NCT03417284

Melphalan Hydrochloride in Treating Participants With Newly-Diagnosed Multiple Myeloma Undergoing Donor Stem Cell Transplantation

Prospective Phase I/II Trial to Jointly Optimize the Administration Schedule(s) and Dose(s) of Melphalan for Injection (Evomela) as a Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Patients With Newly Diagnosed Multiple Myeloma

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This phase I/II trial studies the side effects and best dose of melphalan hydrochloride in treating participants with newly-diagnosed multiple myeloma who are undergoing a donor stem cell transplantation. Giving chemotherapy before a donor stem cell transplantation 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 melphalan hydrochloride before a donor stem cell transplantation may work better than standard chemotherapy in helping to prevent multiple myeloma from coming back.

Detailed description

PRIMARY OBJECTIVES: I. To determine the optimal dose and schedule of melphalan for injection (melphalan hydrochloride \[Evomela\]) prior to autologous hematopoietic stem cell transplantation (auto-HCT) for multiple myeloma (MM). II. To collect the pharmacokinetic data and compare the exposure-response evaluations between the 2 infusion schedules. SECONDARY OBJECTIVES: I. To determine the incidence of treatment related mortality (TRM) at day 90 after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela. II. To determine the rate of minimal residual disease (MRD) negative complete response (CR) rate at day 90 after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela. III. To determine the progression-free survival (PFS) after auto-HCT in newly diagnosed myeloma patients treated on different schedules and doses of Evomela. OUTLINE: This is a phase I, dose escalation study of melphalan hydrochloride followed by a phase II study. PREPARATIVE REGIMEN: Participants are randomized to 1 of 2 groups. GROUP 1: Participants receive melphalan hydrochloride intravenously (IV) over 30-60 minutes on day -2. GROUP 2: Participants receive melphalan hydrochloride IV over 8-9 hours on day -2. TRANSPLANT: Participants in both groups undergo donor stem cell transplantation IV on day 0 over 30-60 minutes. POST-TRANSPLANT: Participants in both groups receive filgrastim-sndz subcutaneously (SC) once daily (QD) starting on day 5 and continuing in the absence of disease progression, unacceptable toxicity, or until evidence of an absolute neutrophil count (ANC) of 0.5 x 10\^9/L. After completion of study treatment, participants are followed up at 3 months, every 3 months for 1 year, and then annually for 1 year.

Conditions

• Plasma Cell Myeloma

Interventions

Type	Name	Description
PROCEDURE	Autologous Hematopoietic Stem Cell Transplantation	Undergo HSCT
BIOLOGICAL	Filgrastim-sndz	Given SC
DRUG	Melphalan Hydrochloride	Given IV

Timeline

• Start date: 2019-10-09
• Primary completion: 2024-07-18
• Completion: 2024-07-18
• First posted: 2018-01-31
• Last updated: 2025-05-01
• Results posted: 2025-05-01

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study