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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.
Last refreshed on: 23 May 2016
Main ID:  NCT01807286
Date of registration: 06/03/2013
Prospective Registration: Yes
Primary sponsor: M.D. Anderson Cancer Center
Public title: Pomalidomide With Melphalan and Dexamethasone for Untreated Systemic AL Amyloidosis
Scientific title: A Phase I/II Clinical Trial of Pomalidomide With Melphalan and Dexamethasone in Patients With Newly Diagnosed Untreated Systemic AL Amyloidosis: Trial Stopped During Phase I
Date of first enrolment: January 2014
Target sample size: 3
Recruitment status: Terminated
Study type:  Interventional
Study design:  Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment  
Phase:  Phase 1
Countries of recruitment
United States
Name:     Robert Orlowski, MD, PHD
Affiliation:  M.D. Anderson Cancer Center
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age >/= 18 years old.

2. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2; Karnofsky
>/= 60%

3. Patients must be willing and able to provide voluntary written informed consent, with
the understanding that consent may be withdrawn by the subject at any time without
prejudice to their future medical care

4. Histologic diagnosis of amyloidosis by Congo red staining of tissue biopsy within 12
months of enrollment.

5. Demonstrable clonal plasma cell disorder based on the presence of an M protein in the
serum and/or urine by immunofixation and/or serum free light chain assay, and/or a
clonal population of plasma cells in the bone marrow based on kappa/lambda staining
of a marrow biopsy.

6. If no demonstrable associated light chain abnormality, then no-light chain
amyloidosis should be excluded by checking for transthyretin, fibrinogen A alpha,
Amyloid A^3.

7. Patients must have measurable disease, as defined by at least one of the following:
Serum or urine immunofixation showing a monoclonal protein and clonal marrow
plasmacytosis by marrow biopsy immunohistochemical staining; Free light chains with
an abnormal free light chain ratio

8. Symptomatic end organ involvement with amyloidosis as defined previously and to
include any one of the following: Renal - albuminuria higher than 0.5 g/day in
24-hour urine analysis; Cardiac - presence of a mean left ventricular wall thickness
on echocardiogram more than 11 mm in the absence of a history of hypertension or
valvular heart disease, or unexplained low voltage (<0.5 mV) on electrocardiogram;
Hepatic - hepatomegaly on physical examination with an alkaline phosphatase level
higher than 200 U/L; Gastrointestinal - gastrointestinal amyloid deposits confirmed
by tissue biopsy.; Soft-tissue or lymphatic involvement - ascertained based on
classic physical exam findings (macroglossia, shoulder pad sign, raccoon eyes, carpal
tunnel syndrome, synovial enlargement, firm enlarged lymph nodes) or biopsy.

9. Inclusion Criteria #8 cont... - Nerve - positive history of autonomic or peripheral
neuropathy and/or nerve conduction defect documented by electromyogram (EMG)/nerve
conduction velocity (NCV) testing.; Skin- measurable skin lesions that are biopsy
proven to be amyloidosis

10. No prior therapy for the monoclonal plasma cell disease.

11. Patients must have evidence of adequate bone marrow reserves, as defined by the
following pretreatment clinical laboratory values within 14 days of study initiation:
Platelet count >/= 100 x 10^9/L without platelet transfusions within 2 weeks of the
initiation of treatment; Hemoglobin >/= 8 g/dLwithout red blood cell transfusions
within 2 weeks of the initiation of treatment; Absolute neutrophil count (ANC) >/=
1.0x10^9/L without growth factor requirement within 1 week of the initiation of

12. Patients must have evidence of adequate hepatic function, as defined by the
following: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) 2.5 times the upper limit of normal; Total bilirubin normal

13. Patients must have evidence of adequate renal function, as defined by the following:
Serum creatinine within the institutional normal limits, OR, if the creatinine is
elevated: Creatinine clearance (CrCl) >/= 30 mL/min., as measured by a 24-hour urine
collection, or estimated by the Cockcroft and Gault formula: Female CrCl = (140 - age
in years) x weight in kg x 0.85/ 72 x serum creatinine in mg/dL ; Male CrCl = (140 -
age in years) x weight in kg x 1.00/72 x serum creatinine in mg/dL

14. Patients must have evidence of adequate cardiac function, as defined by the
following: Absence of New York Heart Association (NYHA) class III or IV congestive
heart failure; Absence of uncontrolled angina or hypertension; Absence of myocardial
infarction in the previous 6 months; Absence of clinically significant bradycardia,
or other uncontrolled cardiac arrhythmia defined as grade 3 or 4 according to
National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events
(CTCAE), version 4.0

15. Patients who have undergone any recent major surgery must have done so at least 4
weeks prior to starting therapy with PMD, with the following
exceptions:Vertebroplasty and/or kyphoplasty, which must have been performed at least
1 week prior to starting PMD; Planned elective surgery unrelated to the patient's
diagnosis of multiple myeloma, such as hernia repair, may be allowed, at the
discretion of the principle investigator, as long as it was performed at least 2
weeks prior to starting PMD, and patients have recovered fully from this procedure

16. Male patients must agree to use an adequate method of contraception for the duration
of the study since the effects of PMD on the developing human fetus are unknown. If a
female partner of a male subject taking Pomalidomide becomes pregnant, the male
subject taking Pomalidomide should notify the Investigator immediately. The pregnant
female partner should notify their healthcare provider. Female patients must be
either post menopausal, free from menses for >/= 2 years, surgically sterilized, or
willing to use two adequate barrier methods of contraception to prevent pregnancy, or
must agree to abstain from heterosexual activity throughout the study. Female
patients of childbearing potential must have a negative serum pregnancy test
(Beta-HCG) before receiving the first dose of PMD. The female participant must also
follow pregnancy testing requirements as outlined in the POMALYST REMS program.

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

18. All study participants must be registered into the mandatory POMALYST REMSTM program,
and be willing and able to comply with the requirements of the POMALYST REMSTM

Exclusion Criteria:

1. Patients who are receiving any concurrent investigational agent with known or
suspected activity against amyloidosis

2. Peripheral neuropathy Grade 2 or higher, as defined by National Cancer Institute
Common Terminology Criteria (NCI CTC) version 4.

3. Uncontrolled or severe cardiovascular disease including myocardial infarction within
6 months of enrollment, New York Heart Association (NYHA) Class III or IV heart
failure uncontrolled angina, or clinically significant pericardial disease.

Age minimum: 18 Years
Age maximum: N/A
Gender: Both
Health Condition(s) or Problem(s) studied
Behavioral: Questionnaires
Drug: Dexamethasone
Drug: Melphalan
Drug: Pomalidomide
Primary Outcome(s)
Maximum Tolerated Dose (MTD) of Pomalidomide with Melphalan and Dexamethasone (PMD). [Time Frame: 28 days]
Secondary Outcome(s)
Complete Response (CR) Rate [Time Frame: Response evaluated after a minimum of 2 cycles and a maximum of 6 cycles of 28-day treatment]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available:
Date Posted:
Date Completed:
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