Acute LeukemiasNCT02145039
Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases
A study testing Fludarabine, Cyclophosphamide and Total Body Irradiation in people with Acute Leukemias and Burkitt's Lymphoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of fludarabine, cyclophosphamide and low dose total body irradiation (TBI), is designed for the treatment of patients with advanced and/or high… Read more about this study
- Who is it for?
- Ages Up to 74 years
- Conditions the study is for
- Acute Leukemias, Burkitt's Lymphoma, Chronic Myelogenous Leukemia
- What is being tested?
- Fludarabine, Cyclophosphamide 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
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2014to Jan 2019
- Phase
- Interventional
- 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 74 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must be <75 years old with no 7/8 or 8/8 HLA-matched sibling donor
- One or more potential related mismatched donors (e.g. biologic parent (s) or siblings (full or half) or children). Low resolution using DNA based typing at HLA-A, -B and -DRB1 for potential haploidentical donors is required.
- All diseases listed below are advanced hematologic malignancies not curable by conventional chemotherapy. Responses to conventional treatment range from zero to 30% but are typically short lived.
- Acute Lymphoblastic Leukemia (ALL) in first complete remission (CR1) that is NOT considered favorable-risk.
- Acute Myelogenous Leukemia (AML) in first complete remission (CR1) that is NOT considered as favorable-risk.
- Acute Leukemias in 2nd or subsequent CR
Show all 28Show fewer
- Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR, adult T-cell leukemia/lymphoma in first or subsequent CR
- Burkitt's lymphoma in CR2 or subsequent CR
- Natural killer cell malignancies after response to initial therapy
- Chronic myelogenous leukemia: all types except refractory blast crisis.
- Large-cell lymphoma, Hodgkin lymphoma and multiple myeloma with chemotherapy sensitive disease that has failed or patients who are ineligible for an autologous transplant.
- Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma, which have progressed within 12 months of achieving a partial or complete remission.
- Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive.
- Refractory leukemia or MDS These patients may be taken to transplant in aplasia after induction or re-induction chemotherapy or radiolabeled antibody.
- Bone marrow failure syndromes, except for Fanconi Anemia
- Myeloproliferative syndromes
- Adequate organ function is defined as:
- Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 35%. For children that are not able to cooperate with multigated acquisition scan (MUGA) and echocardiography, such should be clearly stated in the physician's note
- Pulmonary: Diffusing capacity of lung for carbon monoxide (DLCO) > 30% predicted, and absence of O2 requirements. For children that are not able to cooperate with pulmonary function tests (PFTs), a pulse oximetry with exercise should be attempted. If nether test can be obtained it should be clearly stated in the physician's note.
- Liver: Transaminases < 5 x upper limit of normal and bilirubin < 3 x upper limit of normal
- Renal: serum creatinine < 2.0 mg/dl (adults) or glomerular filtration rate (GFR) >40 mL/min/1.73m2 (peds). Patients with a creatinine > 1.2 mg/dl or a history of renal dysfunction must have glomerular filtration rate (GFR) > 40 mL/min/1.73m2.
- Adequate performance status is defined as Karnofsky score ≥ 60% (> 16 years of age) or Lansky score ≥ 50 (pediatrics)
- If recent mold infection e.g. Aspergillus - must have minimum of 30 days of appropriate treatment before bone marrow transplant (BMT) and infection controlled and be cleared by Infectious Disease.
- Second BMT: Must be > 3 months after prior myeloablative transplant.
- Patients must be ineligible for autologous transplantation due to prior autologous transplant, an inadequate autologous stem cell harvest, inability to withstand a myeloablative preparative regimen, or clinically aggressive/high risk disease.
- Patients are eligible for transplantation if there is no evidence of progressive disease by imaging modalities or biopsy. Persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.
- Patients with stable disease are eligible for transplantation if the largest residual nodal mass is < 5 cm (approximately). For patients who have responded to preceding therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately).
