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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01034592
• Date of registration: 15/12/2009
• Prospective Registration: No
• Primary sponsor: Jason Robert Gotlib
• Public title: Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
• Scientific title: A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia
• Date of first enrolment: November 2009
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01034592
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Jason Robert Gotlib
• Affiliation: Stanford University

Key inclusion & exclusion criteria

INCLUSION CRITERIA

- 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)

EXCLUSION CRITERIA

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Acute Myeloid Leukemia (AML)

Leukemia

Anemia

Myelodysplastic Syndromes (MDS)

Intervention(s)

Drug: Lenalidomide

Primary Outcome(s)

Red Blood Cell (RBC) Transfusion Independence [Time Frame: 6 months]

Secondary Outcome(s)

Hemoglobin Concentration [Time Frame: 6 months]

Toxicity [Time Frame: 6 months]

Platelet Response [Time Frame: 6 months]

Neutrophil Response [Time Frame: 6 months]

Red Blood Cell (RBC) Transfusions [Time Frame: 6 months]

Duration of Response [Time Frame: 6 months]

Secondary ID(s)

HEMMDS0022

IRB-16822

RV-0365

SU-12082009-4523

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Celgene Corporation

Results

• Results available: Yes
• Date Posted: 07/02/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01034592