Acute Myeloid Leukemia (AML)Phase 2NCT02626715
Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS
A Phase 2 study testing Reduced-Intensity Conditioning Regimen and Myeloablative Conditioning Regimen in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Randy Windreich at 1 site in United States.
Trial summary
The purpose of this study is to compare safety and efficacy of reduced-intensity conditioning and myeloablative conditioning regimens prior to HSCT in high-risk AML/MDS pediatric and young adult patients. This study investigates the use of two novel conditioning therapies for hematopoietic stem cell transplant (HSCT). Read more about this study
- Who is it for?
- Ages 0 months to 26 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Hematopoietic Stem Cell Transplant (HSCT)
- What is being tested?
- Reduced-Intensity Conditioning Regimen and Myeloablative Conditioning Regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Sep 2015to 12 Apr 2023
- 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 0 months to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Individuals must meet all the following criteria to be eligible for this study.
- Subject, parent, or legal guardian, if applicable, must have given written informed consent. For patients ≤ 17 years of age who are developmentally able, assent or affirmation will be obtained.
- Age 0-26, inclusive, at time of consent.
- Diagnosis of myelodysplastic syndrome or acute myeloid leukemia, either high-risk (defined below), relapsed or primary refractory, MRD-positive without circulating myeloblasts or active extramedullary disease at the time of transplant. Active marrow disease is permitted. High-risk AML features are defined by the following: RAM phenotype; adverse cytogenetic abnormalities of monosomy 5, monosomy 7, 5q deletion, or other unfavorable prognostic markers according to cytogenetics, FISH, or next generation sequencing (NGS); presence of FLT3 positive internal tandem duplication (FLT3/ITD+), particularly high allelic ratio; treatment-related AML; or positive minimal residual disease (MRD) at end of Induction I.
- Stem cell sources include bone marrow, peripheral blood stem cells, or umbilical cord blood. Related bone marrow, peripheral blood stem cell, or cord blood unit: sibling should be HLA-matched at A, B, and DR-B1 loci. Unrelated cord blood unit should be at a minimum of 4/6 matched at antigen level on HLA A and B, and allele level at HLA DR-B1 loci. Unrelated bone marrow or peripheral blood stem cell donor should be HLA allele level matched at DR-B1.
- Minimum pre-freezing cell dose for cord blood units: 3 x 10^7 total nucleated cells/kg and 1.5 x 10^5 CD34+ cells/kg. If this is not attainable, then double cord blood transplant should be considered.
Show all 22Show fewer
- Subject must have adequate performance status: Lansky score ≥60% for patients <16 years, Karnofsky score ≥60% for patients ≥16 years.
- Subject must have adequate pre-transplant organ function to undergo one of the two conditioning regimens, either the myeloablative conditioning (MAC) OR reduced-intensity conditioning (RIC) regimen. If a subject does not meet the following organ function criteria for the MAC regimen, the RIC regimen will be considered if eligibility criteria is met. The RIC regimen may also be considered, regardless of MAC eligibility, if deemed appropriate by the Principal Investigator and/or treating physician.
- Pre-transplant organ function criteria for Myeloablative Conditioning regimen:
- Renal: creatinine clearance or radioisotope GFR ≥70 mL/min/1.73 m2.
- Hepatic: total bilirubin ≤2.0 mg/dL unless the increase in bilirubin is attributable to Gilbert's syndrome; and SGOT (AST), SGPT (ALT), and Alkaline Phosphatase <4 x upper limit of normal (ULN) for age.
- Cardiac: normal cardiac function by echocardiogram or radionuclide scan, as defined by left ventricular ejection fraction at rest >45% or shortening fraction >26%.
- Pulmonary: FEV1, FVC, and DLCO (corrected for hemoglobin) ≥50% of predicted; if unable to perform pulmonary function tests, then oxygen saturation ≥92% on room air.
- OR
- Pre-transplant organ function criteria for Reduced-Intensity Conditioning regimen:
- Renal: creatinine clearance or radioisotope GFR ≥70 mL/min/1.73 m2.
- Hepatic: total bilirubin ≤2.5 mg/dL unless the increase in bilirubin is attributable to Gilbert's syndrome; and SGOT (AST), SGPT (ALT), and Alkaline Phosphatase <5 x upper limit of normal (ULN) for age.
- Cardiac: normal cardiac function by echocardiogram or radionuclide scan, as defined by left ventricular ejection fraction at rest >40% or shortening fraction >26%.
- Pulmonary: FEV1, FVC, and DLCO (corrected for hemoglobin) ≥40% of predicted; if unable to perform pulmonary function tests, then oxygen saturation ≥92% on room air.
- HIV and HTLV negative, by either PCR or serology.
- Negative pregnancy test for females ≥10 years old or who have reached menarche.