- Voluntary written consent (adult or parental/guardian)
Who cannot join
- Available and clinically suitable 5-6/6 HLA-A, B, DRB1 matched sibling donor
- Pregnant or breastfeeding
- Evidence of HIV infection or known HIV positive serology
- Current active serious infection
- Unless in post-chemotherapy and radioimmunoconjugated antibody induced aplasia, when he/she would be eligible, patients with acute leukemia in morphologic relapse/ persistent disease defined as > 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods) or associated cytogenetic markers that allows morphologic relapse to be distinguished are not eligible.
- Chronic myeloid leukemia (CML) in refractory blast crisis
- Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.
- active central nervous system malignancy
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
About this study
Official title: Reduced Intensity Conditioning (RIC) and Transplantation of HLA(Human Leukocyte Antigen)-Haploidentical Related Bone Marrow (Haplo-BM) For Patients With Hematologic Diseases
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of fludarabine, cyclophosphamide and low dose total body irradiation (TBI), is designed for the treatment of patients with advanced and/or high risk diseases.
- Being tested
- Fludarabine, Cyclophosphamide, Total Body Irradiation, Haploidentical stem cell transplant
- Keywords
- Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Burkitt's lymphoma, Natural killer cell malignancies, Chronic myelogenous leukemia, Myelodysplastic syndrome, Large-cell lymphoma, Hodgkin lymphoma, Multiple myeloma, Chronic lymphocytic leukemia (CLL), Small lymphocytic lymphoma (SLL), Marginal zone B-cell lymphoma, Follicular lymphoma, Lymphoplasmacytic lymphoma, Mantle-cell lymphoma, Prolymphocytic leukemia, Refractory leukemia, Myelodysplastic syndrome (MDS), Bone marrow failure syndromes, Myeloproliferative syndromes
- Medical subject headings
- Burkitt Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Dendritic Cell Sarcoma, Interdigitating, Hodgkin Disease, Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara
Fludarabine 30 mg/m2 IV over 30-60 minutes on days -6 through -2 before transplant.
CyclophosphamideDrug
Cyclophosphamide 14.5 mg/kg IV over 1-2 hours on days -6 and -5 before transplant and Cyclophosphamide 50 mg/kg IV on days 3 and 4 post-transplant.
Total Body IrradiationRadiation
Also called TBI
TBI 200cGy on day -1 before transplant.
Haploidentical stem cell transplantBiological
Also called HSCT
Non-T-cell depleted bone marrow infusion
| Study group | Role | What they receive |
|---|---|---|
| Haploidentical stem cell transplant | Experimental | This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen consists of fludarabine, cyclophosphamide and low dose total body irradiation (TBI). Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Radiation: Total Body Irradiation; Biological: Haploidentical stem cell transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
2 Year Survival
Percentage of patients that survive 2 years post-transplant
Measured: 2 years
Secondary outcomes (5)
Number of Patients With Hematopoietic Engraftment
Engraftment is defined as absolute neutrophil count (ANC) ≥ 5 X 10^8/L for 3 consecutive measurements.
Measured: 42 days
Number of Patients With Chimerism
Number of patients with chimerism at day 100, 6 months and 1 year
Measured: 100 days
Number of Patients Experiencing Acute Graft-versus-host Disease by 100 Days
Measured: 100 days
Number of Patients Experiencing Chronic Graft-versus-host Disease by 1 Year
Measured: 1 year
Number of Patients Experiencing Transplant Related Mortality (TRM)
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2014 (estimated)
- Study startOct 2014
- Primary completionJan 2018
- Study completionJan 2019
Why it stopped: Replaced by another study.
