Clinical trial NCT03303950 (phase 2, status: terminated)
Conditions: anemia, asxl1 gene mutation, ezh2 gene mutation, idh1 gene mutation, idh2 gene mutation, plasma cell myeloma, primary myelofibrosis, recurrent plasma cell myeloma, secondary myelofibrosis, thrombocytopenia
ICD codes: D53.2, D64.9, D46.4, D53.0, D53.9, D61.3, D61.9, D68.52, J84.83, D68.52, J84.83, D68.52, J84.83, D68.52, J84.83, D75.81, C94.41, C94.42, C94.40, G47.13, J01.41, K11.22, K12.0, N96, F33.8, G03.2, D75.81, C94.41, C94.42, C94.40, D69.6, D69.49, D69.59, P61.0, D75.82, D69.42, O36.8211
Drugs: busulfan, cyclophosphamide, fludarabine
SMILES: CS(=O)(=O)OCCCCOS(C)(=O)=O, ClCCN(CCCl)P1(=O)NCCCO1, ClCCN(CCCl)P1(=O)NCCCO1
Reason stopped: 
    slow accrual
  
Eligibility criteria:

        Inclusion Criteria:

          -  Eastern Cooperative Oncology Group (ECOG) performance status 0-1

          -  Patients must have one of the following diagnoses of multiple myeloma (MM) or
             primary/secondary myelofibrosis (MF)

          -  Patients must have histologically documented multiple myeloma (MM)

               -  Patients in early relapse (less than 24 months from initiation of systemic
                  anti-myeloma therapy which may include single or planned tandem autologous
                  transplant) after primary therapy that included and autologous HSCT; OR

               -  Later stage; OR

               -  High risk factors defined by the presence of any one of the following detected at
                  any time prior to enrollment: deletion of chromosome 13 by conventional
                  cytogenetics, hypodiploidy, abnormality in chromosome 1 (1q amplification or 1p
                  deletion), t(4;14), t(14;16), t(14;20) or deletion of 17p by fluorescence in situ
                  hybridization (FISH) or conventional karyotyping; high risk criteria based on
                  commercially available gene expression profiling; OR

               -  Extramedullary disease, plasma cell leukemia or high lactate dehydrogenase (LDH)

          -  Patients must have histologically documented myelofibrosis (MF)

               -  Patients with Dynamic International Prognostic Scoring System (DIPSS) plus
                  intermediate stage 2 or higher risk MF; OR

               -  Subset of intermediate stage 1 patients; defined by:

                    -  Poor-risk molecular profile (triple negative: JAK2, CALR, MPL); OR

                    -  Presence of any of the following mutations: ASXL1, SRSF2, EZH2, IDH1/2; OR

                    -  Severe thrombocytopenia, severe anemia, high peripheral blood blasts
                       percentage; OR

                    -  Unfavorable cytogenetic abnormalities (rearrangements of chromosome 5 or 7
                       or >= 3 abnormalities

          -  Able to provide informed consent and willing to sign an approved consent form that
             conforms to federal and institutional guidelines

          -  DONOR: A related donor - fully matched

          -  DONOR: A related donor - haploidentical

          -  DONOR: An unrelated donor - fully matched

          -  DONOR: An unrelated donor -9/10 matched

        Exclusion Criteria:

          -  Cardiac-left ventricular ejection fraction < 40%, symptomatic coronary artery disease,
             or uncontrolled arrhythmias

          -  Pulmonary-forced expiratory volume at one second (FEV1) or diffusion capacity of lung
             for carbon dioxide (DLCO) < 40% or history of chronic use of supplemental oxygen.
             Temporary use of supplemental oxygen at the time of screening or registration is
             allowed if the investigator feels that the underlying cause of requiring oxygen is
             reversible by the time treatment begins.

          -  Renal-calculated or measured glomerular filtration rate (GFR) < 30 ml/min,
             dialysis-dependent, or history of renal transplant

          -  Hepatic-bilirubin > 2 X upper limit of normal (ULN)

          -  Alanine aminotransferase (ALT) > 2.5 X ULN or cirrhosis

          -  Patients with active or uncontrolled bacterial, viral, or fungal infections requiring
             systemic therapy

          -  Pregnant women, nursing mothers or women of child-bearing potential who are unwilling
             to use medically accepted methods of contraception

          -  Male and female subjects not willing to agree to medically accepted methods of
             contraception