Clinical trial NCT00916058: Conditioning Regimen of Bendamustine and Melphalan Followed by Transplant in Patients With Multiple Myeloma
Official title: A Phase 1-2 Study of a Novel Conditioning Regimen of Bendamustine and Melphalan Followed by Autologous Stem Cell Transplant for Patients With Multiple Myeloma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Multiple Myeloma
Keywords: Multiple Myeloma
Autologous Stem Cell Transplant
ASCT
MM
Lead sponsor: Weill Medical College of Cornell University
Sponsor class: OTHER
First posted: 2009-06-09
Enrollment: 57
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety and efficacy of Bendamustine (TREANDA™), in combination with Melphalan in subjects with multiple myeloma who are undergoing an Autologous Stem Cell Transplant.

Detailed description: Bendamustine (TREANDA™) has been used in clinical trials to treat multiple myeloma. The results from these trials suggest that it may be beneficial in the treatment of multiple myeloma in a different treatment context. Researchers aim to determine if there may be an improved benefit in the context of bone marrow transplant. This initial clinical trial is intended to help determine how safe it is to use bendamustine as a conditioning regimen for bone marrow transplant, and to look for any initial evidence of benefit.

Bendamustine (TREANDA™) is approved by the Food and Drug Administration (FDA) for the treatment of Chronic Lymphocytic Leukemia and Melphalan is a type of chemotherapy drug.

The use of Melphalan alone as a conditioning regimen for Autologous Stem Cell Transplant is considered "Standard of Care," that is, the treatment or process that your doctor would normally follow to treat your disease. Although Bendamustine (TREANDA™) has been used in multiple myeloma research studies, the combination of Bendamustine (TREANDA™) and Melphalan as treatment for Multiple Myeloma is not approved by the FDA, thus the combination therapy used in this research study is considered "investigational."

Eligibility criteria: Inclusion Criteria:

* Patients with multiple myeloma who have received induction therapy and have had stem cells mobilized in preparation for autologous transplantation will be eligible for this study. Patients are also eligible with relapsed or refractory disease, after attempts at more standard approaches, and with the availability of stem cells.
* Patients must be age 18 or older.
* Patients must have a life expectancy of at least 12 weeks.
* Patients must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
* Patients must provide written informed consent.

Exclusion Criteria:

* Impaired renal function with a measured or calculated creatinine clearance of less than 25 ml/min.
* Impaired hepatic function defined as a bilirubin greater than 1.5 x upper limit of normal (ULN) or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 5 x ULN.
* Serious active or uncontrolled infection or medical condition.
* Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
* Impaired pulmonary function with a diffusing capacity of the lung for carbon monoxide (DLCO) less than 45% predicted.
* Impaired cardiac function with an ejection fraction less than 40% of predicted.
* Other systemic anticancer therapy or ongoing toxicities from such therapy.

Primary outcomes:
measure: Maximum Tolerated Dose (Phase 1)
timeFrame: 35 days post-transplant
measure: Overall Response Rate (Phase 2) - Number of Participants Achieving at Least a Partial Response or Better in Disease Status at Day 100 Post-transplant
timeFrame: 100 days post-transplant
Secondary outcomes:
measure: Progression-Free Survival (Phase 1)
timeFrame: From Day 0 to first incidence of disease progression, up to 1,128 days
measure: Progression-Free Survival (Phase 2)
timeFrame: From Day 0 to first incidence of disease progression, up to 86 months
measure: Overall Survival at 2 Years (Phase 1)
timeFrame: From Day 0 until time of death, assessed up to 2 years.
measure: Overall Survival at 3 Years (Phase 2)
timeFrame: From Day 0 until time of death, assessed up to 3 years.
Other outcomes:
Locations:
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
Study officials:
affiliation: Weill Medical College of Cornell University
name: Tsiporah Shore, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Gomez-Arteaga A, Mark TM, Guarneri D, Christos PJ, Gergis U, Greenberg JD, Hsu J, Mayer SA, Niesvizky R, Pearse RN, Phillips AA, Rossi A, Coleman M, van Besien K, Shore TB. High-dose bendamustine and melphalan conditioning for autologous stem cell transplantation for patients with multiple myeloma. Bone Marrow Transplant. 2019 Dec;54(12):2027-2038. doi: 10.1038/s41409-019-0587-0. Epub 2019 Jun 12.