- All females of childbearing potential and sexually active males must agree to use an FDA approved method of birth control for up to 12 months after HSCT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause a birth defect.
Who cannot join
- Individuals who meet any of the following criteria are not eligible for this protocol.
- Uncontrolled bacterial, viral, fungal, or other infection at the time of cytoreduction, defined by positive blood cultures and/or fevers >38.0 within 24 hours of start of conditioning therapy.
- Females who are pregnant or who are lactating.
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.
- Additional Exclusion Criteria for Myeloablative Conditioning (MAC) Only Individuals who meet any of the following criteria are not eligible for the MAC regimen.
- Recipient of either an autologous or allogeneic stem cell transplant within 3 months of the start of conditioning.
- Patients with any inherited bone marrow failure syndrome including, but not limited to, Fanconi anemia, Shwachman-Diamond syndrome, dyskeratosis congenita or Down syndrome (defined as either constitutional trisomy 21 or constitutional mosaicism of trisomy 21).
Trial locations and contacts
1 site in 1 country.
United States1 site
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
About this study
Official title: A Phase II Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children and Young Adults With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
The purpose of this study is to compare safety and efficacy of reduced-intensity conditioning and myeloablative conditioning regimens prior to HSCT in high-risk AML/MDS pediatric and young adult patients. This study investigates the use of two novel conditioning therapies for hematopoietic stem cell transplant (HSCT). The primary focus of both the investigators' myeloablative and reduced-intensity conditioning regimens is to reduce overall toxicity so that pediatric and young adult patients with high-risk AML/MDS with significant pretransplant comorbidities who would have been ineligible to proceed to HSCT previously can now receive potentially life-saving treatment.
- Conditions
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Hematopoietic Stem Cell Transplant (HSCT)
- Being tested
- Reduced-Intensity Conditioning Regimen, Myeloablative Conditioning Regimen
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Reduced-Intensity Conditioning RegimenDrug
Also called Campath, Droxia, Fludara, Alkeran, Thiotepa
Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
Myeloablative Conditioning RegimenDrug
Also called Campath, Thiotepa, Fludara, Busulfex
Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
| Study group | Role | What they receive |
|---|---|---|
| Reduced-Intensity Conditioning | Active comparator | Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name) Receives: Drug: Reduced-Intensity Conditioning Regimen |
| Myeloablative Conditioning | Active comparator | Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) , Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name) Receives: Drug: Myeloablative Conditioning Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Number of non-relapsed deaths that occur
Measured: Day 100
Preliminary Efficacy (Event-free Survival at 6 Months) in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Event-free survival at 6 months, where events are defined as relapse or death
Measured: 6 months
Number of Non-relapsed Deaths by 6 Months Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Number of non-relapsed deaths that occur
Measured: Day 180
Secondary outcomes (11)
The Pace of Neutrophil Recovery
Neutrophil Engraftment (the first of three consecutive days in which the absolute neutrophil count (ANC) exceeded 500/mcL)
Measured: Day of transplant to end of study (Day 365)
The Pace of Platelet Recovery
Platelet Engraftment (the first of three consecutive days in which the platelet count exceeded 20,000/mm3 without platelet transfusions for the preceding 7 days)
Measured: Day of transplant to end of study (Day 365)
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
Grade 0: no stage 1-4 of any organ. Grade I: stage 1-2 skin rash (stage 1: <25%, stage 2: 25-50% body surface area affected by maculopapular rash), no gut or liver involvement.
Grade II: stage 3 skin rash (>50% body surface area affected), or stage 1 GI involvement (10-19.9 mL/kg/day volume of diarrhea), or stage 1 liver involvement (2-3 mg/dL total bilirubin level).
Grade III: stage 0-3 skin rash with stage 2-4 GI involvement (stage 2: 20-30 mL/kg/day, stage 3: >30 mL/kg/day volume of diarrhea, stage 4: severe abdominal pain with or without ileus and/or grossly blood stool) or stage 2-3 liver involvement (stage 2: 3-6 mg/dL, stage 3: 6-15 mg/dL total bilirubin level).
Grade IV: stage 4 skin rash (generalized erythroderma with bullous formation) or stage 4 liver involvement (>15 mg/dL total bilirubin level).
Measured: Day of transplant to end of study (Day 365)
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Limited cGVHD: localized skin involvement and/or hepatic dysfunction due to cGVHD.
Extensive cGVHD: one or more of the following: generalized skin involvement; liver histology showing chronic aggressive hepatitis, bridging necrosis, or cirrhosis; involvement of eye, minor salivary glands or oral mucosa based on biopsy, or any other target organ.
Mild cGVHD: 1 or 2 organs involved with no more than score 1 plus lung score 0. Moderate cGVHD: 3 or more organs involved with no more than score 1, or at least one organ (not lung) with score 2, or lung score 1.
