacity (FVC) ≥ 50% of predicted value or pulse oximetry (Pox) ≥ 92% on room air.
- Karnofsky or Lansky performance score ≥ 50.
- Bilirubin ≤ 3 times the upper limit of normal for age.
- Alanine aminotransferase (ALT) ≤ 5x the upper limit of normal for age.
- Aspartate aminotransferase (AST) ≤ 5x the upper limit of normal for age.
- At least single haplotype matched (≥ 3 of 6) family member
- At least 18 years of age.
- Human immunodeficiency virus (HIV) negative.
- Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).
- Not breast feeding.
- Regarding eligibility, is identified as either:
- Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR
- Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271.
Who cannot join
- Patient has a suitable MSD, volunteer matched unrelated donor (MURD), or killer-immunoglobulin receptors (KIR) mismatched haploidentical donor available in the necessary time for stem cell donation.
- Patient has any other active malignancy other than the one for which HCT is indicated.
- Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
- Patient is breast feeding.
- Patient has Down Syndrome.
- Patient has a current uncontrolled bacterial, fungal, or viral infection per the judgment of the principal investigator.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical donor identified, will receive a combined T cell depleted (TCD) haploidentical peripheral blood stem cell (PBSC) and unrelated umbilical cord blood transplantation (UCBT) using a total lymphoid irradiation (TLI) based preparative regimen.
Primary objective:
Read more
- To estimate the incidence of donor derived neutrophil engraftment by day +42 post-transplant for participants with high-risk hematologic malignancies undergoing a total lymphoid irradiation (TLI)-based hematopoietic cell transplantation (HCT) using a T cell depleted (TCI) haploidentical donor peripheral blood stem cell (PBSC) donor combined with an unrelated umbilical cord blood (UCB) donor.
Secondary objectives:
- Estimate the incidence of malignant relapse, event-free survival (EFS), and overall survival (OS) at one-year post-transplantation.
- Estimate the incidence and severity of acute and chronic graft versus host disease (GVHD) in the first 100 days after transplantation.
- Estimate the incidence of secondary graft failure transplant related mortality (TRM) and transplant related morbidity in the first 100 days after HCT.
Read the detailed description
Prior to stem cell infusion, participants will receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
- Conditions
- Hematological Malignancies
- Being tested
- Cyclophosphamide, Thiotepa, Fludarabine, Melphalan, Mesna
- Keywords
- Cord Blood, Haploidentical, Reduced Intensity Regimen
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Cytoxan
Given by intravenous infusion as part of the preparative regimen.
ThiotepaDrug
Also called Thioplex(R), TESPA, TSPA
Given by intravenous infusion as part of the preparative regimen.
FludarabineDrug
Also called Fludara
Given by intravenous infusion as part of the preparative regimen.
MelphalanDrug
Also called L-phenylalanine mustard, Phenylalanine mustard, L-PAM, L-sarcolysin
Given by intravenous infusion as part of the preparative regimen.
MesnaDrug
Also called Mesnex
Mesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide.
G-CSFBiological
Also called Granulocyte colony-stimulating factor (C-CSF), Filgrastim, Neupogen(R)
Given either by intravenous infusion or subcutaneously daily until absolute neutrophil count (ANC) >2000 for 3 consecutive days.
Also called MMF, CellCept(R)
Given either orally or by intravenous infusion as part of the post-transplantation immunosuppression.
TacrolimusDrug
Also called FK506, Prograf(R), Protopic(R)
Given either orally or by intravenous infusion as part of the post-transplantation immunosuppression.
Also called Medrol(R), Solu-Medrol
Given either intravenously or orally, if needed to treat graft-versus-host-disease (GVHD).
Total lymphoid irradiationRadiation
Also called TLI
TLI will be administered in divided fractions given at a minimum of 6 hours apart.
Lymphocyte infusionsBiological
Also called Donor lymphocyte infusions, DLI
Donors will undergo haploidentical mobilization with G-CSF. Cells will be collected by leukapheresis over two days, then processed using the investigational CliniMACS device and CD34 Microbead reagent as directed by the manufacturer.
