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A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia

18 Years
Open (Enrolling)
Anemia, Leukemia, Leukemia, Myelocytic, Acute, Dysmyelopoietic Syndromes

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Trial Information

A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia

Inclusion Criteria:

1. Understand and voluntarily sign an informed consent form.

2. Diagnosis of DBA.

3.. Age >=18 years at the time of signing the informed consent form.

4. Able to adhere to the study visit schedule and other protocol requirements.

5. Red blood cell transfusion-dependent with a requirement of at least one unit of RBCs
per month for the 2 months prior to study enrollment (e.g. 2 units/8 weeks)

6. If applicable, ongoing therapy with a stable or decreasing dose of prednisone <= 60
mg/d or corticosteroid equivalent, for which there has been no treatment-related
improvement in RBC transfusion requirements for at least 2 months prior to study entry

7. ECOG performance status of <= 2 at study entry.

8. Laboratory test results within these ranges:

- Absolute neutrophil count >= 1500/mm>=

- Platelet count >= 100,000/mm>=

- Serum creatinine <= 2.0 mg/dL

- Direct bilirubin <= 1.5 mg/dL

- AST (SGOT) and ALT (SGPT) <= 2.5 x ULN

- Disease free of prior malignancies for >= 5 years with exception of currently treated
basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of the cervix
or breast.

9. 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 prescribing lenalidomide (prescriptions must be filled
within 7 days) 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. See Appendix A: Risks of Fetal Exposure, Pregnancy
Testing Guidelines and Acceptable Birth Control Methods.

10. Able to take aspirin (81 - >=25 mg) daily as prophylactic anticoagulation
(patients intolerant to ASA may use warfarin or low molecular weight heparin)

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 breast feeding females. (Lactating females must agree not to breast feed
while taking lenalidomide).

3. Any condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds the
ability to interpret data from the study.

4. Use of any other experimental drug or therapy (excluding steroids) specifically used
for DBA within 28 days of baseline including metoclopramide, leucine, danazol, or other
hormonal therapy.

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

6. Known hypersensitivity to thalidomide.

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

8. Any prior use of lenalidomide.

9. Concurrent use of other anti-cancer agents or treatments.

10. Known positive for HIV or infectious hepatitis, type A, B or C.

Type of Study:


Study Design:

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

Outcome Measure:

RBC transfusion independence

Outcome Time Frame:

Assessment done every 56 days: D56, D112, D168, D224, then every month during Maintenance Phase

Safety Issue:


Principal Investigator

Jason Robert Gotlib

Investigator Role:

Principal Investigator

Investigator Affiliation:

Stanford University


United States: Food and Drug Administration

Study ID:




Start Date:

November 2009

Completion Date:

November 2013

Related Keywords:

  • Anemia
  • Leukemia
  • Leukemia, Myelocytic, Acute
  • Dysmyelopoietic Syndromes
  • Anemia
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Myelodysplastic Syndromes
  • Preleukemia
  • Anemia, Diamond-Blackfan



Stanford University School of MedicineStanford, California  94305-5317