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

• Register: ClinicalTrials.gov
• Last refreshed on: 5 June 2023
• Main ID: NCT02994784
• Date of registration: 18/11/2016
• Prospective Registration: Yes
• Primary sponsor: Boston Medical Center
• Public title: Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients
• Scientific title: A Phase II Single-Center, Open-Label, Safety and Efficacy Study of Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients Undergoing Autologous Stem Cell Transplantation
• Date of first enrolment: January 8, 2018
• Target sample size: 27
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02994784
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: John M Sloan, MD
• Affiliation: Attending Physician

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyloidosis; Systemic

Intervention(s)

Drug: Propylene Glycol-Free Melphalan Hydrochloride

Primary Outcome(s)

Number of Participants With Renal Dysfunction [Time Frame: 100 days]

Number of Participants With Cardiac Dysfunction (New Arrhythmia) [Time Frame: 100 days]

Secondary Outcome(s)

Hematologic Overall Response Rate [Time Frame: 6 months]

Platelet Engraftment [Time Frame: 100 days]

Neutrophil Engraftment [Time Frame: 3 weeks]

Treatment Related Mortality [Time Frame: 100 days]

Participants With Peri-transplant Hospitalizations [Time Frame: 100 days]

Organ Response [Time Frame: 12 months]

Secondary ID(s)

H-35835

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Spectrum Pharmaceuticals, Inc

Results

• Results available: Yes
• Date Posted: 24/05/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02994784