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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 12 December 2020
Main ID:  NCT03192397
Date of registration: 16/06/2017
Prospective Registration: Yes
Primary sponsor: Roswell Park Cancer Institute
Public title: Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant
Scientific title: A Phase II Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell Transplantation
Date of first enrolment: August 3, 2017
Target sample size: 30
Recruitment status: Recruiting
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Name:     Roswell Park
Telephone: 877-275-7724
Name:     Christine Ho
Affiliation:  Roswell Park Cancer Institute
Key inclusion & exclusion criteria

Inclusion Criteria:

- The patient must have a diagnosis of one of the following (one must be yes):

- Acute myeloid leukemia (AML)

- Acute lymphoblastic leukemia (ALL)

- Chronic myelogenous leukemia (CML) (chronic phase intolerant or unresponsive to
tyrosine kinase inhibitors, accelerated phase, history of blast crisis)

- Myelodysplastic syndrome (MDS)

- Myeloproliferative neoplasm (MPN)

- Chronic myelomonocytic leukemia (CMML)

- Non-Hodgkin lymphoma (NHL)

- Hodgkin lymphoma (HL) (received and failed frontline therapy or failed autologous
transplantation or inability to collect enough peripheral blood stem cells [PBSC]
for autologous hematopoietic cell transplant [auto-HCT])

- Multiple myeloma (MM)

- Waldenstrom's macroglobulinemia

- Severe aplastic anemia

- Histocompatible donor identified:

- Related donor or unrelated donor matched 5/6 or better (A, B, DRB1)

- Patients with benign hematological disorders such as severe aplastic anemia do not
have disease requirements. Patients with malignant hematologic disorder must be in CR
(MRD is allowed) with the exception of the following:

- Patients with MDS/MPN only require <5% myeloblast on bone marrow evaluation.

- Patients with AML or ALL may be in CRi, patients with MM may be in VGPR

- Have a Karnofsky performance status score of > 50%

- Diffusing capacity of the lung for carbon monoxide (DLCO) > 40% predicted, corrected
for hemoglobin and/or alveolar ventilation

- Left ventricular ejection fraction > 40%

- Bilirubin =< 3 x upper limit of normal

- Liver alkaline phosphatase =< 3 x upper limit of normal

- Serum glutamic-oxaloacetic transaminase (SGOT) or serum glutamate pyruvate
transaminase (SGPT) =< 3 x upper limit of normal

- Calculated creatinine clearance > 40 cc/min by the modified Cockroft-Gault formula

- Patient must be cleared pre-transplant by Radiation Oncology to be able to receive 400

- Participants of child-bearing potential must agree to use adequate contraceptive
methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study
entry; should a woman become pregnant or suspect she is pregnant while she or her
partner is participating in this study, she should inform her treating physician

- Patients who have failed a prior autologous or allogeneic transplant are eligible;
however, at least 6 months must have elapsed between the start of this reduced
intensity conditioning regimen and the last transplant if patient had a prior
autologous or myeloablative allogeneic bone marrow transplant (BMT)

- At least 2 weeks since prior chemotherapy, radiation treatment and/or surgery

- Participant or legal representative must understand the investigational nature of this
study and sign an Independent Ethics Committee/Institutional Review Board approved
written informed consent form prior to receiving any study related procedure

Exclusion Criteria:

- Bone marrow failure disorders:

- Paroxysmal nocturnal hemoglobinuria (PNH)

- Hereditary bone marrow failure disorders include Diamond-Blackfan anemia,
Shwachman- Diamond syndrome, Kostmann syndrome, and congenital amegakaryocytic

- Other non-malignant hematologic or immunologic disorders that require

- Quantitative or qualitative congenital platelet disorders (including but not
limited to congenital megakaryocytopenia, absent-radii syndrome, Glanzmann?s

- Quantitative or qualitative congenital neutrophil disorders (including but
not limited to chronic granulomatous disease, congenital neutropenia)

- Congenital primary immunodeficiencies (including but not limited to severe
combined immunodeficiency syndrome, Wiskott-Aldrich syndrome, CD40 ligand
deficiency, T-cell deficiencies)

- Hemoglobinopathies (including sickle cell disease and thalassemia)

- Presence of human leukocyte antigen (HLA) antibodies

- Uncontrolled central nervous system (CNS) disease (for hematologic malignancies)

- Child-Pugh class B and C liver failure

- Patients who in the opinion of the treating physician are unlikely to comply with the
restrictions of allogeneic stem cell transplantation based on formal psychosocial
screening. (i.e., serious, uncontrolled psychiatric illness/social situations that
would limit compliance with study requirements)

- Uncontrolled diabetes mellitus, cardiovascular disease, active serious infection or
other condition which, in the opinion of treating physician, would make this protocol
unreasonably hazardous for the patient

- Known human immunodeficiency virus (HIV) positive

- Pregnant or nursing female participants

- Unwilling or unable to follow protocol requirements

- Any condition which in the Investigator?s opinion deems the participant an unsuitable
candidate to receive study drug

Age minimum: 18 Years
Age maximum: 79 Years
Gender: All
Health Condition(s) or Problem(s) studied
Acute Myeloid Leukemia in Remission
Adult Acute Lymphoblastic Leukemia in Complete Remission
Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission
Chronic Myelomonocytic Leukemia in Remission
Graft Versus Host Disease
Hodgkin Lymphoma
Minimal Residual Disease
Myelodysplastic Syndrome
Myeloproliferative Neoplasm
Non-Hodgkin Lymphoma
Plasma Cell Myeloma
Severe Aplastic Anemia
Waldenstrom Macroglobulinemia
Drug: Cyclophosphamide
Drug: Fludarabine Phosphate
Drug: Melphalan Hydrochloride
Drug: Mycophenolate Mofetil
Drug: Sirolimus
Other: Laboratory Biomarker Analysis
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Radiation: Total-Body Irradiation
Primary Outcome(s)
Extensive chronic graft versus host disease (GVHD) [Time Frame: Up to 365 days]
Secondary Outcome(s)
Clinical response assessed as per bone marrow transplant (BMT) standard of care [Time Frame: Up to 4 years]
Cumulative incidence of grade III-IV acute graft versus host disease (GVHD) [Time Frame: Up to 4 years]
Cumulative incidence of relapse [Time Frame: Up to 4 years]
Engraftment rate assessed as per bone marrow transplant (BMT) standard of care [Time Frame: Up to 4 years]
Overall survival assessed as per bone marrow transplant (BMT) standard of care [Time Frame: Up to 4 years]
Progression free survival (PFS) assessed as per bone marrow transplant (BMT) standard of care [Time Frame: At 1 year]
Treatment-related mortality rates [Time Frame: Up to 4 years]
Secondary ID(s)
I 44417
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
National Cancer Institute (NCI)
Ethics review
Results available:
Date Posted:
Date Completed:
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