Myelodysplastic SyndromePhase 2NCT01133275
Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS
A Phase 2 study testing Prednisone and Lenalidomide in people with Myelodysplastic Syndrome and MDS, run by H. Lee Moffitt Cancer Center and Research Institute at 2 sites in United States.
Trial summary
The purpose of this research is to evaluate the use of lenalidomide and prednisone in people with Myelodysplastic Syndrome (MDS). Lenalidomide is a drug that alters the immune system and it may also interfere with the development of tiny blood vessels that help support tumor growth. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, MDS
- What is being tested?
- Prednisone and Lenalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 2 sitesUnited States
- Study dates
- 28 Apr 2010to 21 Nov 2019
- 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 18 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 ≥ 18 years at the time of signing the informed consent form
- Able to adhere to the study visit schedule and other protocol requirements
- Documented diagnosis of MDS according Word Health Organization (WHO) that meets International Prognostic Scoring System (IPSS) criteria for Low- to Intermediate-1-risk disease or non-proliferative (white blood count [WBC] < 13,000/uL) chronic myelomonocytic leukemia (CMML) and MDS/myeloproliferative neoplasms (MPN).
- Absence of a chromosome 5q deletion by metaphase cytogenetics or fluorescent in situ hybridization (FISH) analysis
- Red blood cell (RBC) transfusion-dependent anemia defined as having received ≥4 transfusions of RBCs for hemoglobin (Hgb) ≤ 9.0 g/dl within 56 days of randomization or symptomatic anemia (Hgb ≤ 9.0 g/dl)
Show all 13Show fewer
- No response or progression on prior treatment with epoetin alpha (> 40,000 U/wk x 6), darbepoetin alpha (≥ 500 mcg q 3 wk x 2) or serum erythropoietin (EPO) concentration ≥ 500 mU/ml
- All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study.
- Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2 at study entry
- Laboratory test results within these ranges: Absolute neutrophil count ≥ 500 /mm³; Platelet count ≥ 50,000 /mm³; Creatinine Clearance > 60 mL/min by Cockcroft-Gault formula; Total bilirubin ≤ 1.5 mg/dl; aspartic transaminase (AST)and alanine transaminase (ALT) ≤ 2 x upper limit of normal (ULN).
- Disease free of prior malignancies for ≥ 3 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "insitu" of the cervix or breast
- 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 milli-international units per milliliter (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 patient from signing the informed consent form.
- Pregnant or breast feeding. (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.
- 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 or history of desquamating (blistering) rash while taking thalidomide
Show all 16Show fewer
- Any prior use of lenalidomide
- Concurrent use of other anti-cancer agents or treatments
- Known positive for human immunodeficiency virus (HIV) or infectious hepatitis, type B or C
- Proliferative CMML (WBC ≥13,000/μL)
- MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
- Prior ≥ grade-2 National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) allergic reaction to thalidomide
- Clinically significant anemia due to factors such as iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis or gastrointestinal bleeding
- Known HIV-1 positivity
- Chromosome 5q deletion
- Documented thromboembolic event within the past 3 years
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Florida Shands Cancer Center
Gainesville, Florida, 32610
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: A Phase II Clinical Trial of Lenalidomide and Prednisone in Low and Intermediate-1 IPSS Risk, Non-del (5q) MDS Patients
The purpose of this research is to evaluate the use of lenalidomide and prednisone in people with Myelodysplastic Syndrome (MDS).
Lenalidomide is a drug that alters the immune system and it may also interfere with the development of tiny blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. Lenalidomide is approved by the U.S. Food and Drug Administration (FDA) for the treatment of specific types of myelodysplastic syndrome (MDS) and in combination with dexamethasone for people with multiple myeloma (MM) who have received at least 1 prior therapy. MDS and MM are cancers of the blood. It is currently being tested in a variety of cancer conditions. As it is being used in this study it is considered an investigational use. An "investigational use" is a use that is being tested and is not approved by the FDA.
Read more
Prednisone is approved by the FDA to treat numerous conditions. In addition, prednisone is approved by the FDA to treat Low or Intermediate-1 IPSS Risk, non-del (5q) MDS.
"Study drug" refers to the combination of lenalidomide and prednisone.
Read the detailed description
10 mg/day of lenalidomide will be taken by mouth on days 1 - 28 of cycles 1-6. Dosing will be in the morning at approximately the same time each day.
Planned prednisone dose:
- 30 mg by mouth daily, days 1-28 of cycle 1
- 20 mg by mouth daily, days 1-28 of cycle 2
- 10 mg by mouth daily, days 1-28 of cycle 3
- 10 mg by mouth every other day on days 1-28 of cycles 4-6
- 5 mg by mouth every other day for responders beyond cycle 6
- Conditions
- Myelodysplastic Syndrome, MDS
- Being tested
- Prednisone, 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.
PrednisoneDrug
Also called Deltasone
Prednisone therapy for 6 cycles (24 weeks).
LenalidomideDrug
Also called REVLIMID®
Lenalidomide therapy for 6 cycles (24 weeks).
| Study group | Role | What they receive |
|---|---|---|
| Lenalidomide and Prednisone Therapy | Experimental | All participants received lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks). Receives: Drug: Prednisone; Drug: Lenalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Erythroid Response
The rate of erythroid response to treatment with the lenalidomide/prednisone combination in non-del (5q) low and int-1 risk Myelodysplastic Syndrome (MDS) with symptomatic anemia. Hematological improvement erythroid response (HI-E) according to International Working Group (IWG) 2006 criteria.
