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Clinical Trial Details
Research Study Summary
A clinical research study of Pomalidomide and Dexamthasone for the treatment of AL Amyloidosis
Research Study Title
A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis
This study seeks to enroll patients with AL amyloidosis, for whom treatment with one of the
standard melphalan chemotherapy-based regimens is either not recommended or is not their
Pomalidomide (CC-4047) is a drug given by mouth, which can change or regulate the
functioning of the immune system. So, in theory, it may reduce or prevent the production of
the amyloid protein. Pomalidomide is not currently FDA-approved for AL Amyloidosis.
Pomalidomide is chemically similar to thalidomide and lenalidomide, both of these drugs have
been approved by the FDA for treatment of patients with multiple myeloma (MM), a disease
similar to AL Amyloidosis.
Participants in this study will receive pomalidomide and dexamethasone. Phase I is a
dose-escalation study and dose escalation will proceed through 3 dose-levels according to
standard rules in which dose levels are started sequentially after complete evaluation of
the occurrence of dose-limiting toxicities. In the Phase II portion, participants will
receive pomalidomide and dexamethasone using the defined maximum tolerated dose.
To Learn more
Both Male and Female
Both Male and Female ages 18 Years and up
Understand and voluntarily sign an informed consent form (ICF).
≥18yrs old at study entry.
Able to adhere to the study visit schedule and other protocol requirements.
Biopsy proven tissue amyloid deposits or positive fat aspirate
Proof of AL type (a or b)
Measurable plasma cell dyscrasia (a or b and c of the following required):
Monoclonal protein in the serum or urine by immunofixation electrophoresis
Plasmacytosis of bone marrow ( < 30% plasma cells) with monoclonal staining for
kappa or lambda light-chain isotype
dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free
Must have received ≥1 prior treatment for AL amyloidosis, if it is intensive
chemotherapy and an autotransplant it must be ≥6 months prior to enrollment on this
Must have recovered from the reversible side effects of any prior therapy; permanent
and stable side effects/changes are acceptable. Prior treatment for AL amyloidosis
with chemotherapy, thalidomide, lenalidomide or steroids is not an exclusion
SWOG performance status ≤2 at study entry
Laboratory test results within these ranges:
d. Neutrophil ≥1.5 x10e9/L e. Platelets ≥100x10e9/L f. Total bilirubin < 1.5mg/dL g.
AST (SGOT) and ALT (SGPT) < 2xULN h. Serum creatinine < 2.5mg/dL
Disease free of prior malignancies for at least 5yrs with exception of currently
treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of
the cervix or breast.
Females of childbearing potential (FCBP) (a FCBP is a sexually mature woman who has
not undergone a hysterectomy or bilateral oophorectomy, or has not been naturally
postmenopausal for at least 24 consecutive months) must have a negative serum or
urine pregnancy test with a sensitivity ≥ 50 mIU/mL 10-14 days prior to and again ≤
24 hours of starting pomalidomide and must either commit to continued abstinence from
heterosexual intercourse or begin TWO acceptable methods of birth control, one highly
effective method and one additional effective method AT THE SAME TIME, ≥ 28 days
before she starts taking pomalidomide. FCBP must also agree to ongoing pregnancy
testing. Men must agree to use a latex condom during sexual contact with a FCBP even
if they have had a vasectomy. All subjects must be counseled at a minimum of every
28 days about pregnancy precautions and risks of fetal exposure.
Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (subjects
intolerant to ASA may use warfarin or low molecular weight heparin).
Secondary or familial amyloidosis
Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone
Cytotoxic chemotherapy or radiation therapy ≤4 weeks of study entry or following
Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy
Untreated or uncontrolled infections.
Serious medical conditions, laboratory abnormality, or psychiatric illness that would
prevent the subject from signing the ICF.
Pregnant or breast feeding females (lactating females must agree not to breast feed
while taking pomalidomide).
Any condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study.
Use of any other experimental drug or therapy within 28 days of baseline.
Known intolerance to steroids.
Known hypersensitivity to thalidomide or lenalidomide
The development of erythema nodosum if characterized by a desquamating rash while
taking thalidomide or similar drugs.
Concurrent use of other anti-cancer agents or treatments.
Known HIV positivity is not an exclusion, unless CD4 counts < 200/mcL and/or patient
has multi-drug resistant HIV infections and/or other concurrent AIDS-defining
conditions. HIV b-DNA < 75 copies/mL.
Site Locations (1)
||Boston Medical Center
NCT ID: NCT01570387
Date Last Changed: July 21, 2014
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