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 Acute LeukemiasStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Claudio Brunstein, MD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02145039
- Sponsor's protocol ID
- 2013OC116
- Other IDs
- MT2013-33C
- Status last verified
- Dec 2019
- First submitted
- 20 May 2014
- Last updated
- 26 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Intensity Conditioning (RIC) and Transplantation of HLA(Human Leukocyte Antigen)-Haploidentical Related Bone Marrow (Haplo-BM) For Patients With Hematologic Diseases
- ClinicalTrials.gov ID
- NCT02145039
- Acronym / sponsor ID
- 2013OC116
- Keywords
- Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Burkitt's lymphoma, Natural killer cell malignancies, Chronic myelogenous leukemia, Myelodysplastic syndrome, Large-cell lymphoma, Hodgkin lymphoma
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Replaced by another study.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Oct 2014 (actual)
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jan 2019 (actual)
- First posted
- 22 May 2014 (estimated)
- Results
- Posted 26 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of fludarabine, cyclophosphamide and low dose total body irradiation (TBI), is designed for the treatment of patients with advanced and/or high risk diseases.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haploidentical stem cell transplant | Experimental | Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Haploidentical stem cell transplantThis is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen consists of fludarabine, cyclophosphamide and low dose total body irradiation (TBI). |
Who may qualify
- Ages
- Up to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (28 listed)
- Must be <75 years old with no 7/8 or 8/8 HLA-matched sibling donor
- One or more potential related mismatched donors (e.g. biologic parent (s) or siblings (full or half) or children). Low resolution using DNA based typing at HLA-A, -B and -DRB1 for potential haploidentical donors is required.
- All diseases listed below are advanced hematologic malignancies not curable by conventional chemotherapy. Responses to conventional treatment range from zero to 30% but are typically short lived.
- Adequate organ function is defined as:
- If recent mold infection e.g. Aspergillus - must have minimum of 30 days of appropriate treatment before bone marrow transplant (BMT) and infection controlled and be cleared by Infectious Disease.
Key exclusion criteria (8 listed)
- Available and clinically suitable 5-6/6 HLA-A, B, DRB1 matched sibling donor
- Pregnant or breastfeeding
- Evidence of HIV infection or known HIV positive serology
- Current active serious infection
- Unless in post-chemotherapy and radioimmunoconjugated antibody induced aplasia, when he/she would be eligible, patients with acute leukemia in morphologic relapse/ persistent disease defined as > 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods) or associated cytogenetic markers that allows morphologic relapse to be distinguished are not eligible.
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 |
|---|---|---|
| FludarabineAlso: FludaraFludarabine 30 mg/m2 IV over 30-60 minutes on days -6 through -2 before transplant. | Drug | Haploidentical stem cell transplant |
| CyclophosphamideCyclophosphamide 14.5 mg/kg IV over 1-2 hours on days -6 and -5 before transplant and Cyclophosphamide 50 mg/kg IV on days 3 and 4 post-transplant. | Drug | Haploidentical stem cell transplant |
| Total Body IrradiationAlso: TBITBI 200cGy on day -1 before transplant. | Radiation | Haploidentical stem cell transplant |
| Haploidentical stem cell transplantAlso: HSCTNon-T-cell depleted bone marrow infusion | Biological | Haploidentical stem cell transplant |
Primary outcome
2 Year Survival
Time frame: 2 years
Percentage of patients that survive 2 years post-transplant
5 secondary outcomes
Number of Patients With Hematopoietic Engraftment
Time frame: 42 days
Number of Patients With Chimerism
Time frame: 100 days
Number of Patients Experiencing Acute Graft-versus-host Disease by 100 Days
Time frame: 100 days
Number of Patients Experiencing Chronic Graft-versus-host Disease by 1 Year
Time frame: 1 year
Number of Patients Experiencing Transplant Related Mortality (TRM)
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Claudio Brunstein, MD principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fludarabine, Cyclophosphamide and Total Body Irradiation in people with Acute Leukemias and Burkitt's Lymphoma, run by Masonic Cancer Center, University of Minnesota 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 28 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: stopped early, as of the sponsor's last update on 26 Dec 2019. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Dec 2019). 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?
2 Year Survival (time frame: 2 years). 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 Dec 2019.