LeukemiaPhase 1NCT01895842
Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
A Phase 1 study testing Ruxolitinib in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with low or intermediate-1 risk MDS. The safety of this drug will also be studied, and whether it can help to control the disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Ruxolitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2014to 2 Apr 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
- Patients with previously treated low or intermediate-1 risk MDS by the IPSS classification (this is defined in table 1)
- Patients must have one of the following: elevated b2-microglobulin levels (defined as 2 times compared to normal), carry a JAK2 mutation, or presence of phosphorylated p65 NF-kB component in at least 5% of bone marrow cells.
- Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC.
- Age >/= 18 years old
- Prior therapy with growth factor support, lenalidomide, 5-azacytidine, decitabine or other investigational agents are allowed. A four week wash out period will be required before receiving study medication.
- Patients must have the following non-hematologic values Aspartate aminotransferase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) </= 2.5 x Upper Limit of Normal (ULN) or </= 5.0 x ULN if hepatic involvement is present; Serum bilirubin </= 2 x ULN; Serum creatinine </= 2 x ULN or 24-hour creatinine clearance >/= 60 ml/min
- Patients with Childbearing potential must agree to use appropriate forms of birth control
Who cannot join
- Previously untreated low or intermediate-1 risk MDS patients because there are approved therapies for these patients.
- Uncontrolled undercurrent illness that in the opinion of the treating physician would contraindicate the use of the drug.
- Patients with active infections including uncontrolled HIV infection, active hepatitis B, C, or any other symptomatic systemic infection requiring active therapy will be excluded from study
- Patients receiving potent CYP3A4 (such as but not limited to boceprevir, clarithromycin, conivaptan, grapefruit juice, indinavir, itraconazole, ketoconazole, lopinavir/ritonavir, mibefradil, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, voriconazole) inhibitors will be excluded from the study.
- Women who are pregnant or lactating.
- Patients with a white blood cell count of more than 30x10^3 K/uL will not be eligible for this study.
- Patients that have received prior allogeneic stem cell transplantation are excluded from this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Phase I Study of Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with low or intermediate-1 risk MDS. The safety of this drug will also be studied, and whether it can help to control the disease.
Read the detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 4 groups of 3-6 participants will be enrolled in Part 1 of the study, and up to 7 participants will be enrolled in Part 2.
If you are enrolled in Part 1, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose of ruxolitinib. The second group of participants will receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of participants will receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of participants will take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond.
If you are enrolled in Part 2, you will receive ruxolitinib at the highest dose that was tolerated in Part 1.
Study Drug Administration:
Each cycle is 28 days.
You will take ruxolitinib by mouth 2 times a day (about 12 hours apart), with or without food.
Study Visits:
One (1) time a week during Cycles 1 and 2 and then on Day 1 of Cycles 3 and beyond, blood (about 2-3 teaspoons) will be drawn for routine tests. On Day 28 of each cycle and Day 1 of Cycles 3 and beyond, this blood may also be used for cytogenetic testing if your doctor thinks it is needed.
On Day 28 of Cycles 1 and 2 and then every 3 cycles, you will have a bone marrow aspiration to check the status of the disease and for cytogenetic testing, if your doctor thinks it is needed.
On Day 28 of Cycle 1 and Day 1 of Cycles 3 and beyond, you will have a physical exam.
If the doctor thinks it is needed, urine will be collected on Day 1 of each cycle for routine tests.
Length of Dosing:
You may continue taking the study drug for up to 2 years. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the end-of-dosing visit.
End-of-Dosing Visit:
After you are finished taking the study drug:
- You will have a physical exam.
- You will have a bone marrow aspiration to check the status of the disease.
- Blood (about 2-3 teaspoons) will be drawn for routine tests. This blood may also be used for cytogenetic testing if your doctor thinks it is needed.
Follow-up Visit:
About 30 days after the End-of-Dosing visit, a member of the study team will contact you by phone. You will be asked how you are feeling and about any side effects you may be experiencing. This phone call should take 10-15 minutes.
This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of myelofibrosis. Its use in this study is investigational. The study doctor can explain how ruxolitinib is designed to work.
Up to 31 participants will be enrolled in this study. All will take part at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Ruxolitinib
- Keywords
- Leukemia, Myelodysplastic syndrome, MDS, Low or intermediate-1 risk, Maximum tolerated dose, MTD, Ruxolitinib, Jakafi, INCB018424, INC424
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called Jakafi, INCB018424, INC424
Starting dose level for Part 1: 5 mg by mouth twice a day for a 28 day cycle.
