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Clinical Trial Summary

This phase I trial studies the best dose and side effects of mephalan in treating patients with multiple myeloma who are undergoing stem cell transplant. Chemotherapy drugs, such as mephalan, 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.


Clinical Trial Description

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 CMMCs numbers obtained with CELLSEARCH with 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04483206
Study type Interventional
Source Emory University
Contact Craig Hofmeister, MD, MPH
Phone 404-778-1900
Email craig.hofmeister@emory.edu
Status Recruiting
Phase Phase 1
Start date May 20, 2021
Completion date December 31, 2026

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