Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients
Condition: Amyloidosis; Systemic
MeSH terms: Immunoglobulin Light-chain Amyloidosis; Amyloidosis
Intervention: Propylene Glycol-Free Melphalan Hydrochloride
Intervention description: Intravenous Propylene Glycol-Free Melphalan Hydrochloride
Intervention MeSH terms: Melphalan
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 27.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

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.
    

Eligibility 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