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

  • STATUS
    Recruiting
  • End date
    Jan 24, 2023
  • participants needed
    43
  • sponsor
    Boston Medical Center
Updated on 24 March 2022

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.

Description

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.

Details
Condition Amyloidosis; Systemic
Treatment Propylene Glycol-Free Melphalan Hydrochloride
Clinical Study IdentifierNCT02994784
SponsorBoston Medical Center
Last Modified on24 March 2022

Eligibility

Yes No Not Sure

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
Ability to understand and willingness to sign informed consent
institutional guidelines
Pulmonary Function Test demonstrating a diffusion capacity of lung for carbon monoxide ≥ 50%
Systolic blood pressure >90 mm Hg (supine position)
Left ventricular ejection fraction ≥40%
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
Decompensated or uncontrolled heart failure
New York Heart Association ≥3
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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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