Severe cGVHD: at least one organ with score 3, or lung score 2 or 3.
Measured: Day of transplant to end of study (Day 365)
Disease-free Survival (DFS)
The number of subjects who are alive without relapse/leukemia
Measured: Day 100 and 180 post-transplant
Treatment-related Mortality (TRM)
The number of subjects deceased due to transplant-related (i.e. non-relapse) causes
Measured: Day 100 and 180 post-transplant
The Number of Subjects With Overall Survival (OS)
The number of subjects who are alive
Measured: Day 100 and 180 post-transplant
Day 0 Campath (Alemtuzumab) Level
Campath (Alemtuzumab) level measured on the day of transplant
Measured: Day 0
Pace of Immune Reconstitution
Immune recovery as measured by lymphocyte subsets
Measured: Day 180
Incidence of Primary Graft Failure.
The failure to achieve an ANC ≥500/μL after 42 days, determined by three consecutive measurements on different days, and not caused by recurrent leukemia.
Measured: Post-transplant to 42 days post-transplant
Incidence of Grades 4 and 5 Adverse Events
Adverse events as assessed by CTCAE
Measured: Day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2015 (estimated)
- Study start4 Sep 2015
- Primary completion12 Apr 2023
- Study completion12 Apr 2023
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Randy Windreich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Randy Windreich, University of Pittsburgh
- Investigators
- Randy Windreich, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02626715
- Sponsor's protocol ID
- STUDY19030327
- Status last verified
- Jul 2024
- First submitted
- 24 Sep 2015
- Last updated
- 16 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, Thomas ED. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995 Jun;15(6):825-8.
PubMed 7581076Jagasia MH, Greinix HT, Arora M, Williams KM, Wolff D, Cowen EW, Palmer J, Weisdorf D, Treister NS, Cheng GS, Kerr H, Stratton P, Duarte RF, McDonald GB, Inamoto Y, Vigorito A, Arai S, Datiles MB, Jacobsohn D, Heller T, Kitko CL, Mitchell SA, Martin PJ, Shulman H, Wu RS, Cutler CS, Vogelsang GB, Lee SJ, Pavletic SZ, Flowers ME. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2014 Diagnosis and Staging Working Group report. Biol Blood Marrow Transplant. 2015 Mar;21(3):389-401.e1. doi: 10.1016/j.bbmt.2014.12.001. Epub 2014 Dec 18.
PubMed 25529383
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children and Young Adults With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT02626715
- Acronym / sponsor ID
- STUDY19030327
- Conditions
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), Hematopoietic Stem Cell Transplant (HSCT)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Randy WindreichUniversity, hospital or other
- Study start
- 4 Sep 2015 (actual)
- Primary completion
- 12 Apr 2023 (actual)
- Study completion
- 12 Apr 2023 (actual)
- First posted
- 10 Dec 2015 (estimated)
- Results
- Posted 16 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to compare safety and efficacy of reduced-intensity conditioning and myeloablative conditioning regimens prior to HSCT in high-risk AML/MDS pediatric and young adult patients. This study investigates the use of two novel conditioning therapies for hematopoietic stem cell transplant (HSCT). The primary focus of both the investigators' myeloablative and reduced-intensity conditioning regimens is to reduce overall toxicity so that pediatric and young adult patients with high-risk AML/MDS with significant pretransplant comorbidities who would have been ineligible to proceed to HSCT previously can now receive potentially life-saving treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Reduced-Intensity Conditioning | Active comparator | Drug: Reduced-Intensity Conditioning RegimenCampath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide) Trade Name (generic name) |
| Myeloablative Conditioning | Active comparator | Drug: Myeloablative Conditioning RegimenCampath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) , Fludara (fludarabine), Busulfex (busulfan) Trade Name (generic name) |
Who may qualify
- Ages
- 0 months to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (22 listed)
- Individuals must meet all the following criteria to be eligible for this study.
- Subject, parent, or legal guardian, if applicable, must have given written informed consent. For patients ≤ 17 years of age who are developmentally able, assent or affirmation will be obtained.
- Age 0-26, inclusive, at time of consent.
- Diagnosis of myelodysplastic syndrome or acute myeloid leukemia, either high-risk (defined below), relapsed or primary refractory, MRD-positive without circulating myeloblasts or active extramedullary disease at the time of transplant. Active marrow disease is permitted. High-risk AML features are defined by the following: RAM phenotype; adverse cytogenetic abnormalities of monosomy 5, monosomy 7, 5q deletion, or other unfavorable prognostic markers according to cytogenetics, FISH, or next generation sequencing (NGS); presence of FLT3 positive internal tandem duplication (FLT3/ITD+), particularly high allelic ratio; treatment-related AML; or positive minimal residual disease (MRD) at end of Induction I.
