AmyloidosisPhase 1/2NCT00298766
Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis
A Phase 1/2 study testing VELCADE in people with Amyloidosis, run by Millennium Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis
- What is being tested?
- VELCADE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 4 sitesUnited States
- Study dates
- Jun 2005to Sep 2009
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or Female 18 y/o and older
- Female patients must be practicing an effective method of birth control
- Biopsy-proven AL-amyloidosis
- Must have been previously treated (failed at least 1 previous treatment) and in the opinion of the physician, patient requires further treatment
Who cannot join
- Hypersensitivity to boron or mannitol
- Prior treatment with VELCADE
- Patient requires other concomitant chemotherapy, radiotherapy or ancillary therapy considered investigational
- Uncontrolled infection
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Winship Cancer Center - Emory Clinic School of Medicine
Atlanta, Georgia, 30322
Boston Medical Center
Boston, Massachusetts, 02118
Cedars-Sinai Medical Center, Samuel Oschin Comprehensive Cancer Institute
Los Angeles, California, 90049
MSKCC
New York, New York, 10017
About this study
Official title: An Open-Label Phase 1/2 Study of VELCADE for Injection in Subjects With Light-Chain (AL)-Amyloidosis
This is a phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.
- Conditions
- Amyloidosis
- Being tested
- VELCADE
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VELCADEDrug
Also called Bortezomib
Once weekly at: 0.7, 1.0, 1.3 or 1.6 mg/m2
Or
Twice-weekly at: 0.7, 1.0, or 1.3 mg/m2
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | VELCADE Receives: Drug: VELCADE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose
Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts.
DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity.
Measured: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts
Subjects With Treatment Emergent Adverse Events
Treatment emergent adverse events observed during outcome measure time frame
Measured: from first study-related procedure to 30 days after last dose of study medication
Subjects With Serious Treatment Emergent Adverse Events
Serious treatment emergent adverse events observed during outcome measure time frame
Measured: from first study-related procedure to 30 days after last dose of study medication
Subjects Grade 3/4/5 Treatment Emergent Adverse Events
Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame.
Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0.
Measured: from first study-related procedure to 30 days after last dose of study medication
Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination
Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination
Measured: from first study-related procedure to 30 days after last dose of study medication
Secondary outcomes (1)
Best Confirmed Hematologic Responders
Hematologic response was determined by the investigator per the response criteria for immunoglobulin light chain amyloidosis by Gertz (2005). It include Complete and Partial Responders (CR+PR). CR requires serum and urine negative for a monoclonal protein by immunofixation and free light chain ratio normal. PR requires: 1. reduction in quantitative serum M-protein by 50% if baseline value is at least 0.5 g/dL, 2. if light chain is detected in the urine (with a consistent peak and >100 mg/ 24 hours), then 50% reduction is required, 3. if free light chain >10 mg/dL, reduction by 50% is required.
Measured: from first dose of study medication to end of study visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2006 (estimated)
- Study startJun 2005
- Primary completionJul 2009
- Study completionSep 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for AmyloidosisStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Millennium Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Millennium Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00298766
- Sponsor's protocol ID
- 26866138-CAN-2007
- Status last verified
- Jun 2012
- First submitted
- 1 Mar 2006
- Last updated
- 25 Jun 2012
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Kukreti V, Vescio RA, Pei L, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood. 2014 Oct 16;124(16):2498-506. doi: 10.1182/blood-2014-04-568329. Epub 2014 Sep 8.
PubMed 25202139Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Vescio RA, Liu K, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study. Blood. 2011 Jul 28;118(4):865-73. doi: 10.1182/blood-2011-02-334227. Epub 2011 May 11.
PubMed 21562045Reece DE, Sanchorawala V, Hegenbart U, Merlini G, Palladini G, Fermand JP, Vescio RA, Liu X, Elsayed YA, Cakana A, Comenzo RL; VELCADE CAN2007 Study Group. Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study. Blood. 2009 Aug 20;114(8):1489-97. doi: 10.1182/blood-2009-02-203398. Epub 2009 Jun 4.
