Erlotinib Study for Myelodysplastic Syndrome (MDS)

NCT00977548 | Completed Phase 2 Results DMC

• 2 other identifiers: MCC-15961OSI3666s
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to find out what effects, good and/or bad, erlotinib has on the patient and their myelodysplastic syndrome. Erlotinib has been approved by the Food and Drug Administration (FDA) to treat non-small cell lung cancer; however, erlotinib use in this study is considered investigational as the FDA has not approved it for the treatment of myelodysplastic syndrome.

Conditions

Myelodysplastic Syndrome

Keywords

MDS; blood diseases; bone marrow; hematopoietic; leukemia

Outcome Measures

Primary Outcomes (1)

• Combined Overall Response Rate (ORR)

  Best Response Categories: Marrow complete response (CR), Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment; Hematological improvement (HI), Hgb increase by ≥ 1.5 g/dL, Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L, At least 100% increase and an absolute increase of \> 0.5 x 10\^9/L, as defined by the International Working Group (IWG) 2006 criteria.

  Up to 21 Months

Secondary Outcomes (3)

• Median Overall Survival (OS)

  Up to 21 Months

• Median Progression Free Survival (PFS)

  Up to 21 Months

• Leukemia Free Survival (LFS)

  Up to 21 Months

Study Arms (1)

Erlotinib Treatment

EXPERIMENTAL

Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.

Drug: Erlotinib

Interventions

Erlotinib DRUG

Participants took erlotinib at least 1 hour before, or 2 hours after they ate a meal or snack. Participants were advised to take erlotinib at around the same time every day.

Also known as: Tarceva, OSI-774

Erlotinib Treatment

Eligibility Criteria

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

You may qualify if:

• Patients must have an established diagnosis of myelodysplastic syndrome (MDS) and have either: Low or intermediate 1 risk disease by International Prognostic Scoring System (IPSS) for MDS with symptomatic anemia (defined as hemoglobin less than 10.0 g/dl) or transfusion dependent anemia (defined as requiring ≥ 4 units of red blood cells (RBCs) administered with a pretreatment hemoglobin value of ≤ 9 g/dL in the 8 weeks prior to Day 1 of treatment in this study). Patients with anemia must have no response to at least to 6 weeks trial of erythroid stimulating agents (ESA) \[erythropoietin/ darbepoetin\]. Patients with serum erythropoietin levels more than 500 mU/ ml on diagnosis are eligible to the study without erythropoietin/darbepoetin prior treatment. Patients who do not meet anemia criteria are still eligible if they had thrombocytopenia with two or more platelet counts \< 50 x 10\^9/L or a significant clinical hemorrhage requiring platelet transfusions or if they had neutropenia with an absolute neutrophil count (ANC) \< 1 x 10\^9/L; Intermediate-2 or high risk MDS by IPSS.
• Patients ≥ 60 years with Acute Myeloid Leukemia (AML) by WHO classification and myeloblasts percentage 20-30% (RAEB-t by MDS French-American-British (FAB) classification) are eligible for the study if deemed not suitable for induction chemotherapy or declined that option.
• All prior treatment must have been discontinued 28 days prior to Day 1 of treatment in this study except (ESA) and colony stimulating factors where it should be stopped 14 days prior to start therapy on study, and hydroxyurea should be stopped 2 days before.
• Prior bone marrow or stem cell transplant is allowed.
• Secondary or therapy related MDS patients are eligible.
• Patients with chronic myelomonocytic leukemia (CMML) are eligible.
• Patients must have a performance status of 0 - 2 by Zubrod performance status criteria.
• Pretreatment pathology materials must be available for morphologic review. Collection of blood and marrow specimens for pathology review must be completed within 28 days prior to registration.
• No other prior malignancy is allowed except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the patient is currently in complete remission, or any other cancer from which the patient has been disease-free for at least 2 years.
• In calculating days of tests and measurements, the day a test or measurement is done is considered Day 0. Therefore, if a test is done on a Monday, the Monday four weeks later would be considered Day 28. This allows for efficient patient scheduling without exceeding the guidelines. If Day 28 or 60 falls on a weekend or holiday, the limit may be extended to the next working day.
• All patients must be informed of the investigational nature of this study and must sign and give written consent in accordance with institutional and federal guidelines.

You may not qualify if:

• Patients must not have received prior remission induction chemotherapy as treatment for MDS.
• Patients must not be pregnant or nursing because of the potential risks of the drugs used in this study. Women/men of reproductive potential may not participate unless they have agreed to use an effective contraceptive method.
• Patients who are known HIV positive are not eligible for this study.

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Genentech, Inc.: collaborator

Study Sites (1)

H. Lee Moffitt Cancer Center & Research Institute

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Hematologic Diseases; Leukemia

Interventions

Erlotinib Hydrochloride

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hemic and Lymphatic Diseases; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Quinazolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Rami Komrokji, Principal Investigator
• 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: September 14, 2009
• First Posted: September 15, 2009
• Study Start: September 1, 2009
• Primary Completion: June 1, 2012
• Study Completion: June 1, 2012
• Last Updated: September 16, 2013
• Results First Posted: September 16, 2013

Record last verified: 2013-05

Locations

US (1)