CliniMACSDevice
Also called Cell Selection System
The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Interventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, granulocyte colony-stimulating factor (G-CSF), mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions.
Cells for infusion are prepared using the CliniMACS System. Receives: Drug: Cyclophosphamide; Drug: Thiotepa; Drug: Fludarabine; Drug: Melphalan; Drug: Mesna; Biological: G-CSF; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methylprednisolone; Radiation: Total lymphoid irradiation; Biological: Lymphocyte infusions; Device: CliniMACS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Neutrophil Engraftment
Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided.
Measured: Until day 42 post-transplant
Secondary outcomes (8)
Number of Participants With Malignant Relapse
Relapse was evaluated using standard World Health Organization (WHO) criteria for each disease. The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Relapse defined as the recurrence of original disease. Death is the competing risk event. The analysis will be implemented using Statistical Analysis System (SAS) macro (bmacro252-Excel2007\cin).
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced malignant relapse is provided
Measured: One year after transplantation
Number of Participants With Event-free Survival (EFS)
The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided.
Measured: One year after transplantation
Number of Participants With Overall Survival (OS)
The Kaplan-Meier estimate of OS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007\kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) - date of hematopoietic cell transplantation (HCT) and all participants surviving after 1 year post-transplant will be considered as censored.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not die at 1 year post-transplant is provided.
Measured: One year after transplantation
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Cumulative incidence of acute GVHD was estimated using Kalbfleisch-Prentice method. Death is the competing risk event. SAS macro (bmacro252-Excel2007\cin) available at St. Jude was used for analysis. Severity of GVHD and stage were determined using the Clinical Oncology Group (COG) Stem Cell Committee Consensus Guidelines for establishing organ stage and overall grade of acute GVHD. Participants are graded on a scale from I to IV, with I being mild and IV being severe.
Overall Clinical Grade (based on the highest stage obtained):
Grade 0: No stage 1-4 of any organ. Grade I: Stage 1-2 skin and no liver or gut involvement. Grade II: Stage 3 skin, or Stage I liver involvement, or Stage 1 gastrointestinal (GI).
Grade III: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI. Grade IV: Stage 4 skin, liver or GI involvement.
Due to early close of study, a small number of patients were enrolled. The number of patients who experienced acute GVHD is provided.
Measured: 100 days after transplantation
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Cumulative incidence of acute and chronic GVHD was estimated using Kalbfleisch-Prentice method. Death is competing risk event. SAS macro (bmacro252-Excel2007\cin) available St. Jude was used for such analysis. Severity of chronic GVHD was evaluated using National Institutes of Health (NIH) Consensus Global Severity Scoring. Mild is considered a better outcome with severe being the worst.
Criteria for grading chronic GVHD:
Mild: 1-2 organs/sites, maximum organ score of 1, and lung score of 1. Moderate: 3 or more organs/sites and maximum organ score of 1 and lung score of 1, OR at least 1 organ/site and maximum organ score of 2 and lung score of 1. Severe: At least 1 organ/site and maximum organ score of 3 and lung score of 2-3.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced chronic GVHD is provided.
Measured: 100 days after transplantation
Number of Participants With Secondary Graft Failure
The cumulative incidence of secondary graft failure will be estimated using the Kalbfleisch-Prentice method. Deaths due to toxicity and relapse before day 100 are the competing events.
Secondary graft failure or graft rejection will be defined as no evidence of donor chimerism by umbilical cord blood (UCB) and/or haploidentical donor (<10%), or too few cells to perform adequate chimerism analysis, in research participants with prior neutrophil engraftment.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced secondary graft failure is provided.
Measured: 100 days after transplantation
Number of Participants With Transplant-related Mortality (TRM)
TRM is any death in remission and related to protocol therapy. The cumulative incidence of TRM was estimated using the Kalbfleisch-Prentice method. Deaths before day 100 because of other reasons are the completing events.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced TRM is provided.
