MDSPhase 2NCT00440076
Velcade in Myelodysplastic Syndrome - Pilot Study
A Phase 2 study testing Velcade in people with MDS, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 4 sites in Italy.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- MDS
- What is being tested?
- Velcade
- How many people
- 28 participants
- Where
- 4 sitesItaly
- Study dates
- Aug 2006to Mar 2008
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged 18 -75 years.
- Patients with the following clinical diagnosis: RA(FAB) or RCMD (WHO), RARS (FAB) or RCDM-RS(WHO), RAEB (FAB) or RAEB I (WHO), RAEB II (WHO), and:
- IPSS intermediate-2 (score 1.5-2) or high (score 2.5-3) not previously treated with chemotherapy (CT) and not eligible for intensive chemotherapy or allogeneic stem cell transplantation (SCT), or
- IPSS intermediate-1(score 0.5-1) or low (score 0), EPO or immunosuppressive (Cyclosporine, CSA; anti-tymocyte globulin, ATG) therapies resistant or not eligible for these therapies, and transfusion dependent.
- Patients willing and able to comply with the protocol requirements.
- Patients given voluntary written informed consent to participate in the study, with the understanding that consent may be withdrawn at any time without prejudice to future medical care.
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- Male and female patients willing to use acceptable methods for contraception, for the duration of the study.
- Patients with WHO performance Status (PS) 0-2, and life expectancy > 3 months.
- Renal function tests ≤ 2 x upper limit of normal values.
- AST/GOT, ALT/GPT ≤ 2.5 x upper limit of normal values.
- Total bilirubine ≤ 1.5 x upper limit of normal value.
Who cannot join
- Patient has Grade 2 (as defined by the NCI Common Toxicity Criteria-CTC) peripheral neuropathy within 14 days before enrollment.
- Patient has hypersensitivity to bortezomib, boron or mannitol.
- Patient has received prior treatment with bortezomib
- Patient is pregnant or nursing
- Patient has received other investigational drugs within 14 days before enrollment
- Patient has received prior chemotherapy
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- Patient had a major surgery within 4 weeks before enrollment
- Patient had myocardial infarction within 6 months of enrollment or has class III-IV heart failure, uncontrolled angina or arrhythmias
- Patient has been treated for previous malignancy within 5 years before enrollment
- Patient has uncontrolled hypertension or diabetes mellitus
- Patient is known to have HBV or HCV active hepatitis or is human immunodeficiency virus (HIV)-positive
- Patient has systemic infections requiring treatment
Trial locations and contacts
4 sites in 1 country.
Italy4 sites
Istituto di Ematologia e Oncologia Medica L. e A. Seragnoli
Bologna, Bologna
Ospedale Niguarda "Ca Grande"
Milan, Milano
Centro Oncologico Basilicata
Rionero in Vulture, Potenza
A.O Umberto I
Roma, Roma
About this study
Official title: 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.
Read the detailed description
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.
- Conditions
- MDS
- Being tested
- Velcade
- Keywords
- MDS, Velcade
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VelcadeDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Secondary outcomes (1)
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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2007 (estimated)
- Study startAug 2006
- Study completionMar 2008
Why it stopped: Low accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MDSStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Giuliana ALIMENA, MD, PHD, Principal investigator, Università degli Studi di Roma "La Sapienza", Dipartimento di Biotecnologie Cellulari ed Ematolgia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00440076
- Sponsor's protocol ID
- MDS0104
- Status last verified
- Apr 2015
- First submitted
- 23 Feb 2007
- Last updated
- 23 Apr 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of PS341 (VELCADE) in Myelodysplastic Syndromes (MDS). EudraCT Number 2004-002935-23
- ClinicalTrials.gov ID
- NCT00440076
- Acronym / sponsor ID
- MDS0104
- Conditions
- MDS
- Keywords
- MDS, Velcade
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low accrual
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'AdultoUniversity, hospital or other
- Study start
- Aug 2006
- Study completion
- Mar 2008 (actual)
- First posted
- 26 Feb 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patients aged 18 -75 years.
- Patients with the following clinical diagnosis: RA(FAB) or RCMD (WHO), RARS (FAB) or RCDM-RS(WHO), RAEB (FAB) or RAEB I (WHO), RAEB II (WHO), and:
- Patients willing and able to comply with the protocol requirements.
- Patients given voluntary written informed consent to participate in the study, with the understanding that consent may be withdrawn at any time without prejudice to future medical care.
- Male and female patients willing to use acceptable methods for contraception, for the duration of the study.
Key exclusion criteria (12 listed)
- Patient has Grade 2 (as defined by the NCI Common Toxicity Criteria-CTC) peripheral neuropathy within 14 days before enrollment.
- Patient has hypersensitivity to bortezomib, boron or mannitol.
- Patient has received prior treatment with bortezomib
- Patient is pregnant or nursing
- Patient has received other investigational drugs within 14 days before enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Velcade | Drug | – |
Primary outcome
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.
1 secondary outcome
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.
Results and safety
The study was stopped early (reason given: Low accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Giuliana ALIMENA, MD, PHD principal investigator
Università degli Studi di Roma "La Sapienza", Dipartimento di Biotecnologie Cellulari ed Ematolgia
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Velcade in people with MDS, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 4 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Low accrual). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2015.