AL Amyloidosis Clinical Trial

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.

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Full 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.

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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

Study is for people with:

AL Amyloidosis

Phase:

Phase 2

Estimated Enrollment:

27

Study ID:

NCT02994784

Recruitment Status:

Terminated

Sponsor:

Boston Medical Center

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There is 1 Location for this study

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Boston Medical Center
Boston Massachusetts, 02118, United States

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Study is for people with:

AL Amyloidosis

Phase:

Phase 2

Estimated Enrollment:

27

Study ID:

NCT02994784

Recruitment Status:

Terminated

Sponsor:


Boston Medical Center

How clear is this clinincal trial information?

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