Multiple MyelomaPhase 3NCT01002248
Assessment of Efficacy and Safety of Perifosine, Bortezomib and Dexamethasone in Multiple Myeloma Patients
A Phase 3 study testing Perifosine, Bortezomib and Dexamethasone in people with Multiple Myeloma, run by AEterna Zentaris at 86 sites in 9 countries.
Trial summary
This is a randomized Phase III study to evaluate the efficacy and safety of perifosine when added to the combination of bortezomib and dexamethasone in multiple myeloma patients who have relapsed on a prior bortezomib treatment regimen. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- Perifosine, Bortezomib and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 135 participants
- Where
- 86 sitesin 9 countries
- Study dates
- Dec 2009to Mar 2013
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient was previously diagnosed with multiple myeloma based on standard diagnostic criteria.
- Patients must have relapsed (progressed > 60 days) after their last dose of bortezomib-based therapy. In addition, patients may be relapsed or refractory to other non-bortezomib-based therapies.
- Patient has received at least 1 but not more than 4 prior anti-myeloma regimens and has progressive disease after the most recent treatment regimen.
- Patients must have adequate organ and marrow function.
Who cannot join
- Patients must not be refractory to any bortezomib-containing regimen.
- History of allergic reactions or intolerance attributed to compounds of similar chemical or biologic composition to perifosine (miltefosine or edelfosine), bortezomib or dexamethasone or any of their components.
- Prior treatment with perifosine or an investigational proteasome inhibitor.
- Chemotherapy or other therapy experimental or proven that is or may be active against myeloma within two weeks (14 days) prior to Cycle 1 Day 1.
Trial locations and contacts
86 sites in 9 countries.
86 sites in 9 countries
United States39 sites
Study site
Aurora, Colorado, 80012
Study site
Baltimore, Maryland, 21201
Study site
Bedford, Texas, 76022
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Study site
Boulder, Colorado, 80303
Study site
Christiansburg, Virginia, 24073
Study site
Colorado Springs, Colorado, 80909
Study site
Columbia, Maryland, 21044
Study site
Dallas, Texas, 75246
Study site
Denver, Colorado, 80218
Study site
Fargo, North Dakota, 58122
Study site
Fort Worth, Texas, 76104
Study site
Great Neck, New York, 11042
Study site
Kerrville, Texas, 78028
Study site
Kingsport, Tennessee, 37660
Study site
La Verne, California, 91750
Study site
Lakewood, Colorado, 80228
Study site
Littleton, Colorado, 80120
Study site
Longmont, Colorado, 80501
Study site
Milwaukee, Wisconsin, 53226
Study site
Morristown, New Jersey, 07960
Study site
Niles, Illinois, 60714
Study site
Ogden, Utah, 84403
Study site
Parker, Colorado, 80138
Study site
Portland, Oregon, 97225
Study site
Pueblo, Colorado, 81008
Study site
Roanoke, Virginia, 24014
Study site
Salem, Virginia, 24153
Study site
San Antonio, Texas, 78217
Study site
San Antonio, Texas, 78229
Study site
San Francisco, California, 94143
Study site
San Pablo, California, 94806
Study site
Seattle, Washington, 98133
Study site
Spokane, Washington, 99202
Study site
Thornton, Colorado, 80260
Study site
Tualatin, Oregon, 97062
Study site
Tyler, Texas, 75702
Study site
Vancouver, Washington, 98686
Study site
Winfield, Illinois, 60190
Spain12 sites
Study site
Badalona, 08916
Study site
Barcelona, 08025
Study site
Barcelona, 08035
Study site
Barcelona, 08036
Study site
Madrid, 28006
Study site
Madrid, 28034
Study site
Madrid, 28040
Study site
Madrid, 28041
Study site
Pamplona, 31008
Study site
San Cristóbal de La Laguna, 38320
Study site
Valencia, 46026
Study site
Zaragoza, 50009
Israel11 sites
Study site
Afula, 18101
Study site
Ashkelon, 78278
Study site
Beersheba, 84101
