Safety and Anti-Disease Activity of Oral Tosedostat (CHR-2797) in Elderly Subjects With Refractory or Relapsed AML (OPAL)
The purpose of this study is to evaluate the efficacy and safety of tosedostat in elderly patients suffering from refractory or relapsed AML.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||The OPAL Study: A Phase II Study to Evaluate the Efficacy, Safety and Tolerability of Tosedostat (CHR-2797) in Elderly Subjects With Treatment Refractory or Relapsed Acute Myeloid Leukemia|
- The primary objective of the study is to evaluate the efficacy of tosedostat in elderly subjects with treatment refractory or relapsed AML by measuring CR and CRp. [ Time Frame: Months 1, 2, 3 & 6 ] [ Designated as safety issue: No ]
- To evaluate the safety and tolerability of tosedostat in elderly subjects with treatment refractory or relapsed AML [ Time Frame: Screening, Days 1, 2, 8, 15, 29, monthly thereafter + unschedulded visits when deemed necessary ] [ Designated as safety issue: Yes ]
- To evaluate the efficacy of tosedostat in elderly subjects with treatment refractory or relapsed AML, as determined by measures other than CR and CRp for the type and duration of response [ Time Frame: Months 1, 2, 3 & 6 ] [ Designated as safety issue: No ]
|Study Start Date:||October 2009|
|Study Completion Date:||March 2011|
|Primary Completion Date:||March 2011 (Final data collection date for primary outcome measure)|
oral, once daily administration of tosedostat to evaluate its efficacy, safety and tolerability
In Part A, approximately 70 subjects will be randomized to one of 2 dose regimens of tosedostat which will be administered orally, once daily. The dose regimens of tosedostat will be:
In Part B a further 130 subjects will receive the dose regimen of tosedostat identified in Part A as being appropriate, based on the interim analysis during Part A.
There is an urgent need for novel compounds and treatment strategies for elderly patients with AML, particularly those with refractory or relapsed disease for whom there are few effective treatment options. Treatment options for elderly patients are further limited by co-morbidity and tolerability constraints.
Tosedostat is a new aminopeptidase inhibitor, which in preclinical experiments has shown potent activity in both in vitro and in vivo cancer models as a single agent. In early clinical studies particularly good results have been observed in refractory and relapsed AML in older patients and these observations form the basis for the current study.
This multi-center, open label phase II study will enrol approximately 70 subjects in Part A and 130 subjects in Part B.
Show 21 Study Locations
|Principal Investigator:||Jorge E Cortes, MD||M.D. Anderson Cancer Center|
|Principal Investigator:||Karen Yee, MD||Princess Margaret Hospital, Canada|
|Principal Investigator:||Eric Feldman, MD||Weill Cornell Medical College - New York Presbyterian Hospital|
|Principal Investigator:||David Rizzieri, MD||Duke University|
|Principal Investigator:||Joseph Jurcic, MD||Memorial Sloan-Kettering Cancer Center|
|Principal Investigator:||Richard Larson, MD||University of Chicago|
|Principal Investigator:||Hanna J Khoury, MD||Emory University Clinic|
|Principal Investigator:||Harry Erba, MD||University of Michigan Health System|
|Principal Investigator:||Samir Parekh, MD||Montefiore Medical Center|
|Principal Investigator:||Aarthi Shenoy, MD||Washington Hospital Center|
|Principal Investigator:||Anjali Advani, MD||Taussig Cancer Institute|
|Principal Investigator:||Shambavi Richard, MD||Stony Brook University Medical Center|
|Principal Investigator:||Steven Allen, MD||Monter Cancer Center|
|Principal Investigator:||Ehab Attalah, MD||Froedtert Hospital|
|Principal Investigator:||John Storring, MD||Royal Victoria Hospital|
|Principal Investigator:||Gerrit J Ossenkoppele, MD||VUMC|
|Principal Investigator:||Pieter Sonneveld, MD||Erasmus MC|
|Principal Investigator:||Gary Schiller, MD||UCLA Division of Hematology/oncology, Los Angeles|
|Principal Investigator:||Peter Westervelt, MD||Washington University School of Medicine|
|Principal Investigator:||Julio Hajdenberg, MD||MD Anderson Cancer centre, Orlando, FL|
|Principal Investigator:||Stuart Goldberg, MD||John Theurer Cancer Center, Hackensack NJ|