CClinicalTrials.gg
CompletedNCT03041636Updated Apr 21, 2021Results posted

Ruxolitinib Phosphate in Treating Patients With Previously Untreated Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

A Phase 2 interventional study of Ruxolitinib and Ruxolitinib Phosphate in Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma and Untreated Chronic Lymphocytic Leukemia, sponsored by M.D. Anderson Cancer Center. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-04-21.

Sponsored by M.D. Anderson Cancer Center · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
1
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

This phase II trial studies how well ruxolitinib phosphate works in treating patients with previously untreated chronic lymphocytic leukemia/small lymphocytic lymphoma. Ruxolitinib phosphate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVES:

I. To determine the effect of ruxolitinib phosphate (ruxolitinib) in patients with high-risk chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) who do not require anti-neoplastic therapy according to the International Workshop on Chronic Lymphocytic Leukemia (IWCLL) 2008 recommendations and were either previously untreated or treated with Ibrutinib for less than 3 months and were deemed Ibrutinib intolerant:

Ia. On disease burden. Ib. The rate of complete response (CR) and partial response (PR) as assessed by the IWCLL 2008 response criteria.

SECONDARY OBJECTIVES:

I. To evaluate the time to next treatment of high-risk CLL/SLL who do not require anti-neoplastic therapy according to the IWCLL 2008 recommendations.

OUTLINE:

Patients receive ruxolitinib phosphate orally (PO) twice daily (BID). Treatment continues for up to 3 years in the absence of disease progression or unacceptable toxicity. Treatment beyond 3 years may be permitted after discussion with the principal investigator.

After completion of study treatment, patients are followed up at 30 days.

02

Conditions studied

  • Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
  • Untreated Chronic Lymphocytic Leukemia
03

In context

Lymphoma

5,578 studies on the registry are indexed under Lymphoma; 825 are open to participants now.

This study's enrollment of 1 is below the median of 40 across 4,508 interventional studies indexed under Lymphoma.

Browse Lymphoma studies →

Lead sponsor

M.D. Anderson Cancer Center is the lead sponsor of 2,999 studies on the registry; 581 are open to participants now.

Of its 599 completed or terminated interventional studies of FDA-regulated products, 402 (67%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Subjects who are able to understand and sign an informed consent document.
  2. Subjects 18 years of age or older.
  3. Subjects must be diagnosed with CLL/SLL and do not meet the IWCLL criteria for treatment
  4. Patients should be previously untreated or have only been treated with single agent ibrutinib therapy for a period of \< 3 months and were deemed ibrutinib intolerant.
  5. Patients whose expected time to CLL/SLL treatment, according to our nomogram posted on the leukemia protocol priority list, is four years of less.
  6. Subjects with hemoglobin values at the screening visit equal to or greater than 12.0 g/dL.
  7. Subjects with a platelet count of at least 100 x10\^9 at the screening visit.
  8. Subjects with an absolute neutrophil count (ANC) of equal to or higher than 0.5 x10\^9 at the screening visit.
  9. Subject who are willing to undergo a bone marrow aspiration and biopsy and CT scan for disease burden assessment.
  10. Patient who are capable to return to MD Anderson Cancer Center (MDACC) for follow-up
  11. Subjects with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
  12. Patient must be capable of swallowing the Ruxolotinib capsules (tablets).

Exclusion criteria

Exclusion:

  1. Females who are pregnant or are currently breastfeeding.
  2. Subjects of childbearing potential who are unwilling to take appropriate precautions (throughout the study from screening including 30 days after discontinuation of the study drug) to avoid becoming pregnant or fathering a child. A) Females of non-childbearing potential are defined as women who (a) are equal to or greater than 55 years of age with history of amenorrhea for 1 year, OR (b) are surgically sterile for at least 3 months. B) For females of childbearing potential, or for males, appropriate precautions are those that are at least 99% effective in preventing the occurrence of pregnancy. These methods should be communicated to the subjects and their understanding confirmed: a) Double barrier methods; b) Condom with spermicide in conjunction with use of an intrauterine device (IUD); c) Condom with spermicide in conjunction with use of a diaphragm; d) Oral, injectable, or implanted contraceptives; e) Tubal ligation or vasectomy (surgical sterilization)
  3. Subjects with recent history of inadequate bone marrow reserve as demonstrated by previous transfusions except for acute blood loss (e.g. surgery) in the month prior to screening.
  4. Subjects with inadequate liver or renal function at screening and baseline visits: A) Alanine aminotransferase (ALT) > 2.5x Upper limit of normal (ULN). B) Modification of Diet in Renal Disease (MDRD) calculated GFR \< 30 mL/min
  5. Subjects with active uncontrolled infection or who are HIV positive (Subjects with acute infections requiring treatment should delay screening/enrollment until the course of therapy has been completed and the event is considered controlled).
  6. Subjects with a history of or a current malignancy except for treated basal or squamous carcinomas of the skin completely resected.
  7. Subjects with clinically significant uncontrolled cardiac disease.
  8. Subjects being treated concurrently with any prohibited medications, including investigational medication, rifampin, St. John's wort, and potent CYP3A4 inhibitors (excluding ketoconazole) unless continuation of such medications are determined by the investigator to be in the best interest of the patient. Refer to protocol section 2.2.12 for more details.
  9. Subjects who have previously received JAK inhibitor therapy
  10. Subjects with active alcohol or drug addiction that would interfere with their ability to comply with the study requirements.
  11. Subjects with any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol.
  12. Subjects who have unknown transfusion history.
  13. Patients who cannot comply with the study requirements.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
1 participant (actual)

Study arms

  • Experimental
    Treatment (ruxolitinib phosphate)

    Patients receive ruxolitinib phosphate PO BID. Treatment continues for up to 3 years in the absence of disease progression or unacceptable toxicity. Treatment beyond 3 years may be permitted after discussion with the principal investigator.