Measured: 100 days after transplantation
Number of Participants With Transplant-related Morbidity
Any patient who had adverse events listed either as probable or definite in the first 100 days post-transplant are counted as transplant-related morbidity. The cumulative incidence of transplant-related morbidity will be estimated using the Kalbfleisch-Prentice method. Deaths before day 100 are the competing risk events.
Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced at least one-transplant-related morbidity is provided.
Measured: 100 days after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2014 (estimated)
- Study start11 Jul 2014
- Primary completion23 May 2017
- Study completion23 May 2017
Why it stopped: The study was halted early due to slow accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brandon Triplett, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02199041
- Sponsor's protocol ID
- HAPCORD
- Other IDs
- NCI-2014-00526
- Status last verified
- Aug 2017
- First submitted
- 9 Jul 2014
- Last updated
- 7 Feb 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen
- ClinicalTrials.gov ID
- NCT02199041
- Acronym / sponsor ID
- HAPCORD
- Conditions
- Hematological Malignancies
- Keywords
- Cord Blood, Haploidentical, Reduced Intensity Regimen
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was halted early due to slow accrual.
- Enrolment
- 24 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 11 Jul 2014 (actual)
- Primary completion
- 23 May 2017 (actual)
- Study completion
- 23 May 2017 (actual)
- First posted
- 24 Jul 2014 (estimated)
- Results
- Posted 7 Feb 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical donor identified, will receive a combined T cell depleted (TCD) haploidentical peripheral blood stem cell (PBSC) and unrelated umbilical cord blood transplantation (UCBT) using a total lymphoid irradiation (TLI) based preparative regimen.
Primary objective:
- To estimate the incidence of donor derived neutrophil engraftment by day +42 post-transplant for participants with high-risk hematologic malignancies undergoing a total lymphoid irradiation (TLI)-based hematopoietic cell transplantation (HCT) using a T cell depleted (TCI) haploidentical donor peripheral blood stem cell (PBSC) donor combined with an unrelated umbilical cord blood (UCB) donor.
Secondary objectives:
- Estimate the incidence of malignant relapse, event-free survival (EFS), and overall survival (OS) at one-year post-transplantation.
- Estimate the incidence and severity of acute and chronic graft versus host disease (GVHD) in the first 100 days after transplantation.
- Estimate the incidence of secondary graft failure transplant related mortality (TRM) and transplant related morbidity in the first 100 days after HCT.
Detailed description
Prior to stem cell infusion, participants will receive a preparative regimen of total lymphoid irradiation (TLI), fludarabine, cyclophosphamide, melphalan, and thiotepa to prepare their bone marrow. Thereafter, they will receive a hematopoietic cell graft from a haploidentical donor and an unrelated umbilical cord blood donor. Post-transplantation immunosuppressive treatment will include tacrolimus and mycophenolate mofetil.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Cyclophosphamide, Drug: Thiotepa, Drug: Fludarabine, Drug: Melphalan, Drug: Mesna, Biological: G-CSF, Drug: Mycophenolate mofetil, Drug: Tacrolimus, Drug: Methylprednisolone, Radiation: Total lymphoid irradiation, Biological: Lymphocyte infusions, Device: CliniMACSInterventions: cyclophosphamide, thiotepa, fludarabine, melphalan, mesna, granulocyte colony-stimulating factor (G-CSF), mycophenolate mofetil, tacrolimus, methylprednisolone, total lymphoid irradiation, and lymphocyte infusions. Cells for infusion are prepared using the CliniMACS System. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (35 listed)
- Age less than or equal to 21 years old.
- Does not have a suitable matched related/sibling donor (MSD) or volunteer matched unrelated donor (MUD) available in the necessary time for stem cell donation.
- Has a suitable partially human leukocyte antigen (HLA)-matched (≥ 3 of 6) family member donor.
- Has a partially HLA-matched single umbilical cord blood (UCB) unit (≥ 4 of 6) with adequate cell dose. UCB units must fulfill eligibility as outlined in 21 CFR 1271 and agency guidance.
- High-risk hematologic malignancy.
Key exclusion criteria (6 listed)
- Patient has a suitable MSD, volunteer matched unrelated donor (MURD), or killer-immunoglobulin receptors (KIR) mismatched haploidentical donor available in the necessary time for stem cell donation.
- Patient has any other active malignancy other than the one for which HCT is indicated.
- Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
- Patient is breast feeding.
- Patient has Down Syndrome.
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 |
|---|---|---|
| CyclophosphamideAlso: CytoxanGiven by intravenous infusion as part of the preparative regimen. | Drug | Treatment |
| ThiotepaAlso: Thioplex(R), TESPA, TSPAGiven by intravenous infusion as part of the preparative regimen. | Drug | Treatment |
| FludarabineAlso: FludaraGiven by intravenous infusion as part of the preparative regimen. | Drug | Treatment |
| MelphalanAlso: L-phenylalanine mustard, Phenylalanine mustard, L-PAM, L-sarcolysinGiven by intravenous infusion as part of the preparative regimen. | Drug | Treatment |
| MesnaAlso: MesnexMesna is generally dosed at approximately 25% of the cyclophosphamide dose. It is generally given intravenously prior to and again at 3, 6 and 9 hours following each dose of cyclophosphamide. | Drug | Treatment |
| G-CSFAlso: Granulocyte colony-stimulating factor (C-CSF), Filgrastim, Neupogen(R)Given either by intravenous infusion or subcutaneously daily until absolute neutrophil count (ANC) >2000 for 3 consecutive days. | Biological | Treatment |
| Mycophenolate mofetilAlso: MMF, CellCept(R)Given either orally or by intravenous infusion as part of the post-transplantation immunosuppression. | Drug | Treatment |
| TacrolimusAlso: FK506, Prograf(R), Protopic(R)Given either orally or by intravenous infusion as part of the post-transplantation immunosuppression. | Drug | Treatment |
| MethylprednisoloneAlso: Medrol(R), Solu-MedrolGiven either intravenously or orally, if needed to treat graft-versus-host-disease (GVHD). | Drug | Treatment |
| Total lymphoid irradiationAlso: TLITLI will be administered in divided fractions given at a minimum of 6 hours apart. | Radiation | Treatment |
| Lymphocyte infusionsAlso: Donor lymphocyte infusions, DLIDonors will undergo haploidentical mobilization with G-CSF. Cells will be collected by leukapheresis over two days, then processed using the investigational CliniMACS device and CD34 Microbead reagent as directed by the manufacturer. | Biological | Treatment |
| CliniMACSAlso: Cell Selection SystemThe mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells. | Device | Treatment |
Primary outcome
Number of Participants With Neutrophil Engraftment
Time frame: Until day 42 post-transplant
Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided.
8 secondary outcomes
Number of Participants With Malignant Relapse
Time frame: One year after transplantation
Number of Participants With Event-free Survival (EFS)
Time frame: One year after transplantation
Number of Participants With Overall Survival (OS)
Time frame: One year after transplantation
Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Time frame: 100 days after transplantation
Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
Time frame: 100 days after transplantation
Number of Participants With Secondary Graft Failure
Time frame: 100 days after transplantation
Number of Participants With Transplant-related Mortality (TRM)
Time frame: 100 days after transplantation
Number of Participants With Transplant-related Morbidity
Time frame: 100 days after transplantation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink 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.
Brandon Triplett, MD principal investigator
St. Jude Children's Research Hospital
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, Thiotepa and Fludarabine in people with Hematological Malignancies, run by St. Jude Children's Research Hospital at 1 site in United States.
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 35 inclusion and 6 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: stopped early, as of the sponsor's last update on 7 Feb 2018. 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 United States. 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?
Yes. Results are posted in the registry (first posted 7 Feb 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Neutrophil Engraftment (time frame: Until day 42 post-transplant). 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 7 Feb 2018.