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NCT02994784 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
TERMINATED
Registry last update
2023-05-24

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Number of Participants With Renal Dysfunction

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outcome · POSTED

Number of Participants With Cardiac Dysfunction (New Arrhythmia)

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outcome · POSTED

Neutrophil Engraftment

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outcome · POSTED

Platelet Engraftment

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outcome · POSTED

Treatment Related Mortality

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outcome · POSTED

Hematologic Overall Response Rate

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outcome · POSTED

Organ Response

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outcome · POSTED

Participants With Peri-transplant Hospitalizations

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
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
healthy Volunteers
false
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Propylene Glycol-Free Melphalan Hydrochloride (Evomela) administered intravenously at 70-100 mg/m2/day on Days -3 and -2 prior to autologous stem cell transplantation
    intervention Names
    1. Drug: Propylene Glycol-Free Melphalan Hydrochloride
    label
    Evomela
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Evomela
    description
    Intravenous Propylene Glycol-Free Melphalan Hydrochloride
    name
    Propylene Glycol-Free Melphalan Hydrochloride
    other Names
    1. Evomela
    type
    DRUG
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
27
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    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.).
    measure
    Number of Participants With Renal Dysfunction
    time Frame
    100 days
  2. description
    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 cardiac dysfunction (new arrhythmia).
    measure
    Number of Participants With Cardiac Dysfunction (New Arrhythmia)
    time Frame
    100 days
secondary Outcomes
  1. description
    time to neutrophil engraftment
    measure
    Neutrophil Engraftment
    time Frame
    3 weeks
  2. description
    Assess time to platelet engraftment
    measure
    Platelet Engraftment
    time Frame
    100 days
  3. description
    Number of patients who expire within 100 days of transplant
    measure
    Treatment Related Mortality
    time Frame
    100 days
  4. description
    Number of patients with response based on Gertz, Palladini criteria (below) Complete response (CR): Normal serum free light chain ratio,Negative serum and urine immunofixation electrophoresis Very good partial response (VGPR): Difference in serum free light chains less than 40 mg/L Partial Response (PR): \>50% Reduction in the difference in serum free light chains Stable Disease (SD): Meets neither criteria for CR, VGPR, PR or PD
    measure
    Hematologic Overall Response Rate
    time Frame
    6 months
  5. description
    Number of patients with organ response based on Gertz criteria (below) * Kidney: 50% reduction in 24-hour urine protein excretion in the absence of progressive renal insufficiency (defined as a 25% increase in serum creatinine, as long as that is \> to an absolute increase of 0.5 mg/dL). In the case of nephrotic syndrome: a decrease in proteinuria to \< 1g/24h and an improvement in one of 2 extrarenal features - normalization of serum albumin or resolution of edema and/or discontinuation of diuretics in response to improvement in edema. * Heart: ≥ 2 mm reduction in the interventricular septal (IVS) thickness by echocardiogram, improvement of ejection fraction by ≥ 20% (echocardiogram must be performed at the same institution), or decrease in 2 NYHA classes without increase in diuretic need.
    measure
    Organ Response
    time Frame
    12 months
  6. description
    Number of participants with peri-transplant hospitalizations
    measure
    Participants With Peri-transplant Hospitalizations
    time Frame
    100 days
Full study description
brief 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.
detailed 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.
Source references
references
  1. citation
    Palladini G, Dispenzieri A, Gertz MA, Kumar S, Wechalekar A, Hawkins PN, Schonland S, Hegenbart U, Comenzo R, Kastritis E, Dimopoulos MA, Jaccard A, Klersy C, Merlini G. New criteria for response to treatment in immunoglobulin light chain amyloidosis based on free light chain measurement and cardiac biomarkers: impact on survival outcomes. J Clin Oncol. 2012 Dec 20;30(36):4541-9. doi: 10.1200/JCO.2011.37.7614. Epub 2012 Oct 22.
    pmid
    23091105
    type
    BACKGROUND
  2. citation
    Sarosiek S, Lee MH, Doros G, Edwards CV, Quillen K, Brauneis D, Shelton AC, Sanchorawala V, Sloan JM. Safety and Efficacy of Propylene Glycol-Free Melphalan in Patients with AL Amyloidosis Undergoing Autologous Stem Cell Transplantation: Results of a Phase II Study. Transplant Cell Ther. 2023 Nov;29(11):695.e1-695.e7. doi: 10.1016/j.jtct.2023.08.018. Epub 2023 Aug 20.
    pmid
    37607644
    type
    DERIVED
Source notices and limitations
    Discovery and provenance
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    Preserved source evidence · Not medical advice