Myelodysplastic SyndromePhase 1NCT02312102
Velcade and Lenalidomide in Patients With Relapsed AML and MDS After Allogeneic Stem Cell Transplantation
A Phase 1 study testing Lenalidomide and Velcade in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Massachusetts General Hospital at 2 sites in United States.
Trial summary
This research study is evaluating drugs called bortezomib and lenalidomide as a possible treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The purpose of this research study is to determine the safety and efficacy of the bortezomib and lenalidomide investigational combination. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- What is being tested?
- Lenalidomide and Velcade
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 2 sitesUnited States
- Study dates
- Feb 2015to 7 Oct 2024
- Phase
- Phase 1
- 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
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- A diagnosis of recurrent, persistent, or progressive acute myelogenous leukemia (AML), defined as >= 5% blasts in a patient with known prior history of AML, or recurrent, persistent, or progressive myelodysplastic syndrome (MDS) according to WHO criteria.
- Must have undergone an allogeneic SCT (regardless of stem cell source)
- Patients must be 18 years or older
- Able to adhere to study schedule and other protocol requirements
- Must be off all immunosuppressive medications (except prednisone) for at least 2 weeks prior to study entry.
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- Must be on less than 21 mg of oral prednisone daily for GVHD
- ECOG performance status 0-2 (see Appendix 2)
- Participants must have the following organ function all within 21 days prior to enrollment
- Total bilirubin ≤ 2.0 mg/dl unless due to underlying conjugation disease such as Gilbert's
- ALT and AST ≤ 3X the upper limit of normal
- Creatinine < 2.0 mg/dl
- Patients may receive hydroxyurea or leukopheresis as necessary
- Patients must give voluntary written informed consent and HIPA authorization before performance of any study-related procedure not part of normal medical care with the understanding that consent maybe withdrawn by the subject at any time without prejudice to future medical care.
- All previous cancer therapy including donor lymphocyte infusions must have been discontinued at least 2 weeks prior to treatment in this study.
- All study participants must be registered in the RevREMS® program and be willing and able to comply with the requirements of REMS® program.
- Females of reproductive potential must adhere to the scheduled pregnancy testing as required in the Revlimid REMS® program.
Who cannot join
- Participants who exhibit any of the following conditions at screening will not be eligible for admission into the study.
- Ejection fraction < 40% obtained by either MUGA or echocardiogram
- Patients who had had a myocardial infarction within 6 months of enrollment or have New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening has to be documented by the investigator as not medically relevant.
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the consent form.
- Any condition, including laboratory abnormalities, that in the opinion of the investigator places the subject at an unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Patients with major surgery within 28 days prior to trial enrollment
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- Patients with greater than or equal to grade 2 peripheral neuropathy or active herpes infection
- Patients with ≥ grade 3 acute graft-versus-host disease are excluded from the study
- Patients with moderate or severe chronic graft-versus host requiring more than 20 mg of oral prednisone therapy are excluded from the study.
- Patients with uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, cirrhosis, chronic obstructive or restrictive pulmonary disease, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia
- Patients with any serious or medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of treatment according to this protocol
- Female subject is pregnant or breast-feeding. Lactating females must agree not to breastfeed while taking lenalidomide.
- Patient has received an investigational drug within 14 days of enrollment
- Known hypersensitivity to thalidomide or lenalidomide
- Known hypersensitivity to Velcade, boron, or mannitol
- The development of erythema nodosum as characterized by desquamating rash while taking thalidomide or similar drugs
- Concurrent use of other anti-cancer agents or treatment
- Known positive for HIV
- Diagnosed or treated for a non-hematologic malignancy within 2 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy.
- Radiation therapy within 3 weeks before randomization. Enrollment of subjects who require concurrent radiotherapy (which must be localized in its field size) should be deferred until the radiotherapy is completed and 3 weeks have elapsed since the last date of therapy.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
About this study
Official title: Phase I Dose Escalation Study of Velcade in Combination With Lenalidomide in Patients With Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) After Allogeneic Stem Cell Transplantation
This research study is evaluating drugs called bortezomib and lenalidomide as a possible treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).
