Velcade in Myelodysplastic Syndrome - Pilot Study
|First Received Date ICMJE||February 23, 2007|
|Last Updated Date||November 7, 2008|
|Start Date ICMJE||August 2006|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||to determine activity of Velcade in patients with MDS, intermediate-2 or high risk, and intermediate-1 or low risk unresponsive or not eligible for treatment with erythropoietin or immunosuppressive agents as assessed according to response criteria.|
|Original Primary Outcome Measures ICMJE||Same as current|
|Change History||Complete list of historical versions of study NCT00440076 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
||to determine whether Velcade prolongs time to progression and/or survival; safety and tolerability as assessed by incidence of clinical and laboratory toxicities; quality of life in relation to neurotoxicity.|
|Original Secondary Outcome Measures ICMJE||Same as current|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Velcade in Myelodysplastic Syndrome - Pilot Study|
|Official Title ICMJE||Phase II Study of PS341 (VELCADE) in Myelodysplastic Syndromes (MDS). EudraCT Number 2004-002935-23|
This is a four-center open-label study designed to determine activity of Velcade in Myelodysplastic Syndrome (MDS) patients. A total of 28 subjects will be enrolled. The patients will be registered to GIMEMA Data Center before therapy starts and after inclusion criteria verification.
The Myelodysplastic Syndromes (MDS) are an heterogeneous group of clonal disorders of the hematopoietic stem cell characterized by ineffective hematopoiesis leading to peripheral cytopenias, and variable risk of progression to more advanced disease and/or transformation to acute myeloid leukemia (AML). The disease affects predominantly elderly individuals (median age 69 years); the overall incidence is about 4 per 100,000 individuals but this rises to > 30 per 100,000 in the over 70 year age population.
Therapy for MDS has included hematopoietic growth factors (in primis EPO, in combination or not with granulocyte-colony stimulating factor- G-CSF or granulocyte-colony stimulating factor-GM-CSF), differentiating agents, immunotherapy, low dose chemotherapy strategies, AML-like induction regimens, traditional cytotoxic agents, and hematopoietic stem cell transplantation (HSCT) strategies. With the exception of HSCT, which can result in long-term survival in 23% to 50% of patients (Ref. 10) therapy for MDS has not consistently shown a survival advantage, and also modest results have been obtained in an attempt to improve anemia and/or the other cytopenias, and to decrease the number of red blood cell (RBC) transfusions. Furthermore, MDS is primarily a disease of older patients who cannot tolerate aggressive therapy and therefore cannot receive HSCT.
No therapy has thus been considered standard for MDS, and supportive therapy, including RBC transfusions, platelet transfusions and antibiotic therapy has often been focus of care, especially in the older patient population.
VELCADE™ (bortezomib) for injection is a small molecule proteasome inhibitor developed by Millennium Pharmaceuticals, Inc., (MPI) as a novel agent to treat human malignancies. VELCADE is currently approved by the US FDA for the treatment of multiple myeloma patients who have received at least 2 prior therapies and have demonstrated disease progression on the last therapy. VELCADE is a modified dipeptidyl boronic acid derived from leucine and phenylalanine; its chemical name is N pyrazinecarbonyl L phenylalanine L leucine boronic acid and has a molecular weight of 384.25 daltons.
Data from non clinical and clinical studies conducted to date support the development of Velcade for the treatment of human malignancies.
|Study Type ICMJE||Interventional|
|Study Phase||Phase 2|
|Study Design ICMJE||Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Intervention ICMJE||Drug: Velcade|
|Study Arm (s)||Not Provided|
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Withdrawn|
|Estimated Enrollment ICMJE||28|
|Estimated Completion Date||March 2008|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages||18 Years to 75 Years|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Location Countries ICMJE||Italy|
|NCT Number ICMJE||NCT00440076|
|Other Study ID Numbers ICMJE||MDS0104|
|Has Data Monitoring Committee||Not Provided|
|Responsible Party||Not Provided|
|Study Sponsor ICMJE||Gruppo Italiano Malattie EMatologiche dell'Adulto|
|Collaborators ICMJE||Not Provided|
|Information Provided By||Gruppo Italiano Malattie EMatologiche dell'Adulto|
|Verification Date||November 2008|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP