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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
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: 43
Recruitment status: Recruiting
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:     Annie Jose, BS
Address: 
Telephone: 617-638-8213
Email: annie.jose@bmc.org
Affiliation: 
Name:     John M Sloan, MD
Address: 
Telephone: 617-638-7519
Email: mark.sloan@bmc.org
Affiliation: 
Name:     John M Sloan, MD
Address: 
Telephone:
Email:
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 treatment-related adverse events as assessed by CTCAE v4.0 [Time Frame: 100 days]
Secondary Outcome(s)
hematologic overall response rate [Time Frame: 6 months]
neutrophil engraftment [Time Frame: 3 weeks]
number of hospitalizations [Time Frame: 100 days]
organ response [Time Frame: 12 months]
platelet engraftment [Time Frame: 3 weeks]
treatment related mortality [Time Frame: 100 days]
Secondary ID(s)
H-35835
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Spectrum Pharmaceuticals, Inc
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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