Starting dose level for Part 2: Maximum tolerated dose from Part 1.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib | Experimental | Part 1: Dose of ruxolitinib received will depend when patient joined study. The first group of patients receive the lowest dose of ruxolitinib. Starting dose level for Part 1, 5 mg by mouth twice a day for a 28 day cycle. The second group of patients receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of patients receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of patients take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond.
If patients enrolled in Part 2, they will receive ruxolitinib at the highest dose that was tolerated in Part 1. Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) of Ruxolitinib
Maximum tolerated dose (MTD) is the maximum dose at which <33% of patients experience a dose limiting toxicity (DLT). Dose limiting toxicity defined as any grade 3 or higher non-hematologic toxicity.
Measured: 28 days
Secondary outcomes (1)
Number of Participants with Response of Ruxolitinib
Criteria for response follows the modified International Working Group (IWG) response criteria for altering natural history of MDS defined as: Complete Remission, Partial Remission, Stable Disease, Cytogenetic Response, Disease Progression.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2013 (estimated)
- Study startFeb 2014
- Primary completion2 Apr 2019
- Study completion2 Apr 2019
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 LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Guillermo Garcia-Manero, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01895842
- Sponsor's protocol ID
- 2013-0012
- Other IDs
- NCI-2013-02204
- Status last verified
- Feb 2025
- First submitted
- 5 Jul 2013
- Last updated
- 28 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Abaza Y, Hidalgo-Lopez JE, Verstovsek S, Jabbour E, Ravandi F, Borthakur G, Estrov Z, Alvarado Y, Burger J, Schneider H, Soltysiak KA, Wei Y, Kantarjian HM, Bueso-Ramos CE, Garcia-Manero G. Phase I study of ruxolitinib in previously treated patients with low or intermediate-1 risk myelodysplastic syndrome with evidence of NF-kB activation. Leuk Res. 2018 Oct;73:78-85. doi: 10.1016/j.leukres.2018.09.004. Epub 2018 Sep 14.
PubMed 30245189
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT01895842
- Acronym / sponsor ID
- 2013-0012
- Conditions
- Leukemia
- Keywords
- Leukemia, Myelodysplastic syndrome, MDS, Low or intermediate-1 risk, Maximum tolerated dose, MTD, Ruxolitinib, Jakafi
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Incyte Corporation
- Study start
- Feb 2014 (actual)
- Primary completion
- 2 Apr 2019 (actual)
- Study completion
- 2 Apr 2019 (actual)
- First posted
- 11 Jul 2013 (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.
The goal of this clinical research study is to find the highest tolerable dose of ruxolitinib that can be given to patients with low or intermediate-1 risk MDS. The safety of this drug will also be studied, and whether it can help to control the disease.
Detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study. Up to 4 groups of 3-6 participants will be enrolled in Part 1 of the study, and up to 7 participants will be enrolled in Part 2.
If you are enrolled in Part 1, the dose of ruxolitinib you receive will depend on when you joined this study. The first group of participants will receive the lowest dose of ruxolitinib. The second group of participants will receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of participants will receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of participants will take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond.
If you are enrolled in Part 2, you will receive ruxolitinib at the highest dose that was tolerated in Part 1.
Study Drug Administration:
Each cycle is 28 days.
You will take ruxolitinib by mouth 2 times a day (about 12 hours apart), with or without food.
Study Visits:
One (1) time a week during Cycles 1 and 2 and then on Day 1 of Cycles 3 and beyond, blood (about 2-3 teaspoons) will be drawn for routine tests. On Day 28 of each cycle and Day 1 of Cycles 3 and beyond, this blood may also be used for cytogenetic testing if your doctor thinks it is needed.
On Day 28 of Cycles 1 and 2 and then every 3 cycles, you will have a bone marrow aspiration to check the status of the disease and for cytogenetic testing, if your doctor thinks it is needed.
On Day 28 of Cycle 1 and Day 1 of Cycles 3 and beyond, you will have a physical exam.
If the doctor thinks it is needed, urine will be collected on Day 1 of each cycle for routine tests.
Length of Dosing:
You may continue taking the study drug for up to 2 years. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the end-of-dosing visit.
End-of-Dosing Visit:
After you are finished taking the study drug:
- You will have a physical exam.