PubMed 19498019
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Phase 1/2 Study of VELCADE for Injection in Subjects With Light-Chain (AL)-Amyloidosis
- ClinicalTrials.gov ID
- NCT00298766
- Acronym / sponsor ID
- 26866138-CAN-2007
- Conditions
- Amyloidosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Millennium Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Study start
- Jun 2005
- Primary completion
- Jul 2009 (actual)
- Study completion
- Sep 2009 (actual)
- First posted
- 3 Mar 2006 (estimated)
- Results
- Posted 13 Aug 2010
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 phase 1/2 open-label, dose-escalation study investigating single-agent therapy with VELCADE in patients with previously treated systemic AL-amyloidosis who require further treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: VELCADEVELCADE |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male or Female 18 y/o and older
- Female patients must be practicing an effective method of birth control
- Biopsy-proven AL-amyloidosis
- Must have been previously treated (failed at least 1 previous treatment) and in the opinion of the physician, patient requires further treatment
Key exclusion criteria (4 listed)
- Hypersensitivity to boron or mannitol
- Prior treatment with VELCADE
- Patient requires other concomitant chemotherapy, radiotherapy or ancillary therapy considered investigational
- Uncontrolled infection
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 |
|---|---|---|
| VELCADEAlso: BortezomibOnce weekly at: 0.7, 1.0, 1.3 or 1.6 mg/m2 Or Twice-weekly at: 0.7, 1.0, or 1.3 mg/m2 | Drug | 1 |
Primary outcome
Maximum Tolerated Dose
Time frame: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts
Maximum Tolerated Dose (MTD) was defined as the highest dose level that has 0/1 out of 6 patients experiences Dose Limited Toxicity (DLT). MTD is defined separately for QW and BIQ dose cohorts. DLT was defined as adverse events occurring during Cycle 1 and: (1) related to VELCADE, (2) Grade 4 thrombocytopenia or neutropenia, (3) Grade 3 or higher nonhematologic toxicity.
Subjects With Treatment Emergent Adverse Events
Time frame: from first study-related procedure to 30 days after last dose of study medication
Treatment emergent adverse events observed during outcome measure time frame
Subjects With Serious Treatment Emergent Adverse Events
Time frame: from first study-related procedure to 30 days after last dose of study medication
Serious treatment emergent adverse events observed during outcome measure time frame
Subjects Grade 3/4/5 Treatment Emergent Adverse Events
Time frame: from first study-related procedure to 30 days after last dose of study medication
Grade 3/4/5 treatment emergent adverse events observed during outcome measure time frame. Grade is determined according to Common Terminology Criteria for Adverse Event (CTCAE) Version 3.0.
Subjects With Treatment Emergent Adverse Events Leading to Treatment Termination
Time frame: from first study-related procedure to 30 days after last dose of study medication
Treatment emergent adverse events observed during outcome measure time frame leading to treatment termination
1 secondary outcome
Best Confirmed Hematologic Responders
Time frame: from first dose of study medication to end of study visit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25202139Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Kukreti V, Vescio RA, Pei L, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Long-term follow-up from a phase 1/2 study of single-agent bortezomib in relapsed systemic AL amyloidosis. Blood. 2014 Oct 16;124(16):2498-506. doi: 10.1182/blood-2014-04-568329. Epub 2014 Sep 8.
- Derived from this study: PubMed 21562045Reece DE, Hegenbart U, Sanchorawala V, Merlini G, Palladini G, Blade J, Fermand JP, Hassoun H, Heffner L, Vescio RA, Liu K, Enny C, Esseltine DL, van de Velde H, Cakana A, Comenzo RL. Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study. Blood. 2011 Jul 28;118(4):865-73. doi: 10.1182/blood-2011-02-334227. Epub 2011 May 11.
- Derived from this study: PubMed 19498019Reece DE, Sanchorawala V, Hegenbart U, Merlini G, Palladini G, Fermand JP, Vescio RA, Liu X, Elsayed YA, Cakana A, Comenzo RL; VELCADE CAN2007 Study Group. Weekly and twice-weekly bortezomib in patients with systemic AL amyloidosis: results of a phase 1 dose-escalation study. Blood. 2009 Aug 20;114(8):1489-97. doi: 10.1182/blood-2009-02-203398. Epub 2009 Jun 4.
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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.
Medical Monitor study director
Millennium Pharmaceuticals, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VELCADE in people with Amyloidosis, run by Millennium Pharmaceuticals, Inc. at 4 sites in United States.
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 4 inclusion and 4 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: finished, as of the sponsor's last update on 25 Jun 2012. 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 United States. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 13 Aug 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Maximum Tolerated Dose (time frame: 5 weeks in once weekly (QW) dose cohorts and 3 weeks in twice weekly (BIW) dose cohorts). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00298766, which also lists 3 linked publications.
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 25 Jun 2012.