Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

Stopped early · Phase 1

Conditions studied: Anemia, Leukemia, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)

In brief

This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA). Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence \[MDS International Working Group (IWG) 2000 Criteria will be applied\]. Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression

Key facts

Study ID: NCT01034592
Run by: Jason Robert Gotlib
People needed: 2
Starts: 2009-11-01
Expected to finish: 2012-12-01
Last updated by the study team: 2017-08-02

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Understand and voluntarily sign an informed consent form
Diagnosis of DBA
Age ≥ 18 years at the time of signing the informed consent form.
Able to adhere to the study visit schedule and other protocol requirements.
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 (eg, 2 units/8 weeks)
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
Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2 at study entry.
Laboratory test results within these ranges:
Absolute neutrophil count (ANC) ≥ 1500/uL
Platelet (Plt) count ≥ 100,000/uL
Serum creatinine ≤ 2.0 mg/dL
Direct bilirubin ≤ 1.5 mg/dL
Aspartate aminotransferase (AST) ≤ 2.5 x upper limit of normal (ULN)
Alanine aminotransferase (ALT) ≤ 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
Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of ≥ 50 milli-International Units (MIU)/mL within 10 to 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 2 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
Able to take aspirin (81 to 325 mg) daily as prophylactic anticoagulation (patients intolerant to aspirin may use warfarin or low molecular weight heparin)

You may not qualify if…

Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking lenalidomide)
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
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
Clinically significant anemia due to factors such as iron, B12, folate deficiencies, autoimmune or hereditary hemolysis, or gastrointestinal bleeding.
Known hypersensitivity to thalidomide
The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
Any prior use of lenalidomide
Concurrent use of other anti-cancer agents or treatments
Known positive for HIV or infectious hepatitis, type A, B or C

Where it is running

Stanford University School of Medicine — Stanford, California, United States

Full record on ClinicalTrials.gov

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