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Phase I Trial of Pomalidomide for Patients With Relapsed/Refractory Primary CNS Lymphoma and Patients With Newly Diagnosed or Relapsed/Refractory Primary Vitreoretinal Lymphoma

Phase 1
18 Years
Open (Enrolling)
Primary Central Nervous System Lymphoma, Primary Vitreoretinal Lymphoma (Intraocular Lymphoma)

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Trial Information

Phase I Trial of Pomalidomide for Patients With Relapsed/Refractory Primary CNS Lymphoma and Patients With Newly Diagnosed or Relapsed/Refractory Primary Vitreoretinal Lymphoma


I. To establish the maximum tolerated dose (MTD) of pomalidomide in patients with CNS


I. To evaluate the efficacy (overall response rate) and safety of pomalidomide in patients
with primary central nervous system lymphoma (PCNSL) and primary vitreoretinal lymphoma
(PVRL) in an MTD expanded cohort.

II. To evaluate overall survival and progression free survival.

Objectives of correlative research:

I. To study the pharmacokinetics of pomalidomide in the central nervous system.

II. To identify the predictive biomarkers for responsiveness to pomalidomide.

Inclusion Criteria:

- Relapsed or refractory primary central nervous system (CNS) diffuse large B cell
lymphoma (PCNSDLBCL) with a measurable CNS lesion (>= 1 cm in largest dimension);
NOTE: tissue biopsy is not absolutely necessary unless clinical findings strongly
suggest other etiologies as per treating physician; initial diagnosis must be made by
tissue biopsy.

- Relapsed/refractory primary vitreoretinal DLBCL who also have a measureable CNS
lesion >= 1 cm in largest dimension); NOTE: tissue biopsy requirement of the CNS
lesion is as outlined in bullet above; ocular tissue biopsy is required if
ophthalmologic examination findings indicate ocular relapse.

- Newly diagnosed or relapsed/refractory primary vitreoretinal DLBCL with or without
cerebrospinal fluid (CSF) involvement but without brain or spinal cord involvement;
NOTE: ocular tissue biopsy is required for all cases as part of ophthalmologic
examination; Note: these patients will be eligible for participation in MTD expanded
phase of the study.

- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1, 2 or 3

- Absolute neutrophil count (ANC) >= 1000/uL

- Platelets (PLT) >= 100,000/uL

- Total bilirubin =< 1.5 x upper limit of normal (ULN) or if total bilirubin is > 1.5 x
ULN the direct bilirubin must be =< 1.5 x ULN (=< 0.45 mg/dL)

- Aspartate aminotransferase (AST) =< 3 x ULN

- Creatinine =< 2.5 x ULN

- Females of childbearing potential (FCBP)Â must have a negative serum or urine
pregnancy test with a sensitivity of at least 25 mIU/mL within 10 - 14 days prior to
and again within 24 hours of starting Pomalidomide and must either commit to
continued abstinence from heterosexual intercourse or begin TWO acceptable methods of
birth control, one highly effective method and one additional effective method AT THE
SAME TIME, at least 28 days before she starts taking Pomalidomide; FCBP must also
agree to ongoing pregnancy testing; men must agree to use a latex condom during
sexual contact with a FCBP even if they have had a vasectomy; all patients must be
counseled at a minimum of every 28 days about pregnancy precautions and risks of
fetal exposure

- Able to take aspirin (81 or 325 mg) daily as prophylactic anticoagulation (patients
intolerant to acetylsalicylic acid [ASA] may use warfarin or heparin)

- Provide informed written consent

- Willing to return to participating medical institutions for follow-up

Exclusion Criteria:

- Any of the following because this study involves an investigational agent whose
genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are

- Pregnant women

- Nursing women

- Men or women of childbearing potential who are unwilling to employ adequate

- The development of erythema nodosum if characterized by a desquamating rash while
taking thalidomide or similar drugs

- Uncontrolled infection

- Therapy with myelosuppressive chemotherapy or biologic therapy < 21 days prior to
registration; Note: unless the patient has recovered from the nadir of the previous
treatment to a level that meets the inclusion eligibility criteria of this protocol

- Persistent toxicities >= grade 2 from prior chemotherapy or biological therapy
regardless of interval since last treatment

- History of thromboembolic episodes =< 3 months prior to registration

- Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy
considered investigational (utilized for a non-Food and Drug Administration
[FDA]-approved indication and in the context of a research investigation)

- Immunodeficiency states including human immunodeficiency virus (HIV) infection

- Active hepatitis B or C with uncontrolled disease; Note: a detailed assessment of
hepatitis B/C medical history and risk factors must be done at screening for all
patients hepatitis B core immunoglobulin M antibody (HBcIgM Ab), hepatitis B surface
antigen (HBsAg) and hepatitis C antibody screen (HCV Ab Scrn) w/Reflex testing are
required at screening for all patients with a positive medical history based on risk
factors and/or confirmation of prior hepatitis B (HBV) infection

- Active other malignancy requiring treatment that would interfere with the assessments
of response of the lymphoma to protocol treatment

- Inability to swallow or impairment of gastrointestinal function or gastrointestinal
disease that may significantly alter the absorption of the drugs (e.g., ulcerative
disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small
bowel resection) that would preclude use of oral medications

- Any severe and/or uncontrolled medical conditions or other conditions that, in the
treating physician's opinion, could adversely impact their ability to participate in
the study

- Major surgery =< 4 weeks prior to registration or have not recovered from side
effects of such therapy

- New York Heart Association classification III or IV

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Maximum tolerated dose (MTD) of pomalidomide as determined by dose-limiting toxicities graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v4.0

Outcome Description:

MTD is defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least one-third of patients (at least 2 of a maximum of 6 new patients).

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

Han W. Tun, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Mayo Clinic in Florida


United States: Food and Drug Administration

Study ID:




Start Date:

April 2013

Completion Date:

June 2016

Related Keywords:

  • Primary Central Nervous System Lymphoma
  • Primary Vitreoretinal Lymphoma (Intraocular Lymphoma)
  • Lymphoma



Mayo Clinic Rochester, Minnesota  55905
Mayo Clinic Campus in Florida Jaxsonville, Florida