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Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

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ClinicalTrials.gov Identifier: NCT01895842
Recruitment Status : Completed
First Posted : July 11, 2013
Last Update Posted : April 16, 2019
Sponsor:
Collaborator:
Incyte Corporation
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief 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.

Condition or disease Intervention/treatment Phase
Leukemia Drug: Ruxolitinib Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 31 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Study of Ruxolitinib for Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Actual Study Start Date : February 2014
Actual Primary Completion Date : April 2, 2019
Actual Study Completion Date : April 2, 2019


Arm Intervention/treatment
Experimental: Ruxolitinib

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.

Drug: Ruxolitinib

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.

Other Names:
  • Jakafi
  • INCB018424
  • INC424




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Number of Participants with Response of Ruxolitinib [ Time Frame: 8 weeks ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with previously treated low or intermediate-1 risk MDS by the IPSS classification (this is defined in table 1)
  2. 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.
  3. Signed informed consent indicating that patients are aware of the investigational nature of this study in keeping with the policies of UTMDACC.
  4. Age >/= 18 years old
  5. 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.
  6. 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
  7. Patients with Childbearing potential must agree to use appropriate forms of birth control

Exclusion Criteria:

  1. Previously untreated low or intermediate-1 risk MDS patients because there are approved therapies for these patients.
  2. Uncontrolled undercurrent illness that in the opinion of the treating physician would contraindicate the use of the drug.
  3. 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
  4. 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.
  5. Women who are pregnant or lactating.
  6. Patients with a white blood cell count of more than 30x10^3 K/uL will not be eligible for this study.
  7. Patients that have received prior allogeneic stem cell transplantation are excluded from this study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01895842


Locations
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United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Incyte Corporation
Investigators
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Principal Investigator: Guillermo Garcia-Manero, MD M.D. Anderson Cancer Center
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01895842    
Other Study ID Numbers: 2013-0012
NCI-2013-02204 ( Registry Identifier: NCI CTRP )
First Posted: July 11, 2013    Key Record Dates
Last Update Posted: April 16, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by M.D. Anderson Cancer Center:
Leukemia
Myelodysplastic syndrome
MDS
Low or intermediate-1 risk
Maximum tolerated dose
MTD
Ruxolitinib
Jakafi
INCB018424
INC424
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions