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

Phase 2

Completed

• Conditions: POEMS Syndrome
• Interventions: Drug: Lenalidomide, Dexamethasone

• Registration Number: NCT01816620
• Lead Sponsor: Peking Union Medical College Hospital

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.

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 Timeline

• Study First Submitted: 2013-03-14
• Study First Submitted QC: 2013-03-21
• Study First Posted: 2013-03-22
• Study Start: 2014-03
• Primary Completion: 2015-10
• Status Verified: 2017-04
• Study Completion: 2017-04
• Last Update Submitted: 2017-04-23
• Last Update Posted: 2017-04-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 41

Inclusion Criteria

1. Patients must understand and voluntarily sign an informed consent form.
2. Older than 18 years old at the time of signing consent.
3. Meet the diagnostic criteria of POEM syndrome.
4. Must be cytotoxic treatment naive. However, previous or existing corticosteroid (prednisone or dexamethasone) or intravenous immunoglobin (IVIG) therapy is allowed.
5. 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.
6. Male subjects must agree to use condoms throughout study drug therapy.

Exclusion Criteria

1. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.

2. Pregnant or lactating females.

3. 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.

4. 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.

5. Known hypersensitivity or prior history of uncontrollable side effects to dexamethasone therapy.

6. Prior use of cytotoxic drugs.

7. Subjects who are unable or unwilling to undergo antithrombotic therapy.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Lenalidomide, dexamethasone	Lenalidomide, Dexamethasone	Lenalidomide 10mg qd d1-21 \& dexamethasone 40mg qw d1,8,15,22

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
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)
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
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

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China