Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients
study id #: NCT02994784
condition: Amyloidosis; Systemic
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.
intervention: Propylene Glycol-Free Melphalan Hydrochloride
last updated: September 17, 2021
start date: January 8, 2018
estimated completion: January 2023
last updated: September 8, 2021
phase of development: Phase 2
size / enrollment: 43
study 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.
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
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)
- 100 days
- neutrophil engraftment
- platelet engraftment
- treatment related mortality
- hematologic overall response rate
- organ response
- number of hospitalizations
• Eligible Sexes: all
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: 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
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
New Miami Hurricanes DL Coach Todd Stroud has Overcome Life Threatening IllnessNew Miami Hurricanes defensive line coac...
Dr. Jennifer McMahon: Coping With Amyloidosis – ASG Webinar 2/6https://www.youtube.com/watch?v=CkBwTpXh...
The ABCs of Amyloidosis: The Journey From Diagnosis to TreatmentThis article is for people who have amyl...
Leyla Shune, MDLeyla Shune is an Assistant Professor at...
World Heart Day 2021As the world struggles to fight COVID-...
Understanding Transthyretin AmyloidosisTransthyretin amyloidosis is a slowly pr...
Clinical Diagnosis Guidelines for Transthyretin AmyloidosisAmyloidosis is a rare disease caused by ...