LeukemiaPhase 2NCT02469415
Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS)
A Phase 2 study testing Pacritinib, 5-azacitidine and Decitabine 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 learn if pacritinib, either alone or in combination with azacitidine or decitabine, can help to control MDS. The safety of this drug and drug combination will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Pacritinib, 5-azacitidine and Decitabine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2015to 3 Jun 2017
- 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
- Signed informed consent indicating that patients are aware of the investigational nature of this study, in keeping with the policies of MD Anderson Cancer Center (MDACC), must be obtained prior to any study specific procedures.
- Patients with a histologically confirmed diagnosis of MDS by World Health Organization (WHO) classification, and lower-risk MDS as defined by the IPSS classification (Low or Int-1 disease) or R-IPSS classification (Very Low or Low) are eligible. Patients with MDS/MPD overlap syndromes including CMML are also eligible if they have Low or Int-1 disease per IPSS. Patients may have received MDS-directed therapy (i.e. lenalidomide), although patients with prior exposure to hypomethylating agents (e.g. 5-azacitidine or decitabine) are not eligible.
- The interval from prior treatment to time of study drug administration is at least 1 week (except for hydroxyurea or steroid therapy) with recovery from all prior therapy-related toxicities
- Age >/= 18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Adequate liver function, as evidence by serum bilirubin </= 2x the laboratory normal range (except for patients with Gilbert's Disease) or an aspartate aminotransferase (AST) or alanine aminotransferase (ALT) of </= 2.5x the upper limit of normal (ULN) or </= 5x ULN if hepatic disease involvement is present as determined by the investigator.
- Serum creatinine (Cr) </= 2x ULN or 24-hour creatinine clearance >/=50 ml/min
- Subjects of reproductive potential must agree to the use of acceptable contraceptive methods for the duration of the time on study and a further 6 months after completion of treatment. Women of childbearing potential must have a negative blood or urine pregnancy test within 72 hours of start of treatment.
Who cannot join
- Subjects with any prior exposure to the hypomethylating agents (5-azacitidine or decitabine) are excluded.
- Subjects with any prior exposure to JAK2 inhibitor therapy (i.e. ruxolitinib or prior pacritinib therapy) are excluded.
- Any prior or coexisting medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study.
- Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures.
- Active uncontrolled serious infection or sepsis at study enrollment. Patients receiving antibiotics for infections that are under control may be included in the study.
- Gastrointestinal disorders that may significantly interfere with absorption of study drug.
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- Subjects have received potent CYP3A inhibitors within 7 days prior to the initiation of study treatment.
- History of myocardial infarction, severe/unstable angina, or symptomatic congestive heart failure (New York Heart Failure (NYHA) Class III or IV congestive heart failure) within 6 months prior to study enrollment or Left ventricular ejection fraction (LVEF) <50%
- Impaired cardiac function including ongoing cardiac dysrhythmias of Grade > 2, ejection fraction < 50%, atrial fibrillation of any grade, or QTc prolongation > 450 ms, or other factors that increase the risk of QT prolongation (i.e. family history of long QT interval syndrome, hypokalemia defined as serum potassium < 3.0 mEq/L)
- Diagnosis of other malignancies within the last 3 years other than curatively treated non-melanoma skin cancer, carcinoma in situ of the cervix, organ-confined or treated non-metastatic prostate cancer, in situ breast carcinoma after complete surgical resection, or superficial transitional cell bladder carcinoma.
- Known active Hepatitis A, B or C.
- Known HIV seropositivity.
- Women who are pregnant or lactating.
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 II Study of Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes
The goal of this clinical research study is to learn if pacritinib, either alone or in combination with azacitidine or decitabine, can help to control MDS.
The safety of this drug and drug combination will also be studied.
Read the detailed description
Study Drug Administration:
Each cycle is 28 days.
If you are found to be eligible to take part in this study, you will take pacritinib by mouth 2 times each day during Cycles 1-4. Each dose should be about 12 hours apart (1 dose in the morning, 1 dose in the evening).