- You will have a bone marrow aspiration to check the status of the disease.
- Blood (about 2-3 teaspoons) will be drawn for routine tests. This blood may also be used for cytogenetic testing if your doctor thinks it is needed.
Follow-up Visit:
About 30 days after the End-of-Dosing visit, a member of the study team will contact you by phone. You will be asked how you are feeling and about any side effects you may be experiencing. This phone call should take 10-15 minutes.
This is an investigational study. Ruxolitinib is FDA approved and commercially available for the treatment of myelofibrosis. Its use in this study is investigational. The study doctor can explain how ruxolitinib is designed to work.
Up to 31 participants will be enrolled in this study. All will take part at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib | Experimental | Drug: RuxolitinibPart 1: Dose of ruxolitinib received will depend when patient joined study. The first group of patients receive the lowest dose of ruxolitinib. Starting dose level for Part 1, 5 mg by mouth twice a day for a 28 day cycle. The second group of patients receive the lowest dose of ruxolitinib for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. The third group of patients receive the higher dose taken by the second group for 1 cycle and if no intolerable side effects are seen, the dose will be increased for Cycles 2 and beyond. The fourth group of patients take the higher dose taken by the third group for 1 cycle and if no intolerable side effects are seen, the dose will increase to the next higher dose for Cycles 2 and beyond. If patients enrolled in Part 2, they will receive ruxolitinib at the highest dose that was tolerated in Part 1. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients with previously treated low or intermediate-1 risk MDS by the IPSS classification (this is defined in table 1)
- Patients must have one of the following: elevated b2-microglobulin levels (defined as 2 times compared to normal), carry a JAK2 mutation, or presence of phosphorylated p65 NF-kB component in at least 5% of bone marrow cells.
- Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC.
- Age >/= 18 years old
- Prior therapy with growth factor support, lenalidomide, 5-azacytidine, decitabine or other investigational agents are allowed. A four week wash out period will be required before receiving study medication.
Key exclusion criteria (7 listed)
- Previously untreated low or intermediate-1 risk MDS patients because there are approved therapies for these patients.
- Uncontrolled undercurrent illness that in the opinion of the treating physician would contraindicate the use of the drug.
- Patients with active infections including uncontrolled HIV infection, active hepatitis B, C, or any other symptomatic systemic infection requiring active therapy will be excluded from study
- Patients receiving potent CYP3A4 (such as but not limited to boceprevir, clarithromycin, conivaptan, grapefruit juice, indinavir, itraconazole, ketoconazole, lopinavir/ritonavir, mibefradil, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, voriconazole) inhibitors will be excluded from the study.
- Women who are pregnant or lactating.
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 |
|---|---|---|
| RuxolitinibAlso: Jakafi, INCB018424, INC424Starting dose level for Part 1: 5 mg by mouth twice a day for a 28 day cycle. Starting dose level for Part 2: Maximum tolerated dose from Part 1. | Drug | Ruxolitinib |
Primary outcome
Maximum Tolerated Dose (MTD) of Ruxolitinib
Time frame: 28 days
Maximum tolerated dose (MTD) is the maximum dose at which <33% of patients experience a dose limiting toxicity (DLT). Dose limiting toxicity defined as any grade 3 or higher non-hematologic toxicity.
1 secondary outcome
Number of Participants with Response of Ruxolitinib
Time frame: 8 weeks
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 28 Feb 2025.
- Derived from this study: PubMed 30245189Abaza Y, Hidalgo-Lopez JE, Verstovsek S, Jabbour E, Ravandi F, Borthakur G, Estrov Z, Alvarado Y, Burger J, Schneider H, Soltysiak KA, Wei Y, Kantarjian HM, Bueso-Ramos CE, Garcia-Manero G. Phase I study of ruxolitinib in previously treated patients with low or intermediate-1 risk myelodysplastic syndrome with evidence of NF-kB activation. Leuk Res. 2018 Oct;73:78-85. doi: 10.1016/j.leukres.2018.09.004. Epub 2018 Sep 14.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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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.
Guillermo Garcia-Manero, MD principal investigator
M.D. Anderson Cancer Center
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 Ruxolitinib in people with Leukemia, run by M.D. Anderson Cancer Center 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 7 inclusion and 7 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 28 Feb 2025. 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 (MTD) of Ruxolitinib (time frame: 28 days). 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 NCT01895842, 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 28 Feb 2025.