    Drug: Ruxolitinib · Drug: Ruxolitinib Phosphate

Interventions

  • DrugRuxolitinib

    Given PO

    Also known as: INCB-18424, INCB18424, Oral JAK Inhibitor INCB18424

  • DrugRuxolitinib Phosphate

    Given PO

    Also known as: INCB-18424 Phosphate, Jakafi

06

What researchers measure

Primary outcomes

  1. Participants With a Clinical Response

    Clinical response will be assessed based on physical examination, complete blood count (CBC), a bone marrow aspiration, a whole body CT scan to be done at screening and 6 + 2 months in accordance with the International Workshop on Chronic Lymphocytic Leukemia (IWCLL) guidelines (Hallek et al., 2008)

    Time frame: Up to 6 months after initiation of therapy

  2. Number of Participants With Change of Tumor Burden

    Tumor burden will be assessed by bone marrow aspiration, whole body CT scan at screening and 6 months.

    Time frame: 6 months after initiation of therapy

  3. Participants With a Response

    Response Rate is Complete Response (CR) or Partial Response (PR). CR is absence of Lymphadenopathy, Hepatomegaly or Splenomegaly, lymphocytes \< 4000/ul, normocellular, \<30% lymphocytes, no B-lymphoid nodules, Platelets \> 100,000/ul, hemoglobin \>11.0 g/dl and Neutrophils \>1500/ul. PR is \>/= 50% decrease in lymphadenopathy, hepatomegaly, splenomegaly and Blood Lymphocytes from baseline, 50% reduction in marrow infiltrate or B-lymphoid nodules. Platelet count \> 100,000/ul, Hemoglobin \> 11 g/dl and Neutrophils \>1500/ul or increase \>/= 50% of all over base.

    Time frame: Up to 30 days

  4. Time to Next Treatment

    Number of months to subsequent therapy per patient.

    Time frame: Up to 30 days

07

Results

Posted Apr 21, 2021

Participant flow

Recruitment Period: March 2017 to June 2019

Participant flow — Overall Study
MilestoneTreatment (Ruxolitinib Phosphate)
Started1
Completed1
Not completed0

Outcome measures

PrimaryParticipants With a Clinical Response

Clinical response will be assessed based on physical examination, complete blood count (CBC), a bone marrow aspiration, a whole body CT scan to be done at screening and 6 + 2 months in accordance with the International Workshop on Chronic Lymphocytic Leukemia (IWCLL) guidelines (Hallek et al., 2008)

Time frame:
Up to 6 months after initiation of therapy
Reported as:
Count of participants · Participants
Participants With a Clinical Response
ParticipantsTreatment (Ruxolitinib Phosphate)
Participants With a Clinical Response0
PrimaryNumber of Participants With Change of Tumor Burden

Tumor burden will be assessed by bone marrow aspiration, whole body CT scan at screening and 6 months.

Time frame:
6 months after initiation of therapy
Reported as:
Count of participants · Participants
Number of Participants With Change of Tumor Burden
ParticipantsTreatment (Ruxolitinib Phosphate)
Number of Participants With Change of Tumor Burden0
PrimaryParticipants With a Response

Response Rate is Complete Response (CR) or Partial Response (PR). CR is absence of Lymphadenopathy, Hepatomegaly or Splenomegaly, lymphocytes \< 4000/ul, normocellular, \<30% lymphocytes, no B-lymphoid nodules, Platelets \> 100,000/ul, hemoglobin \>11.0 g/dl and Neutrophils \>1500/ul. PR is \>/= 50% decrease in lymphadenopathy, hepatomegaly, splenomegaly and Blood Lymphocytes from baseline, 50% reduction in marrow infiltrate or B-lymphoid nodules. Platelet count \> 100,000/ul, Hemoglobin \> 11 g/dl and Neutrophils \>1500/ul or increase \>/= 50% of all over base.

Time frame:
Up to 30 days
Reported as:
Count of participants · Participants
Participants With a Response
ParticipantsTreatment (Ruxolitinib Phosphate)
Participants With a Response0
PrimaryTime to Next Treatment

Number of months to subsequent therapy per patient.

Time frame:
Up to 30 days
Reported as:
Number · Months
Time to Next Treatment
MonthsTreatment (Ruxolitinib Phosphate)
Time to Next Treatment0

Adverse events

Collected over Up to 3 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Ruxolitinib Phosphate)0/1 (0%)0/1 (0%)1/1 (100%)
Most frequent other events
Most frequent other events
EventTreatment (Ruxolitinib Phosphate)
Skin InfectionInfections and infestations1/1
Upper Respiratory InfectionInfections and infestations1/1

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment (Ruxolitinib Phosphate)
<=18 years0
Between 18 and 65 years1
>=65 years0
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Ruxolitinib Phosphate)
Female0
Male1
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment (Ruxolitinib Phosphate)
Hispanic or Latino0
Not Hispanic or Latino1
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (Ruxolitinib Phosphate)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White0
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(Participants)Treatment (Ruxolitinib Phosphate)
United States1
08

Study locations

1 site
  • M D Anderson Cancer Center
    Houston, Texas 77030, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Mar 2, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Apr 21, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03041636
Lead sponsor
M.D. Anderson Cancer Center
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Feb 3, 2017
Start date
Mar 8, 2017
Primary completion
Apr 29, 2020
Completion
Apr 29, 2020
Results posted
Apr 21, 2021
Last update
Apr 21, 2021

Study contacts

Zeev Estrov
principal investigator · M.D. Anderson Cancer Center

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Mar 2021. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion