NCT02994784

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

A Phase II Single-Center, Open-Label, Safety and Efficacy Study of Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients Undergoing Autologous Stem Cell Transplantation

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Boston Medical Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

After giving written informed consent, subjects will be evaluated for eligibility for enrollment in the study. Baseline evaluations will be performed as outlined in Section 7. Subjects who satisfy all inclusion and exclusion criteria will begin the study drug. Subjects will be monitored from the time of the medication administration until discharge from the transplant program for safety. Organ function and hematologic status will also be measured at 6 and 12 month follow-up visits. Standard response criteria for AL amyloidosis hematologic and organ response will be used. Overall response rate will be measured and participants will be categorized into complete response, very good partial response, partial response and progressive disease. Progression free survival, organ response, and safety and tolerability of propylene glycol-free melphalan hydrochloride will be assessed.

Conditions

• Amyloidosis; Systemic

Interventions

Type	Name	Description
DRUG	Propylene Glycol-Free Melphalan Hydrochloride	Intravenous Propylene Glycol-Free Melphalan Hydrochloride

Timeline

• Start date: 2018-01-08
• Primary completion: 2022-07-01
• Completion: 2022-07-01
• First posted: 2016-12-16
• Last updated: 2023-05-24
• Results posted: 2023-05-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study