Clinical trial · Interventional
Allogeneic Stem Cell Transplantation for Multiple Myeloma and Myelofibrosis
Busulfan, Fludarabine, Donor Stem Cell Transplant, and Cyclophosphamide in Treating Participants With Multiple Myeloma or Myelofibrosis
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Slow accrual
Summary
Brief summary (as posted)
This phase II trial studies how well busulfan, fludarabine, donor stem cell transplant, and cyclophosphamide in treating participants with multiple myeloma or myelofibrosis. Drugs used in chemotherapy, such as busulfan, fludarabine, and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the participant they may help the participant's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving busulfan and fludarabine before and cyclophosphamide after donor stem cell may work better in treating participants with multiple myeloma or myelofibrosis.
Conditions
Conditions (10)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Anemia | — | UNRESOLVED | — |
| ASXL1 Gene Mutation | — | UNRESOLVED | — |
| EZH2 Gene Mutation | — | UNRESOLVED | — |
| IDH1 Gene Mutation | — | UNRESOLVED | — |
| IDH2 Gene Mutation | — | UNRESOLVED | — |
| Plasma Cell Myeloma | Multiple Myeloma | CURATED_BROADER | 0.80 |
| Primary Myelofibrosis | Primary Myelofibrosis | ONTOLOGY_EXACT | 0.98 |
| Recurrent Plasma Cell Myeloma | Multiple Myeloma | CURATED_BROADER | 0.80 |
| Secondary Myelofibrosis | — |
Interventions
Interventions (5)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Busulfan | Drug | Busulfan | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| Hematopoietic Cell Transplantation | Procedure | — | UNRESOLVED |
| Laboratory Biomarker Analysis | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Treatment (busulfan, fludarabine, HSCT, cyclophosphamide)
- description
- Participants receive busulfan IV over 2 hours and fludarabine IV over 30 minutes on days -5 to -2. Participants undergo HSCT on day 0. Participants then receive cyclophosphamide IV over 60 minutes on days 3 and 4.
- interventionNames
- Drug: Busulfan
- Drug: Cyclophosphamide
- Drug: Fludarabine
- Procedure: Hematopoietic Cell Transplantation
- Other: Laboratory Biomarker Analysis
Primary outcomes (1)
- measure
- Non-relapse Mortality (NRM) at Day 100
- timeFrame
- Up to day 100
- description
- NRM is defined as death due to GVHD, infections, sepsis, organ (lung, liver, kidney) toxicity. Death from underlying disease (i.e. progression or relapse) is not considered NRM.
Secondary outcomes (7)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 75 Years
Show eligibility criteria text
Inclusion Criteria:
* Eastern Cooperative Oncology Group (ECOG) performance status 0-1
* Participants must have one of the following diagnoses of multiple myeloma (MM) or primary/secondary myelofibrosis (MF)
* Participants must have histologically documented multiple myeloma (MM)
* Participants 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)
* Participants must have histologically documented myelofibrosis (MF)
* Participants with Dynamic International Prognostic Scoring System (DIPSS) plus intermediate stage 2 or higher risk MF; OR
* Subset of intermediate stage 1 participants; 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
* Participants 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 contraceptionReferences
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