The purpose of this research study is to determine the safety and efficacy of the bortezomib and lenalidomide investigational combination. This drug combination has been used in the treatment of relapsed/refractory multiple myeloma and has been previously investigated in the treatment of MDS and AML, albeit at a lower dose of lenalidomide. In this research study, the investigators are looking for the highest dose of the combination that can be given safely and see how well it works as a combination for MDS and AML in individuals whose disease has relapsed after an SCT.
Read the detailed description
- After the screening procedures confirm participation in the research study:
- The participant will be given a study drug-dosing calendar for each treatment cycle. The investigators are looking for the highest dose of the combination of study drugs that can be administered safely without severe or unmanageable side effects in participants that have, not everyone who participates in this research study will receive the same dose of the study drug. The dose given will depend on the number of participants who have been enrolled in the study prior and how well the dose was tolerated.
- Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.
- Each treatment cycle lasts 28 days (4 weeks). The first two cycles are called the induction cycles. If the participant respond to treatment during the first two cycles, they can continue on to the maintenance cycles.
- Lenalidomide
- Bortezomib:
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Being tested
- Lenalidomide, Velcade
- Keywords
- myelodysplastic syndrome, acute myeloid leukemia, allogeneic stem cell transplantation
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LenalidomideDrug
Also called Revlimid
Chemotherapy
VelcadeDrug
Also called bortezomib
Chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| Velcade and Lenalidomide | Experimental | Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.
Each treatment cycle lasts 28 days (4 weeks). The first two cycles are called the induction cycles. If the participant respond to treatment during the first two cycles, they can continue on to the maintenance cycles.
During the induction and maintenance cycles, patients receive up to the MTD of lenalidomide on days 1-21 days only. During days 22-28 (4th week) there is a rest period.
Bortezomib: During the induction cycles, the medication will be given on days 2, 5, 9, and 12 followed by a 17-day rest period. During the maintenance cycles, bortezomib will be given on days 2, and 5 followed by 23-day rest period. Receives: Drug: Lenalidomide; Drug: Velcade |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD of Bortezomib and Lenalidomide,
Maximum tolerated dose
Measured: 28 days
Secondary outcomes (5)
Assess clinical efficacy in terms of the number of patients experiencing a decrease in blast percentage.
Change in blasts during treatment
Measured: 2 Years
Document the remission frequency in patients receiving up to 2 induction cycles of high-dose lenalidomide combined with Velcade.
Remission rate
Measured: Day 42
Percentage of Complete Response
Overall response rate
Measured: Baseline, Day 84
Percentage of Participants with Disease Free Survival( DFS )
Disease free survival
Measured: 2 Years
Percentage of Overall Survival
Overall survival
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2014 (estimated)
- Study startFeb 2015
- Primary completionApr 2017
- Study completion7 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation, Millennium Pharmaceuticals, Inc.
- Responsible party
- Principal investigator, Andrew Mark Brunner, MD, Massachusetts General Hospital
- Investigators
- Andrew M Brunner, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02312102
- Sponsor's protocol ID
- 14-213
- Status last verified
- Jan 2026
- First submitted
- 5 Dec 2014
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Dose Escalation Study of Velcade in Combination With Lenalidomide in Patients With Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) After Allogeneic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT02312102
- Acronym / sponsor ID
- 14-213
- Conditions
- Myelodysplastic Syndrome, Acute Myeloid Leukemia
- Keywords
- myelodysplastic syndrome, acute myeloid leukemia, allogeneic stem cell transplantation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Celgene Corporation, Millennium Pharmaceuticals, Inc.
- Study start
- Feb 2015 (actual)
- Primary completion
- Apr 2017 (actual)
- Study completion
- 7 Oct 2024 (actual)
- First posted
- 9 Dec 2014 (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 research study is evaluating drugs called bortezomib and lenalidomide as a possible treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).
