Myelodysplastic SyndromePhase 1NCT03359460
Ibrutinib and Lenalidomide in Treating Patients With Myelodysplastic Syndrome
A Phase 1 study testing Ibrutinib and Lenalidomide in people with Myelodysplastic Syndrome and Previously Treated Myelodysplastic Syndrome, run by University of California, Davis at 1 site in United States.
Trial summary
This phase I trial studies the side effects and best dose of ibrutinib when giving together with lenalidomide in treating patients with myelodysplastic syndrome. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome, Refractory High Risk Myelodysplastic Syndrome
- What is being tested?
- Ibrutinib and Lenalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2017to 17 Nov 2019
- 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
- Pathologically confirmed diagnosis of MDS by World Health Organization (WHO) criteria (including secondary and therapy-related disease) who have failed standard therapy, who are intolerant of prior therapy, or who refuse standard therapy; any prior therapy, including ibrutinib and/or lenalidomide (unless intolerant of one or both of these medications), is permitted
- Hypomethylating agent failure is defined as disease progression or stable disease as best response to an adequate course of treatment (at least four cycles) with an injectable hypomethylating agent (azacitidine or decitabine)
- International Prognostic Scoring System (IPSS)-revised (R) intermediate, high or very high risk disease
- No specific hematologic parameters for study entry are required; transfusion-dependent patients are eligible and platelet counts should be maintained greater than 10,000/mm^3
- Serum aspartate transaminase (AST) or alanine transaminase (ALT) less than or equal to 3.0 x upper limit of normal (ULN)
- Estimated creatinine clearance greater than or equal to 60 ml/min (Cockcroft-Gault)
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- Bilirubin less than or equal to 1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
- Prothrombin time (PT)/international normalized ratio (INR) less than or equal to 1.5 x ULN and partial thromboplastin time (PTT) (activated [a]PTT) less than or equal to 1.5 x ULN
- Karnofsky performance status (KPS) performance status of 60% or greater
- Ability to understand and willingness to sign an informed consent form
- Ability to adhere to the study visit schedule and other protocol requirements
- Female subjects who are of non-reproductive potential (i.e., post-menopausal by history - no menses for >= 1 year; OR history of hysterectomy; OR history of bilateral tubal ligation; OR history of bilateral oophorectomy); or, female subjects of childbearing potential must have a negative serum pregnancy test upon study entry
- Male and female subjects who agree to use highly effective methods of birth control (e.g., condoms, implants, injectables, combined oral contraceptives, some intrauterine devices [IUDs], sexual abstinence, or sterilized partner) during the period of therapy and for 30 days after the last dose of study drug
- Females of reproductive potential must adhere to the scheduled pregnancy testing as required in the Revlimid Risk Evaluation and Mitigation Strategies (REMS) program
- All study participants must be registered into the mandatory Revlimid REMS program, and be willing and able to comply with the requirements of the REMS program
- Eligibility of patients receiving any medications or substances known to affect or with the potential to affect the activity or pharmacokinetics of ibrutinib or ability to adhere to the Revlimid REMS program will be determined following review of their case by the principal investigator
Who cannot join
- Anticancer therapy including chemotherapy, immunotherapy, radiotherapy, hormonal or any investigational therapy within 14 days or 5 half-lives (whichever is shorter) prior to first dose of study drug; hydroxyurea is permitted up to the day before initiation of study treatment
- Prior allogeneic (allo)-hematopoietic cell transplantation (HCT) less than three months from the time of enrollment
- Acute graft versus host disease (GVHD) or active chronic GVHD greater than grade 1
- Concurrent systemic immunosuppressant therapy (e.g., cyclosporine A, tacrolimus, etc., or chronic administration [> 14 days] of > 60 mg/day of prednisone or equivalent) within 28 days of the first dose of study drug
- History of allergy to or intolerance of ibrutinib or lenalidomide
- Vaccinated with live, attenuated vaccines within 4 weeks of first dose of study drug
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- Recent culture-documented infection requiring systemic intravenous treatment that was completed =< 7 days before the first dose of study drug or any uncontrolled active systemic infection; fever of unknown origin is not an exclusion criterion, as this may be disease-related
- Unresolved toxicities from prior anti-cancer therapy, defined as having not resolved to Common Terminology Criteria for Adverse Event (CTCAE, version 4.03), grade 2 or less, or to the levels dictated in the inclusion/exclusion criteria with the exception of alopecia
- Known bleeding disorders (e.g., von Willebrand's disease) or hemophilia
- History of stroke or intracranial hemorrhage within 3 months prior to enrollment
- Active infection with human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV); subjects who are positive for hepatitis B core antibody, hepatitis B surface antigen, or hepatitis C antibody must have a negative polymerase chain reaction (PCR) result before enrollment; those who are PCR positive will be excluded; subjects with HIV must have a CD4 count at or above the institutional lower limit of normal and not taking prohibited CYP3A strong inhibitors
- Major surgery within 4 weeks of first dose of study drug
- Any life-threatening illness, medical condition, or organ system dysfunction, including but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, active autoimmune disorder, or psychiatric illness/social situations that, in the investigator's opinion, could compromise the subject's safety or put the study outcomes at undue risk
- History of other malignancies, except for malignancy surgically resected (or treated with other modalities) with curative intent, adequately treated in situ carcinoma of the breast or cervix uteri, basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin
- Concomitant use of warfarin or other vitamin K antagonists
- Subjects who received a strong cytochrome P 450 3A (CYP3A) inhibitor within 7 days prior to the first dose of ibrutinib or subjects who require continuous treatment with a strong CYP3A inhibitor
- Currently active, clinically significant hepatic impairment Child-Pugh class B or C according to the Child Pugh classification
- Lactating or pregnant patients or patients of reproductive potential not willing to use effective methods of contraception and adhere to the Revlimid REMS program
- Participation in clinical trials with other investigational agents not included in this trial throughout the duration of this trial
- Unwilling or unable to participate in all required study evaluations and procedures or unable to understand the purpose and risks of the study and to provide a signed and dated informed consent form (ICF) and authorization to use protected health information (in accordance with national and local subject privacy regulations)
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
About this study
Official title: Phase I Trial of the Combination of Ibrutinib and Lenalidomide for the Treatment of Patients With MDS Who Have Failed or Refuse Standard Therapy
This phase I trial studies the side effects and best dose of ibrutinib when giving together with lenalidomide in treating patients with myelodysplastic syndrome. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ibrutinib and lenalidomide may work better in treating patients with myelodysplastic syndrome.
Read the detailed description
PRIMARY OBJECTIVES:
To determine the recommended Phase II dose (RP2D) for ibrutinib in combination with lenalidomide in patients with myelodysplastic syndrome (MDS).
SECONDARY OBJECTIVES:
To do an early assessment of the activity of the combination of ibrutinib and lenalidomide in patients with MDS.
TERTIARY OBJECTIVES:
To examine the pharmacodynamic and biological effects of the combination of ibrutinib and lenalidomide in MDS.
- Conditions
- Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome, Refractory High Risk Myelodysplastic Syndrome, Secondary Myelodysplastic Syndrome, Therapy-Related Myelodysplastic Syndrome
- Being tested
- Ibrutinib, 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.
IbrutinibDrug
Also called BTK Inhibitor PCI-32765, CRA-032765, Imbruvica, PCI-32765
Given PO
LenalidomideDrug
Also called CC-5013, CC5013, CDC 501, Revlimid
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (lenalidomide, ibrutinib) | Experimental | Patients receive lenalidomide PO QD on days 1-21 and ibrutinib PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unaccepted toxicity. Receives: Drug: Ibrutinib; Drug: Lenalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose
Data will be reported in tables with descriptive statistics used. Will be assessed by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Effects (CTCAE) version 4.03. Tables will be created to summarize the toxicities and adverse effects by dose, course, organ and severity as well as the study demographics.
Measured: Up to 28 days
Secondary outcomes (6)
Disease free survival
Will be summarized with Kaplan-Meier plots.
Measured: From the time of first documented complete response until relapse or the date of death from any cause, assessed up to 6 months
Disease response
Will be assessed per modified International Working Group (IWG) 2006 response criteria. Response rates and the corresponding 95% confidence intervals will be obtained.
Measured: Up to 6 months
Hematologic normalization rate
Will be assessed as complete remission + partial remission + hematologic improvement.
Measured: Up to 6 months
Overall survival
Will be summarized with Kaplan-Meier plots.
Measured: From the time of first study drug administration until the date of death from any cause, assessed up to 6 months
Progression free survival
Will be summarized with Kaplan-Meier plots.
Measured: From the time of first study drug administration until the date of progression or death from any cause, assessed up to 6 months
Time to response
Will be summarized with Kaplan-Meier plots.