After Cycle 4, if the study doctor thinks it is in your best interest, you may be able to continue taking pacritinib in combination with either azacitidine or decitabine. The study doctor will tell you which drug you will receive. The study doctor will tell you which drug you will receive. Decitabine and azacitidine may be administered by local doctor or at MD Anderson. Cycle 1 of Part 2 will be administered at MD Anderson. Commercial supplies of decitabine and azacitidine will be used.
You will receive either azacitidine by vein over about 1 hour on Days 1-5 of Cycles 5 and beyond or decitabine by vein over about 1 hour or as an injection under the skin on Days 1-7 of Cycles 5 and beyond.
You should return any unused study drug and/or any empty bottles to each study visit.
Study Visits:
One (1) time each week during Cycle 1 and then on Day 1 of each cycle after that, blood (about 1½ teaspoons) will be drawn for routine tests. You may have this blood drawn at a local lab or clinic closer to your home, if the study doctor thinks this is acceptable. The results from the blood draw will be sent to the study doctor.
On Day 28 (+/- 5 days) of Cycles 1 and 4, you will have a bone marrow aspiration/biopsy to check for genetic mutations and cytogenetic testing. If you begin receiving pacritinib in combination with either azacitidine or decitabine, you will also have this test repeated at Cycle 4 of your combination therapy.
On Day 1 of Cycle 1, Day 28 of Cycles 1 and 4, and at any time the doctor thinks it is needed, you will have an EKG.
Length of Treatment:
You may continue taking pacritinib for up to 4 cycles. If the doctor thinks it is in your best interest, you may be eligible to continue taking the study drug in combination with either azacitidine or decitabine for as long as the doctor thinks it is in your best interest.
You will no longer be able to take the study drug(s) 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-treatment visit.
After the end of therapy and/or 30 days after your last dose of study drug, the study staff will follow your health status by phone call every 2 months (+/- 2 months) until you receive another cancer treatment.
End-of-Treatment Visit:
About 28 days after the last dose of study drug(s):
- Blood (about 1½ teaspoons) will be drawn for routine tests.
- If the doctor thinks it is needed, you will have a bone marrow aspirate/biopsy to check the status of the disease.
This is an investigational study. Pacritinib is not FDA approved or commercially available. It is currently being used for research purposes only. Azacitidine and decitabine are both FDA approved and commercially available for the treatment of MDS. The study doctor can explain how the study drugs are designed to work.
Up to 40 participants will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Pacritinib, 5-azacitidine, Decitabine
- Keywords
- Leukemia, Myelodysplastic syndromes, MDS, Lower-risk, Pacritinib, 5-azacitidine, Azacitidine, 5-azacytidine, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816, Azacytidine, Decitabine, Dacogen
- 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.
PacritinibDrug
Part 1: Pacritinib 200 mg taken by mouth twice daily.
Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined therapy.
5-azacitidineDrug
Also called Azacitidine, 5-azacytidine, 5-aza, Vidaza
Part 2 Starting Dose of 5-azacitidine: 75 mg/m2 by vein on Days 1 - 5 of Cycles 5 and beyond.
DecitabineDrug
Also called Dacogen
Part 2 Starting Dose of Decitabine: 20 mg/m2 by vein on on Days 1 - 7 of Cycles 5 and beyond.
| Study group | Role | What they receive |
|---|---|---|
| Pacritinib + Azacitidine or Decitabine | Experimental | Part 1: Pacritinib 200 mg taken by mouth twice daily. Study cycles administered every 28 days.
Part 2: After 4 cycles of treatment, Pacritinib combined with 5-azacitidine or Decitabine. Pacritinib decreased to 200 mg in morning and 100 mg in evening for first cycle of combined therapy with Pacritinib increased to 200 mg twice a day on subsequent cycles of combined therapy. Those with disease progression prior to 4 cycles of Pacritinib may initiate Pacritinib + HMA study portion prior to completion of 4 cycles. Starting dose of either 5-azacitidine 75 mg/m2 by vein (IV) or Decitabine 20 mg/m2 IVon Days 1 - 5 of Cycles 5 and beyond. Receives: Drug: Pacritinib; Drug: 5-azacitidine; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
The primary efficacy outcome of both parts is the overall response rate (ORR) based mainly on hematologic improvement defined by (International Working Group) IWG-2006 criteria, and which also includes complete remission (CR), partial remission (PR) and marrow complete remission.
