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

This study has been withdrawn prior to enrollment.
(Rami Komrokji, MD author of this protocol no longer is at the University of Cincinnati and this study has been withdrawn.)
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by:
University of Cincinnati
ClinicalTrials.gov Identifier:
NCT00570375
First received: December 6, 2007
Last updated: April 16, 2009
Last verified: April 2009
  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 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II of Erlotinib an Epidermal Growth Factor Receptor Inhibitor in the Treatment of Myelodysplastic Syndrome

Resource links provided by NLM:


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
Experimental: Single Arm
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.
Other Names:
  • Erlotinib
  • Tarceva®

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Locations
United States, Ohio
University of Cincinnati
Cincinnati, Ohio, United States, 45267
Sponsors and Collaborators
University of Cincinnati
Genentech, Inc.
Investigators
Principal Investigator: Carl W Siegrist, M.D. University of Cincinnati
  More Information

Additional Information:
No publications provided

Responsible Party: Carl Siegrist, M.D., University of Cincinnati
ClinicalTrials.gov Identifier: NCT00570375     History of Changes
Other Study ID Numbers: 07092512
Study First Received: December 6, 2007
Last Updated: April 16, 2009
Health Authority: United States: Institutional Review Board

Keywords provided by University of Cincinnati:
Blood disease, bone marrow

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Disease
Hematologic Diseases
Neoplasms
Pathologic Processes
Precancerous Conditions
Erlotinib
Mitogens
Enzyme Inhibitors
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protein Kinase Inhibitors

ClinicalTrials.gov processed this record on November 25, 2014