Study site
Haifa, 31096
Study site
Jerusalem, 91031
Study site
Jerusalem, 91120
Study site
Nahariya, 22100
Study site
Petah Tikva, 49100
Study site
Rehovot, 76100
Study site
Tel Aviv, 64239
Study site
Tel HaShomer, 52621
Ireland7 sites
Keryx / AOI Pharmaceuticals Investigative Site
Dublin
Study site
Dublin
Study site
Galway
Study site
Limerick
Study site
Sligo
Study site
Tullamore
Study site
Waterford
Russia5 sites
Study site
Moscow, 125284
Study site
Moscow, 129110
Study site
Saint Petersburg, 191024
Study site
Saint Petersburg, 197022
Study site
Samara, 443095
South Korea5 sites
Study site
Seoul, 110-744
Study site
Seoul, 120-752
Study site
Seoul, 135-710
Study site
Seoul, 137-701
Study site
Seoul, 138-736
Canada4 sites
Study site
Montreal, Quebec, H1T 2M4
Study site
Montreal, Quebec, H3A 1A1
Study site
Québec, Quebec, G1R 2J6
Study site
Winnipeg, Manitoba, R3E 0V9
Czechia2 sites
Study site
Brno, Czech Republic, 62500
Study site
Prague, Czech Republic, 12821
About this study
Official title: A Phase III Randomized Study to Assess the Efficacy and Safety of Perifosine Added to the Combination of Bortezomib and Dexamethasone in Multiple Myeloma Patients
This is a randomized Phase III study to evaluate the efficacy and safety of perifosine when added to the combination of bortezomib and dexamethasone in multiple myeloma patients who have relapsed on a prior bortezomib treatment regimen.
Read the detailed description
A pre-planned interim analysis is expected to take place in Q1 of 2013.
- Conditions
- Multiple Myeloma
- Being tested
- Perifosine, Perifosine Placebo, Bortezomib, Dexamethasone
- Keywords
- Multiple Myeloma, Relapsed multiple myeloma, Refractory multiple myeloma, Relapsed refractory multiple myeloma
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PerifosineDrug
Also called D-21266, KRX-0401
Perifosine will be dosed as one 50 mg pill every day of each cycle.
Perifosine PlaceboDrug
Perifosine placebo will be dosed as one 50 mg pill every day of each cycle.
BortezomibDrug
Bortezomib will be dosed at 1.3 mg/m2 on Days 1, 4, 8, and 11 every 21 days.
DexamethasoneDrug
Dexamethasone will be administered orally at 20 mg on Days 1, 2, 4, 5, 8, 9, 11, and 12 of each 21-day cycle.
| Study group | Role | What they receive |
|---|---|---|
| Perifosine added to combination | Experimental | Perifosine added to the combination of Bortezomib and Dexamethasone. Perifosine is is supplied as a film-coated tablet containing 50 mg of active ingredient. Perifosine will be administered orally on an outpatient basis throughout the study. Daily administration will be one 50 mg tablet.
The first dose of perifosine should be taken on the same day that bortezomib and dexamethasone are administered (Cycle 1 Day 1). Receives: Drug: Perifosine; Drug: Bortezomib; Drug: Dexamethasone |
| Perifosine Placebo added to combination | Placebo comparator | Perifosine placebo added to the combination of Bortezomib and Dexamethasone. The placebo for perifosine is provided in 256 mg white to off-white, round, biconvex film-coated tablets to permit a blinded trial with perifosine 50 mg film coated tablets. Placebo will be administered orally on an outpatient basis throughout the study. Daily administration will be one perifosine placebo tablet. The first dose of placebo should be taken on the same day that bortezomib and dexamethasone are administered (Cycle 1 Day 1). Receives: Drug: Perifosine Placebo; Drug: Bortezomib; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the PFS (progression free survival) in patients with multiple myeloma, treated with perifosine, bortezomib and dexamethasone compared to patients treated with placebo, bortezomib and dexamethasone
Progression-free survival will be defined as the time between randomization and the date of progression that occurred during the Core Phase.