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2015 (estimated)
- Study start30 Sep 2015
- Primary completion3 Jun 2017
- Study completion3 Jun 2017
Why it stopped: FDA Clinical Hold
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 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- CTI BioPharma
- Responsible party
- Sponsor
- Investigators
- Courtney DiNardo, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02469415
- Sponsor's protocol ID
- 2013-0224
- Other IDs
- NCI-2015-01306
- Status last verified
- May 2018
- First submitted
- 10 Jun 2015
- Last updated
- 16 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT02469415
- Acronym / sponsor ID
- 2013-0224
- Conditions
- Leukemia
- Keywords
- Leukemia, Myelodysplastic syndromes, MDS, Lower-risk, Pacritinib, 5-azacitidine, Azacitidine, 5-azacytidine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: FDA Clinical Hold
- Enrolment
- 3 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- CTI BioPharma
- Study start
- 30 Sep 2015 (actual)
- Primary completion
- 3 Jun 2017 (actual)
- Study completion
- 3 Jun 2017 (actual)
- First posted
- 11 Jun 2015 (estimated)
- Results
- Posted 23 Apr 2018
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 learn if pacritinib, either alone or in combination with azacitidine or decitabine, can help to control MDS.
The safety of this drug and drug combination will also be studied.
Detailed description
Study Drug Administration:
Each cycle is 28 days.
If you are found to be eligible to take part in this study, you will take pacritinib by mouth 2 times each day during Cycles 1-4. Each dose should be about 12 hours apart (1 dose in the morning, 1 dose in the evening).
After Cycle 4, if the study doctor thinks it is in your best interest, you may be able to continue taking pacritinib in combination with either azacitidine or decitabine. The study doctor will tell you which drug you will receive. The study doctor will tell you which drug you will receive. Decitabine and azacitidine may be administered by local doctor or at MD Anderson. Cycle 1 of Part 2 will be administered at MD Anderson. Commercial supplies of decitabine and azacitidine will be used.
You will receive either azacitidine by vein over about 1 hour on Days 1-5 of Cycles 5 and beyond or decitabine by vein over about 1 hour or as an injection under the skin on Days 1-7 of Cycles 5 and beyond.
You should return any unused study drug and/or any empty bottles to each study visit.
Study Visits:
One (1) time each week during Cycle 1 and then on Day 1 of each cycle after that, blood (about 1½ teaspoons) will be drawn for routine tests. You may have this blood drawn at a local lab or clinic closer to your home, if the study doctor thinks this is acceptable. The results from the blood draw will be sent to the study doctor.
On Day 28 (+/- 5 days) of Cycles 1 and 4, you will have a bone marrow aspiration/biopsy to check for genetic mutations and cytogenetic testing. If you begin receiving pacritinib in combination with either azacitidine or decitabine, you will also have this test repeated at Cycle 4 of your combination therapy.
On Day 1 of Cycle 1, Day 28 of Cycles 1 and 4, and at any time the doctor thinks it is needed, you will have an EKG.
Length of Treatment:
You may continue taking pacritinib for up to 4 cycles. If the doctor thinks it is in your best interest, you may be eligible to continue taking the study drug in combination with either azacitidine or decitabine for as long as the doctor thinks it is in your best interest.
You will no longer be able to take the study drug(s) 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-treatment visit.
After the end of therapy and/or 30 days after your last dose of study drug, the study staff will follow your health status by phone call every 2 months (+/- 2 months) until you receive another cancer treatment.
End-of-Treatment Visit:
About 28 days after the last dose of study drug(s):
- Blood (about 1½ teaspoons) will be drawn for routine tests.
- If the doctor thinks it is needed, you will have a bone marrow aspirate/biopsy to check the status of the disease.