Measured: From the time of first study drug administration to the date of complete remission, partial remission, or hematologic improvement, assessed up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2017
- Study start1 Dec 2017
- Primary completion29 Aug 2019
- Study completion17 Nov 2019
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 University of California, DavisThe 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
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Collaborators
- Pharmacyclics LLC., Celgene, National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Brian Jonas, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03359460
- Sponsor's protocol ID
- 1043625
- Other IDs
- UCDCC#265, UCDCC#265, P30CA093373, NCI-2017-01827
- Status last verified
- May 2025
- First submitted
- 27 Nov 2017
- Last updated
- 10 Mar 2026
- Results posted
- No
- 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 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial of the Combination of Ibrutinib and Lenalidomide for the Treatment of Patients With MDS Who Have Failed or Refuse Standard Therapy
- ClinicalTrials.gov ID
- NCT03359460
- Acronym / sponsor ID
- 1043625
- Conditions
- Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome, Refractory High Risk Myelodysplastic Syndrome, Secondary Myelodysplastic Syndrome, Therapy-Related Myelodysplastic Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Collaborators
- Pharmacyclics LLC., Celgene, National Cancer Institute (NCI)
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 29 Aug 2019 (actual)
- Study completion
- 17 Nov 2019 (actual)
- First posted
- 2 Dec 2017 (actual)
- 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 phase I trial studies the side effects and best dose of ibrutinib when giving together with lenalidomide in treating patients with myelodysplastic syndrome. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as lenalidomide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving ibrutinib and lenalidomide may work better in treating patients with myelodysplastic syndrome.
Detailed description
PRIMARY OBJECTIVES:
To determine the recommended Phase II dose (RP2D) for ibrutinib in combination with lenalidomide in patients with myelodysplastic syndrome (MDS).
SECONDARY OBJECTIVES:
To do an early assessment of the activity of the combination of ibrutinib and lenalidomide in patients with MDS.
TERTIARY OBJECTIVES:
To examine the pharmacodynamic and biological effects of the combination of ibrutinib and lenalidomide in MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (lenalidomide, ibrutinib) | Experimental | Drug: Ibrutinib, Drug: LenalidomidePatients receive lenalidomide PO QD on days 1-21 and ibrutinib PO QD on days 1-28. Courses repeat every 28 days in the absence of disease progression or unaccepted toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Pathologically confirmed diagnosis of MDS by World Health Organization (WHO) criteria (including secondary and therapy-related disease) who have failed standard therapy, who are intolerant of prior therapy, or who refuse standard therapy; any prior therapy, including ibrutinib and/or lenalidomide (unless intolerant of one or both of these medications), is permitted
- International Prognostic Scoring System (IPSS)-revised (R) intermediate, high or very high risk disease
- No specific hematologic parameters for study entry are required; transfusion-dependent patients are eligible and platelet counts should be maintained greater than 10,000/mm^3
- Serum aspartate transaminase (AST) or alanine transaminase (ALT) less than or equal to 3.0 x upper limit of normal (ULN)
- Estimated creatinine clearance greater than or equal to 60 ml/min (Cockcroft-Gault)
Key exclusion criteria (20 listed)
- Anticancer therapy including chemotherapy, immunotherapy, radiotherapy, hormonal or any investigational therapy within 14 days or 5 half-lives (whichever is shorter) prior to first dose of study drug; hydroxyurea is permitted up to the day before initiation of study treatment
- Prior allogeneic (allo)-hematopoietic cell transplantation (HCT) less than three months from the time of enrollment
- Acute graft versus host disease (GVHD) or active chronic GVHD greater than grade 1
- Concurrent systemic immunosuppressant therapy (e.g., cyclosporine A, tacrolimus, etc., or chronic administration [> 14 days] of > 60 mg/day of prednisone or equivalent) within 28 days of the first dose of study drug
- History of allergy to or intolerance of ibrutinib or lenalidomide
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 |
|---|---|---|
| IbrutinibAlso: BTK Inhibitor PCI-32765, CRA-032765, Imbruvica, PCI-32765Given PO | Drug | Treatment (lenalidomide, ibrutinib) |
| LenalidomideAlso: CC-5013, CC5013, CDC 501, RevlimidGiven PO | Drug | Treatment (lenalidomide, ibrutinib) |
Primary outcome
Maximum tolerated dose
Time frame: Up to 28 days
Data will be reported in tables with descriptive statistics used. Will be assessed by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Effects (CTCAE) version 4.03. Tables will be created to summarize the toxicities and adverse effects by dose, course, organ and severity as well as the study demographics.
6 secondary outcomes
Disease free survival
Time frame: From the time of first documented complete response until relapse or the date of death from any cause, assessed up to 6 months
Disease response
Time frame: Up to 6 months
Hematologic normalization rate
Time frame: Up to 6 months
Overall survival
Time frame: From the time of first study drug administration until the date of death from any cause, assessed up to 6 months
Progression free survival
Time frame: From the time of first study drug administration until the date of progression or death from any cause, assessed up to 6 months
Time to response
Time frame: From the time of first study drug administration to the date of complete remission, partial remission, or hematologic improvement, assessed up to 6 months
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 10 Mar 2026.
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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.
Brian Jonas principal investigator
University of California, Davis
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ibrutinib and Lenalidomide in people with Myelodysplastic Syndrome and Previously Treated Myelodysplastic Syndrome, run by University of California, Davis 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 16 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 10 Mar 2026. 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 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?
Maximum tolerated dose (time frame: Up to 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 10 Mar 2026.