Myelodysplastic SyndromePhase 2NCT01178814
Revlimid in Transfusion Dependent Patients
A Phase 2 study testing Revlimid (Lenalidomide) in people with Myelodysplastic Syndrome, run by Columbia University at 1 site in United States.
Trial summary
This is a prospective trial of Revlimid for subjects who have a blood cell cancer called myelodysplastic syndrome (MDS). Cells in their marrow make proteins through messages that are carried from the genes. The amount of the message tells researchers if the protein it is going to make is high or low. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- Revlimid (Lenalidomide)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jun 2009to 7 Dec 2022
- Phase
- Phase 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 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Understand and voluntarily sign an informed consent form.
- Age ≥ 21 years at the time of signing the informed consent form.
- Able to adhere to the study visit schedule and other protocol requirements.
- A confirmed diagnosis (using standard FAB criteria) of a myelodysplastic syndrome with low/Int-1 risk and with a non-del(5q) karyotype must be established.
- Patients must have transfusion dependence (at least 2 units within 8 weeks prior to starting therapy).
- All transfusion dependent non-del(5q) low/Int-1 risk patients will be eligible for treatment with Revlimid®
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- Newly diagnosed as well as previously treated patients will be eligible
- Patients with primary de novo or secondary MDS will be eligible
- All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study.
- ECOG performance status of 0-2 at study entry (see Appendix II).
- Laboratory test results within these ranges:
- Absolute neutrophil count > 250/uL
- Platelet count > 30,000/uL
- Serum creatinine ≤ 2.0 mg/dL
- Total bilirubin ≤ 1.5 mg/dL
- AST (SGOT) and ALT (SGPT) ≤ 3 x ULN
- BUN ≤ 2 x ULN
- Disease free of prior malignancies for ≥ 2 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
- All study participants must be registered into the mandatory RevAssist® program, and be willing and able to comply with the requirements of RevAssist®.
- Females of childbearing potential (FCBP)† must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to and again within 24 hours of prescribing lenalidomide (prescriptions must be filled within 7 days) and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy.
Who cannot join
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
- Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking lenalidomide).
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Any clinically significant cardiac disease, including congestive heart failure
- Liver function studies including SGOT/SGPT > 3 x ULN
- Clinically significant renal disease.
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- Any previous chemotherapy, hematopoietic growth factors, erythropoietin, or cytokines within 4 weeks of starting treatment. Note: prior therapy with G-CSF within 4 weeks is allowed.
- Use of any other experimental drug or therapy within 28 days of baseline.
- Known hypersensitivity to thalidomide.
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
- Any prior use of lenalidomide. (Patients with prior therapy with thalidomide will be eligible as long as at least 4 weeks have elapsed between end of the drug and accrual on the present trial)
- Concurrent use of other anti-cancer agents or treatments.
- Known positive for HIV or infectious hepatitis, type B or C.
- Life expectancy < 3 months
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: A Prospective Trial of Revlimid® in Transfusion Dependent Patients With Non-del (5q) Low/Intermediate-1 Risk Myelodysplastic Syndrome
This is a prospective trial of Revlimid for subjects who have a blood cell cancer called myelodysplastic syndrome (MDS). Cells in their marrow make proteins through messages that are carried from the genes. The amount of the message tells researchers if the protein it is going to make is high or low. This is known as expression of genes. The purpose of this study is to conduct a prospective trial testing the idea that expression of specific genes can help to predict which patients will respond to study drug administration with Revlimid (lenalidomide).
Read the detailed description
The first Revlimid® study reported by List et al included all transfusion dependent MDS patients, but given the early experience which showed a better response in del(5q) patients and those with lower risk disease, more patients belonging to this category were entered on the trial. The subsequent multi-center studies were focused entirely on the low/Int-1 patients who were transfusion dependent. In the present study, we would like to recapitulate the initial study by only treating the transfusion dependent, low and intermediate-1 risk MDS patients without deletion (5q). Since the predictive assay is most applicable for the non-del(5q) patients, and to avoid confusion resulting from an admixture of patients with del(5q) for whom the drug is already FDA approved, we have elected to restrict this clinical trial to only the non-del(5q) patients.