This is an investigational study. Pacritinib is not FDA approved or commercially available. It is currently being used for research purposes only. Azacitidine and decitabine are both FDA approved and commercially available for the treatment of MDS. The study doctor can explain how the study drugs are designed to work.
Up to 40 participants will take part in this study. All will be enrolled 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 |
|---|---|---|
| Pacritinib + Azacitidine or Decitabine | Experimental | Drug: Pacritinib, Drug: 5-azacitidine, Drug: DecitabinePart 1: Pacritinib 200 mg taken by mouth twice daily. Study cycles administered every 28 days. Part 2: After 4 cycles of treatment, Pacritinib combined with 5-azacitidine or Decitabine. Pacritinib decreased to 200 mg in morning and 100 mg in evening for first cycle of combined therapy with Pacritinib increased to 200 mg twice a day on subsequent cycles of combined therapy. Those with disease progression prior to 4 cycles of Pacritinib may initiate Pacritinib + HMA study portion prior to completion of 4 cycles. Starting dose of either 5-azacitidine 75 mg/m2 by vein (IV) or Decitabine 20 mg/m2 IVon Days 1 - 5 of Cycles 5 and beyond. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Signed informed consent indicating that patients are aware of the investigational nature of this study, in keeping with the policies of MD Anderson Cancer Center (MDACC), must be obtained prior to any study specific procedures.
- Patients with a histologically confirmed diagnosis of MDS by World Health Organization (WHO) classification, and lower-risk MDS as defined by the IPSS classification (Low or Int-1 disease) or R-IPSS classification (Very Low or Low) are eligible. Patients with MDS/MPD overlap syndromes including CMML are also eligible if they have Low or Int-1 disease per IPSS. Patients may have received MDS-directed therapy (i.e. lenalidomide), although patients with prior exposure to hypomethylating agents (e.g. 5-azacitidine or decitabine) are not eligible.
- The interval from prior treatment to time of study drug administration is at least 1 week (except for hydroxyurea or steroid therapy) with recovery from all prior therapy-related toxicities
- Age >/= 18 years old.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
Key exclusion criteria (13 listed)
- Subjects with any prior exposure to the hypomethylating agents (5-azacitidine or decitabine) are excluded.
- Subjects with any prior exposure to JAK2 inhibitor therapy (i.e. ruxolitinib or prior pacritinib therapy) are excluded.
- Any prior or coexisting medical condition that in the investigator's judgment will substantially increase the risk associated with the subject's participation in the study.
- Psychiatric disorders or altered mental status precluding understanding of the informed consent process and/or completion of the necessary study procedures.
- Active uncontrolled serious infection or sepsis at study enrollment. Patients receiving antibiotics for infections that are under control may be included in 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 |
|---|---|---|
| PacritinibPart 1: Pacritinib 200 mg taken by mouth twice daily. Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined therapy. | Drug | Pacritinib + Azacitidine or Decitabine |
| 5-azacitidineAlso: Azacitidine, 5-azacytidine, 5-aza, VidazaPart 2 Starting Dose of 5-azacitidine: 75 mg/m2 by vein on Days 1 - 5 of Cycles 5 and beyond. | Drug | Pacritinib + Azacitidine or Decitabine |
| DecitabineAlso: DacogenPart 2 Starting Dose of Decitabine: 20 mg/m2 by vein on on Days 1 - 7 of Cycles 5 and beyond. | Drug | Pacritinib + Azacitidine or Decitabine |
Primary outcome
Overall Response Rate (ORR)
Time frame: 28 days
The primary efficacy outcome of both parts is the overall response rate (ORR) based mainly on hematologic improvement defined by (International Working Group) IWG-2006 criteria, and which also includes complete remission (CR), partial remission (PR) and marrow complete remission.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Courtney DiNardo, 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 2 study testing Pacritinib, 5-azacitidine and Decitabine 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 8 inclusion and 13 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: stopped early, as of the sponsor's last update on 16 Oct 2018. 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 23 Apr 2018). 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?
Overall Response Rate (ORR) (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 Oct 2018.