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Home » Clinical Trials » Amyloidosis

Clinical Trial Details

Overview

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

Purpose

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 preference.

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
Phase

1/2

Gender

Both Male and Female

Age

18 and up

Overall Status

Recruiting

Lead Sponsor

Boston Medical Center

Duration

40 Months

Facility Type

N/A

Eligibility

Both Male and Female ages 18 Years and up

Inclusion Criteria:

  1. Understand and voluntarily sign an informed consent form (ICF).

  2. ≥18yrs old at study entry.

  3. Able to adhere to the study visit schedule and other protocol requirements.

  4. Biopsy proven tissue amyloid deposits or positive fat aspirate

  5. Proof of AL type (a or b)

  6. Measurable plasma cell dyscrasia (a or b and c of the following required):

  7. Monoclonal protein in the serum or urine by immunofixation electrophoresis

  8. Plasmacytosis of bone marrow ( < 30% plasma cells) with monoclonal staining for kappa or lambda light-chain isotype

  9. dFLC of 50mg/L (dFLC=difference in involved and uninvolved serum free light-chain levels)

  10. 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 study

  11. 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

  12. SWOG performance status ≤2 at study entry

  13. 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

  1. 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.

  2. 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.

  3. Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (subjects intolerant to ASA may use warfarin or low molecular weight heparin).

Exclusion Criteria:

  1. Secondary or familial amyloidosis

  2. Multiple myeloma (≥30% plasma cells in a bone marrow biopsy specimen or lytic bone lesions)

  3. Cytotoxic chemotherapy or radiation therapy ≤4 weeks of study entry or following baseline evaluation

  4. Symptomatic cardiac arrhythmias or O2-dependent restrictive cardiomyopathy

  5. Dialysis-dependent

  6. Untreated or uncontrolled infections.

  7. Serious medical conditions, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the ICF.

  8. Pregnant or breast feeding females (lactating females must agree not to breast feed while taking pomalidomide).

  9. 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.

  10. Use of any other experimental drug or therapy within 28 days of baseline.

  11. Known intolerance to steroids.

  12. Known hypersensitivity to thalidomide or lenalidomide

  13. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.

  14. Concurrent use of other anti-cancer agents or treatments.

  15. 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)

Country State City Zip Facility and Contact
United States Massachusetts Boston 02118 Boston Medical Center
Sally Fennessey
617-638-8265
sally.fennessey@bmc.org

Contact

Sally Fennessey
617-638-8265
E-mail:

NCT ID: NCT01570387

Date Last Changed: July 21, 2014

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