Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients

Summary

This is a single arm, open label study designed to evaluate the safety and efficacy of propylene glycol-free melphalan hydrochloride in patients with AL amyloidosis. Treatment will be comprised of propylene glycol-free melphalan hydrochloride administered intravenously at a dose of 70-100 mg/m2/day on Days -3 and -2 as conditioning prior to autologous stem cell transplantation.

Timeline

Start

2018-01-08

Primary completion

2022-07-01

Completion

2022-07-01

Outcome

Outcome not reported

Stopped (Enrollment): “Slow Accrual during COVID pandemic, alternative treatments available. Study halted early.”

Publications

• Background Palladini G, Dispenzieri A, Gertz MA, Kumar S, Wechalekar A, Hawkins PN, Schonland S, Hegenbart U, Comenzo R, Kastritis E, Dimopoulos MA, Jaccard A, Klersy C, Merlini G. New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers: impact on survival outcomes. J Clin Oncol. 2012 Dec 20;30(36):4541-9. doi: 10.1200/JCO.2011.37.7614. Epub 2012 Oct 22.
• Sarosiek S, Lee MH, Doros G, Edwards CV, Quillen K, Brauneis D, Shelton AC, Sanchorawala V, Sloan JM. Safety and Efficacy of Propylene Glycol-Free Melphalan in Patients with AL Amyloidosis Undergoing Autologous Stem Cell Transplantation: Results of a Phase II Study. Transplant Cell Ther. 2023 Nov;29(11):695.e1-695.e7. doi: 10.1016/j.jtct.2023.08.018. Epub 2023 Aug 20.

Drugs

Indications