NCT03123588

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2017

Geographic Reach
1 country

26 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 17, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 21, 2017

Completed
7 months until next milestone

Study Start

First participant enrolled

November 14, 2017

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 3, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 3, 2020

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

November 19, 2021

Completed
Last Updated

November 19, 2021

Status Verified

October 1, 2021

Enrollment Period

2.7 years

First QC Date

April 17, 2017

Results QC Date

August 2, 2021

Last Update Submit

October 21, 2021

Conditions

Keywords

Essential thrombocythemiahydroxyurea-resistanthydroxyurea-intolerantruxolitinibanagrelide

Outcome Measures

Primary Outcomes (1)

  • Proportion of Subjects Who Achieve Platelet and White Blood Cell (WBC) Control

    Defined as proportion of subjects who achieve a simultaneous reduction of platelet counts to \< 600 × 10\^9/L with a reduction of WBC counts to \< 10 × 10\^9/L for at least 80% of biweekly measurements for a consecutive 12-week period between Weeks 32 and 52.

    52 weeks

Secondary Outcomes (6)

  • Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    Baseline through the end of randomized period -up to 14 months per participant

  • Proportion of Subjects Who Achieve Complete Remission or Partial Remission

    32 weeks

  • Time to Treatment Discontinuation

    98 weeks

  • Duration of Response

    142 weeks

  • Proportion of Subjects Who Achieve Reduction of Platelet Counts to < 600 × 10^9/L

    Between 32 and 52 weeks

  • +1 more secondary outcomes

Study Arms (2)

Group A : Ruxolitinib and anagrelide placebo

EXPERIMENTAL

Ruxolitinib or placebo will be administered orally twice a day at a starting dose of 10 mg.

Drug: RuxolitinibDrug: Placebo

Group B : Anagrelide and Ruxolitinib PLacebo

ACTIVE COMPARATOR

Anagrelide or placebo will be administered orally twice a day at a starting dose of 1 mg. Use of anagrelide will be consistent with approved prescribing information.

Drug: AnagrelideDrug: Placebo

Interventions

Ruxolitinib administered orally twice daily (BID) at the protocol-defined starting dose.

Also known as: Jakafi, INCB018424
Group A : Ruxolitinib and anagrelide placebo

Anagrelide administered orally at a starting dose of 1 mg BID.

Group B : Anagrelide and Ruxolitinib PLacebo

Anagrelide-placebo administered orally BID

Group A : Ruxolitinib and anagrelide placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of essential thrombocythemia according to revised World Health Organization (WHO) 2016 criteria.
  • Resistant to or intolerant of hydroxyurea, that is, fulfilling at least 1 of the following criteria:
  • Platelet count \> 600 × 10\^9/L after 3 months of at least 2 g/day of hydroxyurea (2.5 g/day in subjects with a body weight over 80 kg) OR at the subject's maximally tolerated dose if that dose is \< 2 g/day.
  • Platelet count \> 400 × 10\^9/L and WBC count \< 2.5 × 10\^9/L or hemoglobin \< 10 g/dL at any dose of hydroxyurea.
  • Presence of leg ulcers or other unacceptable mucocutaneous manifestations at any dose of hydroxyurea.
  • Hydroxyurea-related fever.
  • Platelet count ≥ 650 × 10\^9/L at screening.
  • WBC ≥ 11.0 × 10\^9/L at screening.

You may not qualify if:

  • Subjects previously treated with anagrelide or Hydroxyurea (HU).
  • Prior anagrelide use is allowed provided the reason for discontinuation is not AE-related and anagrelide is stopped at least 28 days before the start of study medications (ie, Day 1).
  • Inadequate liver function at screening and Day 1 (before drug administration) as demonstrated by:
  • Total bilirubin \> 1.5 × upper limit of normal (ULN)
  • Aspartate aminotransferase or alanine aminotransferase \> 1.5 × ULN
  • Hepatocellular disease (eg, cirrhosis)
  • Inadequate renal function at screening as demonstrated by creatinine clearance \< 40 mL/min calculated by Cockcroft-Gault equation.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (26)

Mayo Clinic

Phoenix, Arizona, 85054, United States

Location

Pacific Shores Medical Group

Long Beach, California, 90813, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

Ventura County Hematology-Oncology Specialists

Oxnard, California, 93030, United States

Location

Compassionate Cancer Care Medical Group

Riverside, California, 92501, United States

Location

Innovative Clinical Research Institute

Whittier, California, 90603, United States

Location

Bond Clinic, PA

Winter Haven, Florida, 33880, United States

Location

Tift Regional

Tifton, Georgia, 31794, United States

Location

Edward Cancer Center

Naperville, Illinois, 60540, United States

Location

North Shore Cancer Research Association-Skokie

Skokie, Illinois, 60076, United States

Location

Southern Illinois University

Springfield, Illinois, 62702, United States

Location

Clinical Trials of SWLA LLC

Lake Charles, Louisiana, 70601, United States

Location

St. Agnes Hospital

Baltimore, Maryland, 21229, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Summit Medical Group

Morristown, New Jersey, 07960, United States

Location

Columbia Weill Cornell Cancer Centers - Herbert Irving Comprehensive Cancer Center (HICCC)

New York, New York, 10032, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Waverly Hem Onc

Cary, North Carolina, 27518, United States

Location

Vidant Medical Center

Greenville, North Carolina, 27858, United States

Location

Gabrail Cancer Center- Canton Facility

Canton, Ohio, 44718, United States

Location

INTEGRIS Southwest Medical Center

Oklahoma City, Oklahoma, 73109, United States

Location

INTEGRIS Cancer Institute Proton Campus

Oklahoma City, Oklahoma, 73142, United States

Location

Kaiser Permanente Northwest

Portland, Oregon, 97227, United States

Location

Geisinger - Knapper Clinic

Danville, Pennsylvania, 17822, United States

Location

Prairie Lakes Health Care System Inc.

Watertown, South Dakota, 57201, United States

Location

Renovatio Clinical

The Woodlands, Texas, 77401, United States

Location

MeSH Terms

Conditions

Myeloproliferative DisordersThrombocythemia, Essential

Interventions

ruxolitinibanagrelide

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesBlood Coagulation DisordersThrombocytosisBlood Platelet DisordersHemorrhagic Disorders

Results Point of Contact

Title
Call Center
Organization
Incyte Corporation

Study Officials

  • Albert Assad, MD

    Incyte Corporation

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

April 17, 2017

First Posted

April 21, 2017

Study Start

November 14, 2017

Primary Completion

August 3, 2020

Study Completion

August 3, 2020

Last Updated

November 19, 2021

Results First Posted

November 19, 2021

Record last verified: 2021-10

Data Sharing

IPD Sharing
Will not share

Locations