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

• Status: Recruiting
• Trial end date: 2023-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Boston Medical Center; Shayna Sarosiek
• Collaborator: Spectrum Pharmaceuticals, Inc
• Treatments: Melphalan

Criteria

Inclusion Criteria:

• Age 18 years or older

• Eastern Cooperative Oncology Group Performance Status 0-2

• Histologic diagnosis of primary systemic (AL) amyloidosis based on:

• Deposition of amyloid material by Congo red stain showing characteristic apple green birefringence AND

• Evidence of a clonal plasma cell dyscrasia with monoclonal protein in the serum or urine by immunofixation electrophoresis AND/OR abnormal serum free light chain assay AND/OR clonal plasma cells in the bone marrow exam demonstrated by immunohistochemistry, flow cytometry, or in situ hybridization AND

• Evidence of organ involvement

• Eligible for treatment with high dose melphalan and stem cell transplantation per institutional guidelines

• Ability to understand and willingness to sign informed consent

• Pulmonary Function Test demonstrating a diffusion capacity of lung for carbon monoxide ≥ 50%

• Left ventricular ejection fraction ≥40%

• Systolic blood pressure >90 mm Hg (supine position)

• Eastern Cooperative Oncology Group Performance status of 2 or better (unless patient is diagnosed with AL amyloidosis involving the gastrointestinal and peripheral/autonomic nervous systems, then performance status of 3 is acceptable)

Exclusion Criteria:

• Previous high-dose melphalan and stem cell transplant

• Previous total cumulative dose of oral melphalan > 300 mg

• Cytotoxic chemotherapy within the previous 28 days

• New York Heart Association ≥3

• Decompensated or uncontrolled heart failure

• Oxygen dependence

• epidermal growth factor receptor < 30 ml/min

• Active infection (i.e HIV, Hepatitis B or C)

• Pregnancy or breastfeeding

• Exposure to another investigational drug within 3-4 weeks prior to start of study treatment

• Ongoing alcohol or drug addiction

• Unable or unwilling to comply with the protocol