The purpose of this research study is to determine the safety and efficacy of the bortezomib and lenalidomide investigational combination. This drug combination has been used in the treatment of relapsed/refractory multiple myeloma and has been previously investigated in the treatment of MDS and AML, albeit at a lower dose of lenalidomide. In this research study, the investigators are looking for the highest dose of the combination that can be given safely and see how well it works as a combination for MDS and AML in individuals whose disease has relapsed after an SCT.
Detailed description
- After the screening procedures confirm participation in the research study:
- The participant will be given a study drug-dosing calendar for each treatment cycle. The investigators are looking for the highest dose of the combination of study drugs that can be administered safely without severe or unmanageable side effects in participants that have, not everyone who participates in this research study will receive the same dose of the study drug. The dose given will depend on the number of participants who have been enrolled in the study prior and how well the dose was tolerated.
- Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation.
- Each treatment cycle lasts 28 days (4 weeks). The first two cycles are called the induction cycles. If the participant respond to treatment during the first two cycles, they can continue on to the maintenance cycles.
- Lenalidomide
- Bortezomib:
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Velcade and Lenalidomide | Experimental | Drug: Lenalidomide, Drug: VelcadeDose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation and de-escalation. Each treatment cycle lasts 28 days (4 weeks). The first two cycles are called the induction cycles. If the participant respond to treatment during the first two cycles, they can continue on to the maintenance cycles. During the induction and maintenance cycles, patients receive up to the MTD of lenalidomide on days 1-21 days only. During days 22-28 (4th week) there is a rest period. Bortezomib: During the induction cycles, the medication will be given on days 2, 5, 9, and 12 followed by a 17-day rest period. During the maintenance cycles, bortezomib will be given on days 2, and 5 followed by 23-day rest period. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- A diagnosis of recurrent, persistent, or progressive acute myelogenous leukemia (AML), defined as >= 5% blasts in a patient with known prior history of AML, or recurrent, persistent, or progressive myelodysplastic syndrome (MDS) according to WHO criteria.
- Must have undergone an allogeneic SCT (regardless of stem cell source)
- Patients must be 18 years or older
- Able to adhere to study schedule and other protocol requirements
Key exclusion criteria (20 listed)
- Participants who exhibit any of the following conditions at screening will not be eligible for admission into the study.
- Ejection fraction < 40% obtained by either MUGA or echocardiogram
- Patients who had had a myocardial infarction within 6 months of enrollment or have New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities. Prior to study entry, any ECG abnormality at screening has to be documented by the investigator as not medically relevant.
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the consent form.
- Any condition, including laboratory abnormalities, that in the opinion of the investigator places the subject at an unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
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 |
|---|---|---|
| LenalidomideAlso: RevlimidChemotherapy | Drug | Velcade and Lenalidomide |
| VelcadeAlso: bortezomibChemotherapy | Drug | Velcade and Lenalidomide |
Primary outcome
MTD of Bortezomib and Lenalidomide,
Time frame: 28 days
Maximum tolerated dose
5 secondary outcomes
Assess clinical efficacy in terms of the number of patients experiencing a decrease in blast percentage.
Time frame: 2 Years
Document the remission frequency in patients receiving up to 2 induction cycles of high-dose lenalidomide combined with Velcade.
Time frame: Day 42
Percentage of Complete Response
Time frame: Baseline, Day 84
Percentage of Participants with Disease Free Survival( DFS )
Time frame: 2 Years
Percentage of Overall Survival
Time frame: 2 Years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
- Individual participant dataSharing planned: Undecided
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Myelodysplastic Syndromes
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.
Andrew M Brunner, MD principal investigator
Massachusetts General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Lenalidomide and Velcade in people with Myelodysplastic Syndrome and Acute Myeloid Leukemia, run by Massachusetts General Hospital at 2 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 17 inclusion and 20 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 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 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.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
MTD of Bortezomib and Lenalidomide, (time frame: 28 days). 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 16 Jan 2026; AzTrials last refreshed its copy on 7 Nov 2026.