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

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

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

Brief 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 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.

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.


Sponsor: Shayna Sarosiek

Current Primary Outcome: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 100 days ]

To determine the safety profile of propylene glycol-free melphalan hydrochloride in the conditioning regimen prior to autologous stem cell transplantation in AL amyloidosis patients, including adverse events related to renal dysfunction (was acute renal failure is defined as either a >/=1 mg/dL increase in serum creatinine or a doubling of serum creatinine to >/=1.5 mg/dL for at least 2 days.), cardiac dysfunction (new arrhythmia), or autonomic dysfunction (decline in sitting systolic blood pressure of ≥20mm Hg compared to baseline)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Assess time to neutrophil engraftment [ Time Frame: 3 weeks ]
  • Assess time to platelet engraftment [ Time Frame: 3 weeks ]
  • Assess treatment related mortality [ Time Frame: 100 days ]
    Number of patients who expire within 100 days of transplant
  • Assess hematologic overall response rate [ Time Frame: 6 months ]
    Number of patients with response based on Gertz, Palladini criteria
  • Assess organ response [ Time Frame: 12 months ]
    Number of patients with organ response based on Gertz criteria
  • Monitor number of hospitalizations [ Time Frame: 100 days ]
    Number of hospitalizations per patient


Original Secondary Outcome: Same as current

Information By: Boston Medical Center

Dates:
Date Received: November 18, 2016
Date Started: January 2017
Date Completion: January 2021
Last Updated: January 10, 2017
Last Verified: January 2017