In the present study, only the non-del(5q) transfusion dependent, low and intermediate-1 risk MDS patients will be treated with single agent Revlimid®. All patients will have their pre-therapy bone marrows studied by gene expression microarray, Luminex bead assay, and real-time PCR.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- Revlimid (Lenalidomide)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Revlimid (Lenalidomide)Drug
Also called Revlimid
The starting dose will be 10 mg days 1-28 with the first dose reduction going to 5 mg days 1-28 of a 28 day cycle. Therapy will cease if there is no response after 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Revlimid | Experimental | Revlimid (Lenalidomide) capsule taken orally once a day Receives: Drug: Revlimid (Lenalidomide) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Signature in Transfusion Dependent MDS Patients
The trial will be testing the Revlimid® (lenalidomide) response signature in patients who have transfusion dependent, non-del(5q), low and intermediate-1 risk myelodysplastic syndromes (MDS) in order to confirm the predictive value of the signature and to establish the boundaries of the z-score which can be used to pre-select patients in future clinical studies. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions.
Measured: Up to 12 weeks from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2010 (estimated)
- Study start1 Jun 2009
- Primary completion1 Sep 2022
- Study completion7 Dec 2022
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.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Mark Heaney, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01178814
- Sponsor's protocol ID
- AAAF2696
- Status last verified
- Jun 2024
- First submitted
- 6 Aug 2010
- Last updated
- 24 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Trial of Revlimid® in Transfusion Dependent Patients With Non-del (5q) Low/Intermediate-1 Risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT01178814
- Acronym / sponsor ID
- AAAF2696
- Conditions
- Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- 1 Jun 2009 (actual)
- Primary completion
- 1 Sep 2022 (actual)
- Study completion
- 7 Dec 2022 (actual)
- First posted
- 10 Aug 2010 (estimated)
- Results
- Posted 20 Jul 2021
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 prospective trial of Revlimid for subjects who have a blood cell cancer called myelodysplastic syndrome (MDS). Cells in their marrow make proteins through messages that are carried from the genes. The amount of the message tells researchers if the protein it is going to make is high or low. This is known as expression of genes. The purpose of this study is to conduct a prospective trial testing the idea that expression of specific genes can help to predict which patients will respond to study drug administration with Revlimid (lenalidomide).
Detailed description
The first Revlimid® study reported by List et al included all transfusion dependent MDS patients, but given the early experience which showed a better response in del(5q) patients and those with lower risk disease, more patients belonging to this category were entered on the trial. The subsequent multi-center studies were focused entirely on the low/Int-1 patients who were transfusion dependent. In the present study, we would like to recapitulate the initial study by only treating the transfusion dependent, low and intermediate-1 risk MDS patients without deletion (5q). Since the predictive assay is most applicable for the non-del(5q) patients, and to avoid confusion resulting from an admixture of patients with del(5q) for whom the drug is already FDA approved, we have elected to restrict this clinical trial to only the non-del(5q) patients.
In the present study, only the non-del(5q) transfusion dependent, low and intermediate-1 risk MDS patients will be treated with single agent Revlimid®. All patients will have their pre-therapy bone marrows studied by gene expression microarray, Luminex bead assay, and real-time PCR.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Revlimid | Experimental | Drug: Revlimid (Lenalidomide)Revlimid (Lenalidomide) capsule taken orally once a day |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Understand and voluntarily sign an informed consent form.
- Age ≥ 21 years at the time of signing the informed consent form.
- Able to adhere to the study visit schedule and other protocol requirements.
- A confirmed diagnosis (using standard FAB criteria) of a myelodysplastic syndrome with low/Int-1 risk and with a non-del(5q) karyotype must be established.
- Patients must have transfusion dependence (at least 2 units within 8 weeks prior to starting therapy).
Key exclusion criteria (14 listed)
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
- Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking lenalidomide).
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Any clinically significant cardiac disease, including congestive heart failure
- Liver function studies including SGOT/SGPT > 3 x ULN
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 |
|---|---|---|
| Revlimid (Lenalidomide)Also: RevlimidThe starting dose will be 10 mg days 1-28 with the first dose reduction going to 5 mg days 1-28 of a 28 day cycle. Therapy will cease if there is no response after 12 weeks. | Drug | Revlimid |
Primary outcome
Response Signature in Transfusion Dependent MDS Patients
Time frame: Up to 12 weeks from baseline
The trial will be testing the Revlimid® (lenalidomide) response signature in patients who have transfusion dependent, non-del(5q), low and intermediate-1 risk myelodysplastic syndromes (MDS) in order to confirm the predictive value of the signature and to establish the boundaries of the z-score which can be used to pre-select patients in future clinical studies. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Mark Heaney, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Revlimid (Lenalidomide) in people with Myelodysplastic Syndrome, run by Columbia University at 1 site 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 20 inclusion and 14 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 24 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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 20 Jul 2021). 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?
Response Signature in Transfusion Dependent MDS Patients (time frame: Up to 12 weeks from baseline). 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 24 Jun 2024.