Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS

NCT01133275 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: MCC-16099RV-MDS-PI-045625005/1
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this research is to evaluate the use of lenalidomide and prednisone in people with Myelodysplastic Syndrome (MDS). Lenalidomide is a drug that alters the immune system and it may also interfere with the development of tiny blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. Lenalidomide is approved by the U.S. Food and Drug Administration (FDA) for the treatment of specific types of myelodysplastic syndrome (MDS) and in combination with dexamethasone for people with multiple myeloma (MM) who have received at least 1 prior therapy. MDS and MM are cancers of the blood. It is currently being tested in a variety of cancer conditions. As it is being used in this study it is considered an investigational use. An "investigational use" is a use that is being tested and is not approved by the FDA. Prednisone is approved by the FDA to treat numerous conditions. In addition, prednisone is approved by the FDA to treat Low or Intermediate-1 IPSS Risk, non-del (5q) MDS. "Study drug" refers to the combination of lenalidomide and prednisone.

Conditions

Myelodysplastic Syndrome; MDS

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Erythroid Response

  The rate of erythroid response to treatment with the lenalidomide/prednisone combination in non-del (5q) low and int-1 risk Myelodysplastic Syndrome (MDS) with symptomatic anemia. Hematological improvement erythroid response (HI-E) according to International Working Group (IWG) 2006 criteria.

  Up to 7 months

Secondary Outcomes (1)

• Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment

  Up to 54 months

Study Arms (1)

Lenalidomide and Prednisone Therapy

EXPERIMENTAL

All participants received lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks).

Drug: Prednisone; Drug: Lenalidomide

Interventions

Prednisone DRUG

Prednisone therapy for 6 cycles (24 weeks).

Also known as: Deltasone

Lenalidomide and Prednisone Therapy

Lenalidomide DRUG

Lenalidomide therapy for 6 cycles (24 weeks).

Also known as: REVLIMID®

Lenalidomide and Prednisone Therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Understand and voluntarily sign an informed consent form
• Age ≥ 18 years at the time of signing the informed consent form
• Able to adhere to the study visit schedule and other protocol requirements
• Documented diagnosis of MDS according Word Health Organization (WHO) that meets International Prognostic Scoring System (IPSS) criteria for Low- to Intermediate-1-risk disease or non-proliferative (white blood count \[WBC\] \< 13,000/uL) chronic myelomonocytic leukemia (CMML) and MDS/myeloproliferative neoplasms (MPN).
• Absence of a chromosome 5q deletion by metaphase cytogenetics or fluorescent in situ hybridization (FISH) analysis
• Red blood cell (RBC) transfusion-dependent anemia defined as having received ≥4 transfusions of RBCs for hemoglobin (Hgb) ≤ 9.0 g/dl within 56 days of randomization or symptomatic anemia (Hgb ≤ 9.0 g/dl)
• No response or progression on prior treatment with epoetin alpha (\> 40,000 U/wk x 6), darbepoetin alpha (≥ 500 mcg q 3 wk x 2) or serum erythropoietin (EPO) concentration ≥ 500 mU/ml
• All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study.
• Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2 at study entry
• Laboratory test results within these ranges: Absolute neutrophil count ≥ 500 /mm³; Platelet count ≥ 50,000 /mm³; Creatinine Clearance \> 60 mL/min by Cockcroft-Gault formula; Total bilirubin ≤ 1.5 mg/dl; aspartic transaminase (AST)and alanine transaminase (ALT) ≤ 2 x upper limit of normal (ULN).
• Disease free of prior malignancies for ≥ 3 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "insitu" of the cervix or breast
• Must be registered into the mandatory RevAssist® program, and be willing and able to comply with the requirements of RevAssist®.
• Females of childbearing potential (FCBP)† must have a negative serum or urine pregnancy test with a sensitivity of at least 50 milli-international units per milliliter (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.

You may not qualify if:

• Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from signing the informed consent form.
• Pregnant or breast feeding. (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 within 28 days of baseline
• Known hypersensitivity to thalidomide
• The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs or history of desquamating (blistering) rash while taking thalidomide
• Any prior use of lenalidomide
• Concurrent use of other anti-cancer agents or treatments
• Known positive for human immunodeficiency virus (HIV) or infectious hepatitis, type B or C
• Proliferative CMML (WBC ≥13,000/μL)
• MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
• Prior ≥ grade-2 National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) allergic reaction to thalidomide
• Clinically significant anemia due to factors such as iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding
• Known HIV-1 positivity
• Chromosome 5q deletion

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Celgene Corporation: collaborator

Study Sites (2)

University of Florida Shands Cancer Center

Gainesville, Florida, 32610, United States

Location

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Prednisone; Lenalidomide

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Limitations and Caveats

The study was terminated at the end of step one, as no signal of higher response of the combination was observed compared to what was reported on the MDS-002 study.

Results Point of Contact

• Title: Dr. Rami Komrokji
• Organization: H. Lee Moffitt Cancer Center and Research Institute

rami.komrokji@moffitt.org

813-745-4692

Study Officials

• Rami Komrokji, M.D.

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 25, 2010
• First Posted: May 28, 2010
• Study Start: April 28, 2010
• Primary Completion: January 31, 2015
• Study Completion: November 21, 2019
• Last Updated: December 17, 2019
• Results First Posted: January 18, 2016

Record last verified: 2019-12

Locations

US (2)