Study of Pomalidomide for Treatment of Relapsed/Refractory Primary Central Nervous System Lymphoma and Newly Diagnosed or Relapsed/Refractory Intraocular Lymphoma

  • Study type:

    Interventional What is this?

    Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Study phase:

    I What is this?

    During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

Study IDs

  • Site IRB:

    • Scottsdale/Phoenix, Arizona: 12-003419
    • Jacksonville, Florida: 12-003419
    • Rochester, Minnesota: 12-003419
  • NCT ID:

    NCT01722305
  • Sponsor Protocol Number:

    MC1281

About this study

This phase I clinical trial aims to elucidate the maximal tolerated dose and side effects of pomalidomide in patients with relapsed or refractory primary central nervous system lymphoma or newly diagnosed or relapsed or refractory vitreoretinal lymphoma (Intraocular lymphoma). The study will also determine the efficacy and survival related to pomalidomide therapy in a MTD expanded cohort.

Rationale - Pomalidomide is a novel immunomodulatory agent, which has shown significant activity against CNS lymphoma in preclinical studies. Blood brain barrier is an obstacle in treatment of brain tumors such as CNS lymphoma. Pomalidomide was shown to have an excellent CNS penetration in a murine model: approximately 39% of systemically administered pomalidomide could penetrate the central nervous system. As such, pomalidomide is a promising immunomodulator to be tested in a clinical trial for CNS lymphoma. It works by killing the lymphoma cells directly as well as indirectly via activation of the immune response against lymphoma cells.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

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) 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 unknown:
    • Pregnant women
    • Nursing women
    • Men or women of childbearing potential who are unwilling to employ adequate contraception
  • 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

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Han Tun, M.D.

Closed for enrollment

Cancer Center Clinical Trials Referral Office

855-776-0015

Jacksonville, Fla.

Mayo Clinic principal investigator

Han Tun, M.D.

Contact us for the latest status

Cancer Center Clinical Trials Referral Office

855-776-0015

Rochester, Minn.

Mayo Clinic principal investigator

Han Tun, M.D.

Contact us for the latest status

Cancer Center Clinical Trials Referral Office

855-776-0015