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

NCT01816620 | Completed Phase 2

• 1 other identifier: POEMS-01
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

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.

Conditions

POEMS Syndrome

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (7)

• 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)

Study Arms (1)

Lenalidomide, dexamethasone

EXPERIMENTAL

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

Drug: Lenalidomide, Dexamethasone

Interventions

Lenalidomide, Dexamethasone DRUG

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.

Also known as: Revlimid

Lenalidomide, dexamethasone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead
• Celgene Corporation: collaborator

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100005, China

Location

Related Publications (3)

• 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.

  PMID: 34025681 DERIVED

• 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.

  PMID: 32429967 DERIVED

• 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.

  PMID: 29603764 DERIVED

MeSH Terms

Conditions

POEMS Syndrome; Neoplasms, Plasma Cell

Interventions

Lenalidomide; Dexamethasone

Condition Hierarchy (Ancestors)

Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunoproliferative Disorders; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Jian Li, M.D.

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

• Dao-bin Zhou, MD

  Peking Union Medical College Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate professor of Hematology Department

Study Record Dates

• First Submitted: March 14, 2013
• First Posted: March 22, 2013
• Study Start: March 1, 2014
• Primary Completion: October 1, 2015
• Study Completion: April 1, 2017
• Last Updated: April 25, 2017

Record last verified: 2017-04

Locations

CN (1)