Measured: Up to 7 months
Secondary outcomes (1)
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Grade 3 or 4 events Related/Possibly Related/Probably Related to study treatment. Number of participants with events specified in the study protocol: Neutropenia, Thrombocytopenia, Febrile Neutropenia, Infection, Sepsis, Venous Thromboembolic Events. Evaluations according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) V4.0.
Measured: Up to 54 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2010 (estimated)
- Study start28 Apr 2010
- Primary completion31 Jan 2015
- Study completion21 Nov 2019
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 H. Lee Moffitt Cancer Center and Research InstituteThe 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
- 28 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Rami Komrokji, M.D., Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01133275
- Sponsor's protocol ID
- MCC-16099
- Other IDs
- RV-MDS-PI-0456, 25005/1
- Status last verified
- Dec 2019
- First submitted
- 25 May 2010
- Last updated
- 17 Dec 2019
- 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 17 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Clinical Trial of Lenalidomide and Prednisone in Low and Intermediate-1 IPSS Risk, Non-del (5q) MDS Patients
- ClinicalTrials.gov ID
- NCT01133275
- Acronym / sponsor ID
- MCC-16099
- Conditions
- Myelodysplastic Syndrome, MDS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- 28 Apr 2010 (actual)
- Primary completion
- 31 Jan 2015 (actual)
- Study completion
- 21 Nov 2019 (actual)
- First posted
- 28 May 2010 (estimated)
- Results
- Posted 18 Jan 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research is to evaluate the use of lenalidomide and prednisone in people with Myelodysplastic Syndrome (MDS).
Lenalidomide is a drug that alters the immune system and it may also interfere with the development of tiny blood vessels that help support tumor growth. Therefore, in theory, it may reduce or prevent the growth of cancer cells. Lenalidomide is approved by the U.S. Food and Drug Administration (FDA) for the treatment of specific types of myelodysplastic syndrome (MDS) and in combination with dexamethasone for people with multiple myeloma (MM) who have received at least 1 prior therapy. MDS and MM are cancers of the blood. It is currently being tested in a variety of cancer conditions. As it is being used in this study it is considered an investigational use. An "investigational use" is a use that is being tested and is not approved by the FDA.
Prednisone is approved by the FDA to treat numerous conditions. In addition, prednisone is approved by the FDA to treat Low or Intermediate-1 IPSS Risk, non-del (5q) MDS.
"Study drug" refers to the combination of lenalidomide and prednisone.
Detailed description
10 mg/day of lenalidomide will be taken by mouth on days 1 - 28 of cycles 1-6. Dosing will be in the morning at approximately the same time each day.
Planned prednisone dose:
- 30 mg by mouth daily, days 1-28 of cycle 1
- 20 mg by mouth daily, days 1-28 of cycle 2
- 10 mg by mouth daily, days 1-28 of cycle 3
- 10 mg by mouth every other day on days 1-28 of cycles 4-6
- 5 mg by mouth every other day for responders beyond cycle 6
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lenalidomide and Prednisone Therapy | Experimental | Drug: Prednisone, Drug: LenalidomideAll participants received lenalidomide and prednisone therapy for 6 cycles (24 weeks). Each cycle is 28 days (4 weeks). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Understand and voluntarily sign an informed consent form
- Age ≥ 18 years at the time of signing the informed consent form
- Able to adhere to the study visit schedule and other protocol requirements
- Documented diagnosis of MDS according Word Health Organization (WHO) that meets International Prognostic Scoring System (IPSS) criteria for Low- to Intermediate-1-risk disease or non-proliferative (white blood count [WBC] < 13,000/uL) chronic myelomonocytic leukemia (CMML) and MDS/myeloproliferative neoplasms (MPN).
- Absence of a chromosome 5q deletion by metaphase cytogenetics or fluorescent in situ hybridization (FISH) analysis
Key exclusion criteria (16 listed)
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from signing the informed consent form.
- Pregnant or breast feeding. (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.
- Use of any other experimental drug or therapy within 28 days of baseline
- Known hypersensitivity to thalidomide
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 |
|---|---|---|
| PrednisoneAlso: DeltasonePrednisone therapy for 6 cycles (24 weeks). | Drug | Lenalidomide and Prednisone Therapy |
| LenalidomideAlso: REVLIMID®Lenalidomide therapy for 6 cycles (24 weeks). | Drug | Lenalidomide and Prednisone Therapy |
Primary outcome
Number of Participants With Erythroid Response
Time frame: Up to 7 months
The rate of erythroid response to treatment with the lenalidomide/prednisone combination in non-del (5q) low and int-1 risk Myelodysplastic Syndrome (MDS) with symptomatic anemia. Hematological improvement erythroid response (HI-E) according to International Working Group (IWG) 2006 criteria.
1 secondary outcome
Number of Participants With Grade 3 or 4 Adverse Events Possibly Related to Treatment
Time frame: Up to 54 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Myelodysplastic Syndrome
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Rami Komrokji, M.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
2 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 Prednisone and Lenalidomide in people with Myelodysplastic Syndrome and MDS, run by H. Lee Moffitt Cancer Center and Research Institute 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 13 inclusion and 16 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 17 Dec 2019. 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 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 18 Jan 2016). 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?
Number of Participants With Erythroid Response (time frame: Up to 7 months). 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 17 Dec 2019.