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A Phase I Pilot Study of Immunotherapy Using Lenalidomide Plus "Bystander" Vaccine in Patients With High-Risk Myelodysplastic Syndrome (MDS)

Phase 1
18 Years
Open (Enrolling)

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Trial Information

A Phase I Pilot Study of Immunotherapy Using Lenalidomide Plus "Bystander" Vaccine in Patients With High-Risk Myelodysplastic Syndrome (MDS)

Fixed dose of lenalidomide at 10 mg/day, Days 1- 21 of 28 days of treatment cycle, and 4
dose escalations of GM.CD40L vaccine: 10 X 10^6 GM.CDL cells per vaccination; 30 X 10^6
GM.CDL cells per vaccination; 60 X 10^6 GM.CDL cells per vaccination; 120 X 10^6 GM.CDL
cells per vaccination; Vaccination at 2-week intervals, on days 8 and 22, for a total of
four 28-day cycles.

Inclusion Criteria:

- Must understand and voluntarily sign an informed consent form

- Age ≥18 years at the time of signing the informed consent form

- Able to comply with the study visit schedule and assessments required by the protocol

- Documented diagnosis of MDS with subtypes of Refractory Anemia with Excess Blast 1
(RAEB-1) (myeloblast ≥5-9%) or Refractory Anemia with Excess Blast 2 (RAEB-2)
(myeloblast ≥10-19%) or intermediate 2, Acute myelogenous leukemia with bone marrow
myeloblast >30% and high risk defined by International Prognostic Scoring System
(IPSS) scores or refractory anemia with excess blast in transformation (RAEB-t)
(myeloblast ≥ 20-30%) as per French-American-British Classification System (FAB)
criteria. Any single or combination of cytogenetic abnormalities is allowed.

- Study treatment can be offered as first line treatment as long as the available food
and Drug Administration (FDA) approved treatment options are explained by the
treating physician and the participant declines such options.

- Study treatment can be offered to patients who have failed, cannot tolerate or do not
wish to continue other therapeutic agents for MDS.

- Prior chemotherapy is allowed but should be off chemotherapy of any kind for at last
4 weeks prior to initiation of study therapy.

- Must be able to provide adequate bone marrow (BM) aspirate and biopsy specimens for
histopathological evaluation, cytogenetic analysis and tissue banking during the
screening procedure.

- Platelet count must be > 20,000/ µl without platelet transfusion.

- Absolute neutrophil count (ANC) must be >500/ µl without myeloid growth factor

- Should not be receiving erythropoietin and/or myeloid growth factor for at least 14
days prior to initiation of study therapy.

- Should not have current diagnosis or prior history of any autoimmune or immune
deficiency disorders including human immunodeficiency virus positive/acquired
immunodeficiency syndrome (HIV+/AIDS).

- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.

- Females of childbearing potential (FCBP) must have a negative serum or urine
pregnancy test with a sensitivity of at least 50 mIU/mL within 10 14 days prior to
and again within 24 hours of starting lenalidomide 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 lenalidomide. 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 successful vasectomy. All patients
must be counseled at a minimum of every 28 days about pregnancy precautions and risks
of fetal exposure.

- Both male and female and members of all races and ethnic groups are eligible for this

Exclusion Criteria:

- Prior therapy with lenalidomide.

- Proliferative chronic myelomonocytic leukemia (CMML with WBC≥12,000/µL in peripheral
blood), confirmed by bone marrow biopsy.

- Acute myelogenous leukemia with bone marrow myeloblast ≥30%

- MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for
malignant or autoimmune diseases are excluded.

- Any of the following laboratory abnormalities:

- Serum creatinine > 1.5 x upper limit of normal (ULN)

- Serum aspartic transaminase (AST) or alanine transaminase (ALT) >2.0 x ULN

- Serum total bilirubin > 2.0 mg/dL (34 µmol/L)

- Prior ≥ grade-2 national Cancer Institute Common Toxicity Criteria (NCI CTC) allergic
reaction to thalidomide.

- Prior desquamating (blistering) rash while taking thalidomide.

- Prior allergic reaction to vaccination of any sort.

- Participants with ≥ grade-2 neuropathy.

- Clinically significant anemia due to factors such as iron, B12 or folate
deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding.

- Use of cytotoxic chemotherapeutic agents, growth factors, or experimental agents
(agents that are not commercially available) for the treatment of MDS within 28 days
of the start of drug treatment.

- Prior history of malignancy other than MDS (except basal cell or squamous cell
carcinoma or carcinoma in situ of the cervix) unless the participant has been free of
disease for ≥3 years.

- Any serious medical condition or psychiatric illness that will prevent the
participant from signing the informed consent form or will place the participant at
unacceptable risk if he/she participates in the study.

- Pregnant or nursing females.

- Use of corticosteroids greater than the equivalent of prednisone 10mg daily within 4
weeks of the first vaccination, and on-going need for corticosteroids greater than
the equivalent of prednisone 10 mg daily

Type of Study:


Study Design:

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

Outcome Measure:

Maximum Tolerated Dose (MTD)

Outcome Description:

Determination of MTD of GM.CD40L bystander vaccine with lenalidomide in high-risk MDS patients.

Outcome Time Frame:

24 months

Safety Issue:


Principal Investigator

Javier Pinilla, M.D., Ph.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

H. Lee Moffitt Cancer & Research Institute


United States: Food and Drug Administration

Study ID:




Start Date:

February 2009

Completion Date:

August 2013

Related Keywords:

  • Leukemia
  • Myeloid
  • Monocytic
  • Immunotherapy
  • Vaccine
  • MDS
  • High Risk Myelodysplastic Syndrome
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



H. Lee Moffitt Cancer Center & Research InstituteTampa, Florida  33612