Measured: 6 - 24 months
Secondary outcomes (3)
Overall survival (OS)
OS is defined as time from randomization to death from any cause during the Core Phase of the study.
Measured: Up to 24 months
Overall response rate (ORR)
The ORR for each treatment arm will be estimated as the proportion of responders, defined as a patient whose best overall response is PR or better during the treatment period, using criteria prospectively established.
Measured: 6 - 24 months
Adverse Events
Each AE and SAE term submitted will be mapped to a preferred term (PT) using the MedDRA dictionary. The investigator will classify the severity of AEs using the NCI CTCAE v3.0 and will assess the relationship of each event to each study treatment.
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2009 (estimated)
- Study startDec 2009
- Primary completionMar 2013
- Study completionMar 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 135 participants (actual)
Sponsor and investigators
- Lead sponsor
- AEterna Zentaris
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Dana-Farber Cancer Institute
- Responsible party
- Sponsor
- Investigators
- Paul Richardson, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01002248
- Sponsor's protocol ID
- Perifosine 339
- Status last verified
- Mar 2013
- First submitted
- 23 Oct 2009
- Last updated
- 9 Feb 2018
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Richardson PG, Nagler A, Ben-Yehuda D, Badros A, Hari P, Hajek R, Spicka I, Kaya H, Le Blanc R, Yoon SS, Kim K, Martinez-Lopez J, Mittelman M, Shpilberg O, Tothova E, Laubach JP, Ghobria IM, Leiba M, Gatt ME, Sportelli P, Chen M, Anderson KC. Randomized Placebo-Controlled Phase III Study Of Perifosine Combined With Bortezomib and Dexamethasone In Relapsed, Refractory Multiple Myeloma Patients Previously Treated With Bortezomib. Blood 2013 ASH Meeting Proceedings;122:abstract 3189
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III Randomized Study to Assess the Efficacy and Safety of Perifosine Added to the Combination of Bortezomib and Dexamethasone in Multiple Myeloma Patients
- ClinicalTrials.gov ID
- NCT01002248
- Acronym / sponsor ID
- Perifosine 339
- Conditions
- Multiple Myeloma
- Keywords
- Multiple Myeloma, Relapsed multiple myeloma, Refractory multiple myeloma, Relapsed refractory multiple myeloma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 135 participants (actual)
- Lead sponsor
- AEterna ZentarisIndustry (pharma, biotech, devices)
- Collaborators
- Dana-Farber Cancer Institute
- Study start
- Dec 2009
- Primary completion
- Mar 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 27 Oct 2009 (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 randomized Phase III study to evaluate the efficacy and safety of perifosine when added to the combination of bortezomib and dexamethasone in multiple myeloma patients who have relapsed on a prior bortezomib treatment regimen.
Detailed description
A pre-planned interim analysis is expected to take place in Q1 of 2013.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Perifosine added to combination | Experimental | Drug: Perifosine, Drug: Bortezomib, Drug: DexamethasonePerifosine added to the combination of Bortezomib and Dexamethasone. Perifosine is is supplied as a film-coated tablet containing 50 mg of active ingredient. Perifosine will be administered orally on an outpatient basis throughout the study. Daily administration will be one 50 mg tablet. The first dose of perifosine should be taken on the same day that bortezomib and dexamethasone are administered (Cycle 1 Day 1). |
| Perifosine Placebo added to combination | Placebo comparator | Drug: Perifosine Placebo, Drug: Bortezomib, Drug: DexamethasonePerifosine placebo added to the combination of Bortezomib and Dexamethasone. The placebo for perifosine is provided in 256 mg white to off-white, round, biconvex film-coated tablets to permit a blinded trial with perifosine 50 mg film coated tablets. Placebo will be administered orally on an outpatient basis throughout the study. Daily administration will be one perifosine placebo tablet. The first dose of placebo should be taken on the same day that bortezomib and dexamethasone are administered (Cycle 1 Day 1). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patient was previously diagnosed with multiple myeloma based on standard diagnostic criteria.
