Melphalan before Stem Cell Transplant for the Treatment of Multiple Myeloma

Active:	Yes
Cancer Type:	Multiple Myeloma Plasma cell neoplasm	NCT ID:	NCT04483206
Trial Phases:	Phase I	Protocol IDs:	WINSHIP5001-20 (primary) NCI-2020-04920 STUDY00000449
Eligibility:	18 Years and older, Male and Female	Study Type:	Treatment
Study Sponsor:	Emory University Hospital/Winship Cancer Institute
NCI Full Details:	http://clinicaltrials.gov/show/NCT04483206

Summary

This phase I trial studies the best dose and side effects of melphalan in treating patients with multiple myeloma who are undergoing stem cell transplant. Chemotherapy drugs, such as melphalan, 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. This trial uses a new method of dosing that is based on analysis of each individual’s blood levels of melphalan after receiving a part of the dose, termed pharmacokinetic analysis. This may help to learn more about how to dose melphalan correctly and which patients are likely to benefit from a personalized dose.

Objectives

PRIMARY OBJECTIVES:
I. Measure achievement of target melphalan systemic exposure in the first 20 patients.
II. Identify safety and preliminary efficacy within each systemic exposure range of melphalan using a 5+5 design.

SECONDARY OBJECTIVES:
I. Describe International Myeloma Working Group response levels and selected grade 3/4 toxicities in an expansion set of patients at the recommended phase 2 area under the curve (AUC) range.
II. Measure deoxyribonucleic acid (DNA) repair score from formalin-fixed paraffin-embedded diagnostic bone marrow sample (FFPE) and from pretransplant marrow aspirate sample.
III. Assess melphalan-induced DNA damage in peripheral blood mononuclear cells (PBMCs) from melphalan-treated patients.
IV. To correlate peripheral blood circulating multiple myeloma cells (CMMCs) numbers obtained with (CELLSEARCH) with minimal residual disease (MRD) assessment at day +90.

OUTLINE: This is a dose-escalation study.

Patients receive standard of care high dose melphalan hydrochloride intravenously (IV) over 30 minutes on day -3 and PK-directed melphalan hydrochloride IV over 30 minutes on day -1. Patients then undergo autologous stem cell transplantation (ASCT) on day 0.

After completion of study treatment, patients are followed up at 7, 14, 30, 60, and 90 days.

**Clinical trials are research studies that involve people. These studies test new ways to prevent, detect, diagnose, or treat diseases. People who take part in cancer clinical trials have an opportunity to contribute to scientists’ knowledge about cancer and to help in the development of improved cancer treatments. They also receive state-of-the-art care from cancer experts... Click here to learn more about clinical trials.