- Stem cell sources include bone marrow, peripheral blood stem cells, or umbilical cord blood. Related bone marrow, peripheral blood stem cell, or cord blood unit: sibling should be HLA-matched at A, B, and DR-B1 loci. Unrelated cord blood unit should be at a minimum of 4/6 matched at antigen level on HLA A and B, and allele level at HLA DR-B1 loci. Unrelated bone marrow or peripheral blood stem cell donor should be HLA allele level matched at DR-B1.
Key exclusion criteria (7 listed)
- Individuals who meet any of the following criteria are not eligible for this protocol.
- Uncontrolled bacterial, viral, fungal, or other infection at the time of cytoreduction, defined by positive blood cultures and/or fevers >38.0 within 24 hours of start of conditioning therapy.
- Females who are pregnant or who are lactating.
- Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose additional risks from participation in the study, may interfere with the participant's ability to comply with study requirements or that may impact the quality or interpretation of the data obtained from the study.
- Additional Exclusion Criteria for Myeloablative Conditioning (MAC) Only Individuals who meet any of the following criteria are not eligible for the MAC regimen.
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 |
|---|---|---|
| Reduced-Intensity Conditioning RegimenAlso: Campath, Droxia, Fludara, Alkeran, ThiotepaCampath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent | Drug | Reduced-Intensity Conditioning |
| Myeloablative Conditioning RegimenAlso: Campath, Thiotepa, Fludara, BusulfexCampath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent | Drug | Myeloablative Conditioning |
Primary outcome
Number of Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Time frame: Day 100
Number of non-relapsed deaths that occur
Preliminary Efficacy (Event-free Survival at 6 Months) in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Time frame: 6 months
Event-free survival at 6 months, where events are defined as relapse or death
Number of Non-relapsed Deaths by 6 Months Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
Time frame: Day 180
Number of non-relapsed deaths that occur
11 secondary outcomes
The Pace of Neutrophil Recovery
Time frame: Day of transplant to end of study (Day 365)
The Pace of Platelet Recovery
Time frame: Day of transplant to end of study (Day 365)
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
Time frame: Day of transplant to end of study (Day 365)
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Time frame: Day of transplant to end of study (Day 365)
Disease-free Survival (DFS)
Time frame: Day 100 and 180 post-transplant
Treatment-related Mortality (TRM)
Time frame: Day 100 and 180 post-transplant
The Number of Subjects With Overall Survival (OS)
Time frame: Day 100 and 180 post-transplant
Day 0 Campath (Alemtuzumab) Level
Time frame: Day 0
Pace of Immune Reconstitution
Time frame: Day 180
Incidence of Primary Graft Failure.
Time frame: Post-transplant to 42 days post-transplant
Incidence of Grades 4 and 5 Adverse Events
Time frame: Day 180
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 7581076Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, Thomas ED. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995 Jun;15(6):825-8.
- Background: PubMed 25529383Jagasia MH, Greinix HT, Arora M, Williams KM, Wolff D, Cowen EW, Palmer J, Weisdorf D, Treister NS, Cheng GS, Kerr H, Stratton P, Duarte RF, McDonald GB, Inamoto Y, Vigorito A, Arai S, Datiles MB, Jacobsohn D, Heller T, Kitko CL, Mitchell SA, Martin PJ, Shulman H, Wu RS, Cutler CS, Vogelsang GB, Lee SJ, Pavletic SZ, Flowers ME. National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: I. The 2014 Diagnosis and Staging Working Group report. Biol Blood Marrow Transplant. 2015 Mar;21(3):389-401.e1. doi: 10.1016/j.bbmt.2014.12.001. Epub 2014 Dec 18.
- Individual participant dataSharing planned: No
Related trials for Acute Myeloid Leukemia (AML)
RecruitingPhase 3
Comparison of VA and D/IA Induction Regimens in Elderly Fit Acute Myeloid Leukemia Patients
AML, Adult
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
Not yet recruitingPhase 1/2
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Active, not recruitingPhase 2
Cord Blood Transplant in Adults With Blood Cancers
Acute Myelogenous Leukemia (AML), Acute Lymphoblastic Leukemia (ALL)
RecruitingPhase 1
CER-1236 in Patients With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myelofibrosis (MF)
AML, Acute Myeloid Leukemia
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.
Randy Windreich, MD principal investigator
University of Pittsburgh
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 Reduced-Intensity Conditioning Regimen and Myeloablative Conditioning Regimen in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Randy Windreich 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 22 inclusion and 7 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: finished, as of the sponsor's last update on 16 Jul 2024. 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 16 Jul 2024). 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 Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS. (time frame: Day 100). The study lists 3 primary outcomes in all. 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 NCT02626715, which also lists 2 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 16 Jul 2024; AzTrials last refreshed its copy on 26 Oct 2026.