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Trial record 1 of 1 for:    Phase 2 LCL-161 in Patients With Primary Myelofibrosis
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Phase 2 LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF) or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)

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ClinicalTrials.gov Identifier: NCT02098161
Recruitment Status : Recruiting
First Posted : March 27, 2014
Last Update Posted : February 5, 2018
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

March 21, 2014
March 27, 2014
February 5, 2018
December 2014
December 2019   (Final data collection date for primary outcome measure)
Objective Response (OR) of LCL-161 [ Time Frame: After 3, 28 day cycles ]
Objective response (OR), defined as CR (complete remission) + PR (partial remission) + CI (clinical improvement) for myelofibrosis (MF) patients after 3 cycles of treatment. Categorized according to the International Working Group (IWG) consensus criteria for myelofibrosis .
Same as current
Complete list of historical versions of study NCT02098161 on ClinicalTrials.gov Archive Site
Time to Response [ Time Frame: Day 1 of third, 28 day cycle ]
Time to response defined as time from study registration to the first date at which the subject's objective status was classified as a response (CR or PR). In subjects who do not achieve a response, time to response censored at the subject's last evaluation date. The distribution for each of these event-time variables (duration of response and time to response) estimated by Kaplan-Meier curves.
Same as current
Not Provided
Not Provided
 
Phase 2 LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF) or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)
Open Label Phase 2 Single Agent Study of LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)
To goal of this clinical research study is to learn if LCL161 can help to control myelofibrosis. The safety of this drug will also be studied.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will take LCL161 by mouth on Days 1, 8, 15, and 22 of each cycle, +/- 3 days. You will fast for 2 hours before and 2 hours after taking LCL161. This means you will have no food during this time. You will be able to have about 8 ounces of fluids such as water, milk, or non-citrus juices.

Each cycle is 28 days.

If you vomit a dose of LCL161, do not make up the dose.

On Day 1 of Cycle 1, you will take your first dose of study drug at the clinic. You will stay at the clinic for 4 hours after your first dose of study drug.

After your first dose, you will take the study drug at home. You should bring any unused study drug with you to each study visit. It is very important that you take the study drug just as the doctor tells you to. Do not miss any tablets.

If the doctor thinks it is needed, you will be given standard drugs to help decrease the risk of side effects. You may ask the study staff for information about how the drugs are given and their risks.

Study Visits:

On Day 1 of Cycle 1, you will have a physical exam, if you have not had a screening physical exam within 3 days of Day 1 of Cycle 1.

On Day 1 of Cycles 2, 3, 4, 7 and 10, and then every 6 cycles (Cycle 16, 22 etc.) thereafter while participating in the study:

  • You will have a physical exam
  • Blood (about 2-3 teaspoons) will be drawn for routine tests.
  • If your doctor thinks it is needed, urine will be collected for routine tests.
  • You will complete the symptom questionnaires.

If your doctor agrees, you may have your blood drawn at your local doctor's office or laboratory. You will not have to fill out the questionnaires at your local doctor's office. The study staff will also call you every 4-6 weeks. You will be asked how you are feeling and about any side effects you may be having. This phone call and/or email should take about 10-15 minutes. You will return to MD Anderson at the start of Cycles 1, 2, 3, 4, 7, and 10, and every 6 months thereafter while participating in the study.

On Day 1 of Cycle 4 and then every 6 cycles (Cycles 10, 16, and so on), you will have a bone marrow biopsy and/or aspirate to check the status of the disease and for cytogenetic testing.

If the doctor thinks it is needed, you may be asked to return to the clinic more often.

Length of Treatment:

You may continue taking the study drug for as long as the doctor thinks it is in your best interest. 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 follow-up call.

End-of-Treatment Visit:

Within 7 days after you are finished taking the study drug:

  • You will have a physical exam.
  • Blood (about 2-3 teaspoons) will be drawn for routine tests.
  • You will complete the symptom questionnaires.

Follow-up Call:

About 30 days after the end-of-treatment visit, the study staff will call you to ask how you are feeling and about any side effects you may be having. This phone call and/or email should take about 10-15 minutes.

This is an investigational study. LCL161 is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work.

Up to 50 participants will be enrolled in this study. All will take part at MD Anderson.

Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Leukemia
  • Drug: LCL-161
    Starting dose of LCL-161 1500 mg by mouth on Days 1, 8, 15, and 22 of each 28 day cycle.
  • Behavioral: Questionnaires
    Questionnaire completion at baseline, day 1 of cycle 2 and beyond, and at end of treatment visit. The questionnaires should take about 5-10 minutes to complete.
    Other Name: Surveys
  • Other: Phone Calls
    Study staff to call participant 1 time each month, and at 30 days after end of treatment visit.
Experimental: LCL-161
Starting dose of LCL-161 1500 mg by mouth on Days 1, 8, 15, and 22 of each 28 day cycle. Participants remain on study treatment, in the absence of disease progression or toxicity warranting discontinuation of therapy, as long as there is evidence of clinical benefit, as judged by the treating physician.
Interventions:
  • Drug: LCL-161
  • Behavioral: Questionnaires
  • Other: Phone Calls
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
40
December 2020
December 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Patients must provide written informed consent.
  2. Age 18 years or older.
  3. Willing and able to comply with scheduled visits, treatment plan and laboratory tests
  4. Patient is able to swallow and retain oral medication
  5. Must be diagnosed with treatment requiring PMF or post ET/PV MF with intermediate-1, intermediate -2 or high risk disease according to the IWG prognostic scoring system, or if with low risk disease then with symptomatic splenomegaly that is >/=5 cm below left costal margin by physical exam.
  6. Patients who are not candidates for, intolerant, or relapsed/refractory to Ruxolitinib
  7. ECOG performance status 0-2
  8. Required baseline laboratory status: Absolute neutrophil count (ANC) >/= 0.5 x 109/L (1500/mm3); Serum direct bilirubin </= 2.0 x ULN (upper limit of normal); AST (SGOT) and ALT (SGPT) </= 2.5 x ULN, except for patients with MF involvement of the liver who must have AST and ALT </= 5 x ULN; Serum creatinine </= 1.5 x ULN
  9. Treatment-related toxicities from prior therapies must have resolved to Grade </= 1
  10. At least 2 weeks from prior MF-directed treatment (till the start of study drug)

Exclusion Criteria:

  1. Any concurrent severe and/or uncontrolled medical conditions that could increase the patient's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities.
  2. Impaired cardiac function or clinically significant cardiac diseases, including any of the following: History or presence of ventricular tachyarrhythmia; Presence of unstable atrial fibrillation (ventricular response > 100 bpm). Patients with stable atrial fibrillation are eligible, provided they do not meet any of the other cardiac exclusion criteria. Clinically significant resting bradycardia (< 50 bpm). Angina pectoris or acute myocardial infarction </= 3 months prior to starting study drug. Other clinically significant heart disease (e.g., symptomatic congestive heart failure; uncontrolled arrhythmia or hypertension; history of labile hypertension or poor compliance with an antihypertensive regimen).
  3. Patients who are currently receiving chronic (>14 days) treatment with corticosteroids at a dose >/= 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug
  4. Patients who are currently receiving treatment with agents that are metabolized solely through CYP3A4/5 and have a narrow therapeutic index or are strong CYP2C8 inhibitors; or are receiving treatment with agents that carry a risk for QT prolongation and are CYP3A substrates.
  5. Patients with impairment of GI function or GI disease that may significantly alter the absorption of LCL161 as per physicians opinion
  6. Pregnant or breast feeding (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive beta-HCG laboratory test.
  7. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 90 days after study treatment. Highly effective contraception methods include: Total abstinence or Male partner or female sterilization or Combination of any two of the following (a+b or a+c, or b+c): a. Use of oral, injected or implanted hormonal methods of contraception, b. Placement of an intrauterine device (IUD) or intrauterine system (IUS), c. Barrier methods of contraception: condom for male partner or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository.
  8. Continued from #7 above: Note: Postmenopausal women are allowed to participate in this study. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago. In the case of oophorectomy alone, a woman is considered to be of not child bearing potential only when her reproductive status has been confirmed by follow-up hormone level assessment.
  9. Sexually active males must use a condom during intercourse while taking the drug and for 3 months after stopping study drug and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact: Naveen Pemmaraju, MD 713-792-4956
United States
 
 
NCT02098161
2013-0612
NCI-2014-01241 ( Registry Identifier: NCI CTRP )
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
Novartis
Principal Investigator: Naveen Pemmaraju, MD M.D. Anderson Cancer Center
M.D. Anderson Cancer Center
February 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP