Study to Evaluate Lenalidomide Plus Dexamethasone in Patients With Newly Diagnosed POEMS Syndrome

Sponsor

Peking Union Medical College Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT01816620

Collaborator

Celgene Corporation (Industry)

Enrollment

Location

Arm

Actual Duration (Months)

1.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this prospective study is to determine the efficacy and safety of lenalidomide plus dexamethasone in patients with newly diagnosed POEMS syndrome.

Condition or Disease	Intervention/Treatment	Phase
POEMS Syndrome	Drug: Lenalidomide, Dexamethasone	Phase 2

Detailed Description

The investigators propose to use the adverse effects as well as the need for dose reduction as a criterion to judge tolerability of treatment.

The primary endpoint would be hematological response rate according to international myeloma working group (IMWG) response criteria for amyloidosis and neurological response rate defined by overall neuropathy limitation scale (ONLS) score.

This study expects to enroll approximately 41 subjects.

Study Design

Study Type:

Interventional

Actual Enrollment :

41 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-label Phase II Study to Determine the Efficacy and Safety of Lenalidomide Plus Dexamethasone (LDex) in Patients With Newly Diagnosed POEMS Syndrome

Actual Study Start Date :

Mar 1, 2014

Actual Primary Completion Date :

Oct 1, 2015

Actual Study Completion Date :

Apr 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Lenalidomide, dexamethasone Lenalidomide 10mg qd d1-21 & dexamethasone 40mg qw d1,8,15,22	Drug: Lenalidomide, Dexamethasone 12 cycles of lenalidomide and dexamethasone, each cycle lasts 28 days. For patients with Ccr equal to or higher than 30ml/min, oral lenalidomide 10mg once daily for days 1-21 out of a 28 cycle. For patients with Ccr lower than 30ml/min, oral lenalidomide 10mg once every other day for days 1-21 out of a 28 cycle. All the patients take Dexamethasone orally 40mg once weekly for day 1,8,15,22 out of a 28 cycle. Other Names: Revlimid

Arm

Intervention/Treatment

Experimental: Lenalidomide, dexamethasone

Lenalidomide 10mg qd d1-21 & dexamethasone 40mg qw d1,8,15,22

Drug: Lenalidomide, Dexamethasone

12 cycles of lenalidomide and dexamethasone, each cycle lasts 28 days. For patients with Ccr equal to or higher than 30ml/min, oral lenalidomide 10mg once daily for days 1-21 out of a 28 cycle. For patients with Ccr lower than 30ml/min, oral lenalidomide 10mg once every other day for days 1-21 out of a 28 cycle. All the patients take Dexamethasone orally 40mg once weekly for day 1,8,15,22 out of a 28 cycle.

Other Names:

Revlimid

Outcome Measures

Primary Outcome Measures

Hematological response rate [last day of the LDx treatment regimen (up to 9 months)]
Neurological response rate defined by ONLS score [last day of the LDx treatment regimen (up to 9 months)]

Secondary Outcome Measures

response rate of critical organs [every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months]
Response rate of serum vascular endothelial growth factor (VEGF) level [every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months]
Time to initial neurological response [every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months]
Overall survival [From date of first diagnosis until the date of death from any cause, otherwise patients will be censored in June, 2018 (up to 5 years)]
Relapse free survival [From date of first diagnosis until the date of first documented relapse or date of death from any cause, whichever came first, otherwise patients will be censored in June, 2018 (up to 5 years)]
adverse events [throughout the treatment and until 30 days after the administration of the last dose of a study drug]
time to the best neurological response [every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients must understand and voluntarily sign an informed consent form.
Older than 18 years old at the time of signing consent.
Meet the diagnostic criteria of POEM syndrome.
Must be cytotoxic treatment naive. However, previous or existing corticosteroid (prednisone or dexamethasone) or intravenous immunoglobin (IVIG) therapy is allowed.
Women of childbearing potential must understand that the study medication could have a potential teratogenic risk. They should undergo complete contraception during the study period.
Male subjects must agree to use condoms throughout study drug therapy.

Exclusion Criteria:

Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
Pregnant or lactating females.
Any of the following laboratory abnormalities:

Absolute neutrophil count(ANC) of<1.0×10E9 cell/L. Platelet count<50×10E9 cell/L. Renal failure requiring dialysis. Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3 times of the normal upper limit.

Prior history of malignancies, but not including basal cell carcinoma of the skin, squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, and T1a or T1b prostate cancer.
Known hypersensitivity or prior history of uncontrollable side effects to dexamethasone therapy.
Prior use of cytotoxic drugs.
Subjects who are unable or unwilling to undergo antithrombotic therapy.