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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01816620
• Date of registration: 14/03/2013
• Prospective Registration: Yes
• Primary sponsor: Peking Union Medical College Hospital
• Public title: Study to Evaluate Lenalidomide Plus Dexamethasone in Patients With Newly Diagnosed POEMS Syndrome
• Scientific 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
• Date of first enrolment: March 2014
• Target sample size: 41
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01816620
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

China

Contacts

• Name: Dao-bin Zhou, MD
• Affiliation: Peking Union Medical College Hospital

• Name: Jian Li, M.D.
• Affiliation: Peking Union Medical College Hospital

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

POEMS Syndrome

Intervention(s)

Drug: Lenalidomide, Dexamethasone

Primary Outcome(s)

Hematological response rate [Time Frame: last day of the LDx treatment regimen (up to 9 months)]

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

Secondary Outcome(s)

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]

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]

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)]

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]

adverse events [Time Frame: throughout the treatment and until 30 days after the administration of the last dose of a study drug]

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]

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)]

Secondary ID(s)

POEMS-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Celgene Corporation