NCT01085838

Erlotinib in Higher Risk Myelodysplastic Syndrome

Phase I-II Trial of Erlotinib in Higher Risk Myelodysplastic Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Groupe Francophone des Myelodysplasies · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The aim of this study is to evaluate the toxicity and therapeutic efficacy of erlotinib in high-risk myelodysplastic syndrome (MDS) patients (with at least 10% of bone marrow blasts) ineligible for or having failed intensive chemotherapy and ineligible or after failure of treatment with a hypomethylating agent.

Detailed description

This is a phase I-II multicenter, open label, sequential cohort dose escalation study of erlotinib designed to assess the safety and efficacy of a daily administration of erlotinib in high risk MDS patients. Five patients per cohort will be enrolled into sequential cohorts receiving increasing dosages of erlotinib. The first cohort of 5 patients will start with a dosage of 100 mg erlotinib daily. Response will be determined after 12 weeks of treatment (or earlier upon major hematologic improvement, whichever event occurs first). At the completion of each cohort, defined as the fifth subject completing the week 12 visit, the safety review panel will be responsible for making the decision as to whether the next cohort will begin, an intermediate dose cohort will be added, or if additional subjects will be enrolled into an earlier dose cohort. Upon agreement of the safety review panel, the second cohort of patients will receive 150 mg of erlotinib daily, and - upon agreement of the safety review panel - the third cohort of five patients will be enrolled to receive 300 mg of erlotinib daily. Since it is to be expected that the therapeutically required dosage of erlotinib is higher than the dosage for which a patient was initially enrolled (i.e. patient enrolled in the first cohort receiving 100 mg daily), dosage of erlotinib should be increased (for the same patient) to the next higher level, if no response is documented after 12 weeks of continuous treatment and no grade III or IV toxicity is documented. In contrast, responders will continue their treatment with the same dosage of erlotinib until grade III or IV toxicity arises or treatment loses efficacy (as defined by relapse/progression of the disease). Consequently, this study plans to enrol 15 patients in 3 cohorts of 5 patients. Once the dose limiting toxicity has been defined, additional confirmatory subjects (20) will be enrolled into the appropriate lower dose as recommended by the safety review panel.

Conditions

• Myelodysplastic Syndrome

Interventions

Type	Name	Description
DRUG	Erlotinib	Erlotinib oral capsule, 100, 150, or 300 mg/day during 12 weeks at study start

Timeline

• Start date: 2010-07-01
• Primary completion: 2014-03-01
• Completion: 2014-03-01
• First posted: 2010-03-12
• Last updated: 2016-11-08

Locations

14 sites across 1 country: France