The Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome - Full Text View

Sponsors and Collaborators:	University of Cincinnati Genentech
Information provided by:	University of Cincinnati
ClinicalTrials.gov Identifier:	NCT00570375

Purpose

The purpose of this research study is to find out what effects, good and/or bad, Erlotinib has on Myelodysplastic syndrome. Myelodysplastic syndrome is a group of blood diseases where the bone marrow (spongy space in long bones which is the factory for blood cell production) does not make enough blood cells and therefore there is a lack of healthy blood cells in the body. This can result in anemia, risk for infection and/or bleeding..

Condition	Intervention	Phase
Myelodysplastic Syndrome	Drug: Erlotinib	Phase II

Drug Information available for: Erlotinib Erlotinib hydrochloride Epidermal Growth Factor

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Phase II of Erlotinib an Epidermal Growth Factor Receptor Inhibitor in the Treatment of Myelodysplastic Syndrome

Further study details as provided by University of Cincinnati:

Primary Outcome Measures:

First 20 patients will be evaluated for overall response rate (CR, PR or HI). [ Time Frame: Estimated to be about 1 year. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

If the first analysis of 20 patients warrant further enrollment, an additional 15 eligible patients will be registered. If 8 or more of the 35 patients achieve CR, PR or HI, then Phase III study will be warranted. [ Time Frame: Estimated to be about 1 year. ] [ Designated as safety issue: No ]

Estimated Enrollment:	35
Study Start Date:	November 2007
Estimated Study Completion Date:	November 2012
Estimated Primary Completion Date:	November 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Single Arm: Experimental All patients will receive 150 mg of Erlotinib	Drug: Erlotinib 150 mg, PO, QD beginning day 1 week 1. Patients will receive treatment for 16 weeks as long as there is no evidence of disease progression. In no response is noted after 16 weeks of treatment, patients will be taken off the study. Patients achieving response (HI, CR, or PR) will continue on treatment until evidence of disease progression or relapse.

Detailed Description:

MDS is a neoplastic clonal stem disorder characterized by bone marrow failure with cytopenia, dyslastic morphological features and tendency to progress to acute myeloid leukemia. It is estimated that MDS is the most common hematological malignancy in the USA. Several treatment options are available for MDS ranging from supportive care, growth factor use, chemotherapy, stem cell transplantation, and newer novel agents such as thalidomide, lenalidomide, and hypomethylating agents. Each of the different available treatments for MDS work in certain subset and relatively small percentage of patients, keeping the door open for novel therapeutic strategies to be explored. The NIH has published requests for applications on myeloproliferative and myelopdysplastic syndrome emphasizing the need for more research in this area.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Patients must have an established diagnosis of myelodysplastic syndrome (MDS) and have either symptomatic anemia (defined as hemoglobin less than 10.0 g/dl) or transfusion dependent anemia (defined as 4 units of blood in the last 60 days).
Patients treated previously with 5-azacytidine, decitabine, thalidomide, revlimid, amifostine, hydroxyurea, Histone deacytlase inhibitors and arsenic trioxide are allowed. Prior treatment with cytokines (i.e., interferon, interleukin), colony stimulating factors, or hydroxyurea is also permitted. Patients should be 28 days off prior treatment.
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
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.

Exclusion Criteria:

Patients must not have undergone bone marrow or stem cell transplant.
Patients must not have received prior remission induction chemotherapy as treatment for MDS.
Patients must not have secondary or therapy related MDS
Patients who are known HIV positive are not eligible for this study.
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.
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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00570375

Contacts

Contact: Tammy Roads	513-558-2192	roadst@ucmail.uc.edu
Contact: Rami S Komrokji, M.D.	513-584-5802	rami.komrokji@uc.edu

Locations

United States, Ohio
University of Cincinnati	Recruiting
Cincinnati, Ohio, United States, 45267
Contact: Jennifer McGuire, R.N. 513-584-2207 mcguirjn@ucmail.uc.edu
Principal Investigator: Rami S Komrokji, M.D.

Sponsors and Collaborators

University of Cincinnati

Genentech

Investigators

Principal Investigator:

Carl W Siegrist, M.D.

University of Cincinnati

More Information

University of Cincinnati Cancer Center This link exits the ClinicalTrials.gov site

Responsible Party:	University of Cincinnati ( Carl Siegrist, M.D. )
Study ID Numbers:	07092512
Study First Received:	December 6, 2007
Last Updated:	October 29, 2008
ClinicalTrials.gov Identifier:	NCT00570375
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Cincinnati:

Blood disease, bone marrow

Study placed in the following topic categories:

Erlotinib
Myelodysplastic syndromes
Preleukemia
Precancerous Conditions

Hematologic Diseases
Myelodysplasia
Myelodysplastic Syndromes
Bone Marrow Diseases

Additional relevant MeSH terms:

Neoplasms
Pathologic Processes
Disease
Molecular Mechanisms of Pharmacological Action

Syndrome
Enzyme Inhibitors
Protein Kinase Inhibitors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 13, 2009