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

Primary Purpose

POEMS Syndrome

Status

Completed

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

Lenalidomide, Dexamethasone

About this trial

This is an interventional treatment trial for POEMS Syndrome focused on measuring POEMS Syndrome, lenalidomide, newly diagnosed, plasma cell disorder, Dexamethasone

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

Peking Union Medical College Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lenalidomide, dexamethasone

Arm Description

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

Outcomes

Primary Outcome Measures

Hematological response rate

Neurological response rate defined by ONLS score

Secondary Outcome Measures

response rate of critical organs

Response rate of serum vascular endothelial growth factor (VEGF) level

Time to initial neurological response

Overall survival

Relapse free survival

adverse events

time to the best neurological response

Full Information

NCT ID

NCT01816620

First Posted

March 14, 2013

Last Updated

April 23, 2017

Sponsor

Peking Union Medical College Hospital

Collaborators

Celgene Corporation

1. Study Identification

Unique Protocol Identification Number

NCT01816620

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2017

Overall Recruitment Status

Completed

Study Start Date

March 2014 (Actual)

Primary Completion Date

October 2015 (Actual)

Study Completion Date

April 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking Union Medical College Hospital

Collaborators

Celgene Corporation

4. Oversight

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

POEMS Syndrome

Keywords

POEMS Syndrome, lenalidomide, newly diagnosed, plasma cell disorder, Dexamethasone

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

41 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Lenalidomide, dexamethasone

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Lenalidomide, Dexamethasone

Other Intervention Name(s)

Revlimid

Intervention Description

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.

Primary Outcome Measure Information:

Title

Hematological response rate

Time Frame

last day of the LDx treatment regimen (up to 9 months)

Title

Neurological response rate defined by ONLS score

Time Frame

last day of the LDx treatment regimen (up to 9 months)

Secondary Outcome Measure Information:

Title

response rate of critical organs

Time Frame

every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months

Title

Response rate of serum vascular endothelial growth factor (VEGF) level

Time Frame

every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months

Title

Time to initial neurological response

Time Frame

every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months

Title

Overall survival

Time Frame

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)

Title

Relapse free survival

Time Frame

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)

Title

adverse events

Time Frame

throughout the treatment and until 30 days after the administration of the last dose of a study drug

Title

time to the best neurological response

Time Frame

every month during the first 3 months calculated from the day study started, then every 3 months in the next 9 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jian Li, M.D.

Organizational Affiliation

Peking Union Medical College Hospital

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Dao-bin Zhou, MD

Organizational Affiliation

Peking Union Medical College Hospital

Official's Role

Study Director

Facility Information:

Facility Name

Peking Union Medical College Hospital

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100005

Country

China

12. IPD Sharing Statement

Citations:

PubMed Identifier

34025681

Citation

Gao Y, Zhang S, Yang L, Li J, Liu Y, Wang T. Skin Responses in Newly Diagnosed Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes (POEMS) Syndrome After Therapy With Low-Dose Lenalidomide Plus Dexamethasone. Front Immunol. 2021 May 6;12:681360. doi: 10.3389/fimmu.2021.681360. eCollection 2021.

Results Reference

derived

PubMed Identifier

32429967

Citation

Liu LS, Zhang X, Zhao H, Gao XM, Zhou DB, Dai RP, Li J. Reliability of optic disc edema area in estimating the severity of papilledema in patients with POEMS syndrome. Orphanet J Rare Dis. 2020 May 19;15(1):116. doi: 10.1186/s13023-020-01392-x.

Results Reference

derived

PubMed Identifier

29603764

Citation

Li J, Huang XF, Cai QQ, Wang C, Cai H, Zhao H, Zhang L, Cao XX, Gale RP, Zhou DB. A prospective phase II study of low dose lenalidomide plus dexamethasone in patients with newly diagnosed polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome. Am J Hematol. 2018 Jun;93(6):803-809. doi: 10.1002/ajh.25100. Epub 2018 Apr 18.

Results Reference

derived

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

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