- Patients must have relapsed (progressed > 60 days) after their last dose of bortezomib-based therapy. In addition, patients may be relapsed or refractory to other non-bortezomib-based therapies.
- Patient has received at least 1 but not more than 4 prior anti-myeloma regimens and has progressive disease after the most recent treatment regimen.
- Patients must have adequate organ and marrow function.
Key exclusion criteria (4 listed)
- Patients must not be refractory to any bortezomib-containing regimen.
- History of allergic reactions or intolerance attributed to compounds of similar chemical or biologic composition to perifosine (miltefosine or edelfosine), bortezomib or dexamethasone or any of their components.
- Prior treatment with perifosine or an investigational proteasome inhibitor.
- Chemotherapy or other therapy experimental or proven that is or may be active against myeloma within two weeks (14 days) prior to Cycle 1 Day 1.
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 |
|---|---|---|
| PerifosineAlso: D-21266, KRX-0401Perifosine will be dosed as one 50 mg pill every day of each cycle. | Drug | Perifosine added to combination |
| Perifosine PlaceboPerifosine placebo will be dosed as one 50 mg pill every day of each cycle. | Drug | Perifosine Placebo added to combination |
| BortezomibBortezomib will be dosed at 1.3 mg/m2 on Days 1, 4, 8, and 11 every 21 days. | Drug | Perifosine Placebo added to combination, Perifosine added to combination |
| DexamethasoneDexamethasone will be administered orally at 20 mg on Days 1, 2, 4, 5, 8, 9, 11, and 12 of each 21-day cycle. | Drug | Perifosine Placebo added to combination, Perifosine added to combination |
Primary outcome
Determine the PFS (progression free survival) in patients with multiple myeloma, treated with perifosine, bortezomib and dexamethasone compared to patients treated with placebo, bortezomib and dexamethasone
Time frame: 6 - 24 months
Progression-free survival will be defined as the time between randomization and the date of progression that occurred during the Core Phase.
3 secondary outcomes
Overall survival (OS)
Time frame: Up to 24 months
Overall response rate (ORR)
Time frame: 6 - 24 months
Adverse Events
Time frame: Up to 24 months
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2018.
- Results publicationRichardson PG, Nagler A, Ben-Yehuda D, Badros A, Hari P, Hajek R, Spicka I, Kaya H, Le Blanc R, Yoon SS, Kim K, Martinez-Lopez J, Mittelman M, Shpilberg O, Tothova E, Laubach JP, Ghobria IM, Leiba M, Gatt ME, Sportelli P, Chen M, Anderson KC. Randomized Placebo-Controlled Phase III Study Of Perifosine Combined With Bortezomib and Dexamethasone In Relapsed, Refractory Multiple Myeloma Patients Previously Treated With Bortezomib. Blood 2013 ASH Meeting Proceedings;122:abstract 3189
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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.
Paul Richardson, MD principal investigator
Dana-Farber Cancer Institute
86 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Perifosine, Bortezomib and Dexamethasone in people with Multiple Myeloma, run by AEterna Zentaris at 86 sites in 9 countries.
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: stopped early, as of the sponsor's last update on 9 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 86 sites in United States, Spain, Israel, Ireland, Russia and 4 more countries. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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. 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?
Determine the PFS (progression free survival) in patients with multiple myeloma, treated with perifosine, bortezomib and dexamethasone compared to patients treated with placebo, bortezomib and dexamethasone (time frame: 6 - 24 months). 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 NCT01002248, which also lists 1 linked publication.
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 9 Feb 2018; AzTrials last refreshed its copy on 8 Nov 2026.