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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00952289




Registration number
NCT00952289
Ethics application status
Date submitted
4/08/2009
Date registered
6/08/2009
Date last updated
12/03/2018

Titles & IDs
Public title
COntrolled MyeloFibrosis Study With ORal JAK Inhibitor Treatment: The COMFORT-I Trial
Scientific title
A Randomized, Double-blind, Placebo-controlled Study of the JAK Inhibitor INCB018424 Tablets Administered Orally to Subjects With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis or Post-Essential Thrombocythemia Myelofibrosis
Secondary ID [1] 0 0
INCB 18424-351
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
MPN (Myeloproliferative Neoplasms) 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Leukaemia - Acute leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ruxolitinib
Treatment: Drugs - Placebo

Experimental: Ruxolitinib - Participants received ruxolitinib orally twice a day. The starting dose was determined based on Baseline platelet count. Patients with Baseline platelet count \> 200,000/µL began a dose regimen of 20 mg twice daily. Patients with Baseline platelet count of 100,000/µL to 200,000/µL (inclusive) began a dose regimen of 15 mg twice daily. The dose was adjusted by the Investigator based on efficacy and safety to a maximum of 25 mg twice daily. Patients receiving benefit could continue treatment until the later of marketing approval or when the last randomized patient remaining in the study had completed Week 144 (36 months).

Placebo comparator: Placebo - Placebo tablets matching ruxolitinib were administered orally twice a day at a starting dose based on Baseline platelet count. Doses were titrated using the same guidelines as for active drug. Patients meeting certain protocol requirements detailed below were given the opportunity to cross over to ruxolitinib treatment.


Treatment: Drugs: Ruxolitinib
Ruxolitinib phosphate tablets 5 mg administered as oral doses.

Treatment: Drugs: Placebo
Matching placebo tablets were administered as oral doses in the same manner as active drug.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Achieving = 35% Reduction in Spleen Volume From Baseline to Week 24
Timepoint [1] 0 0
Baseline and Week 24
Secondary outcome [1] 0 0
Maintenance of a = 35% Reduction From Baseline in Spleen Volume Among Patients Initially Randomized to Receive Ruxolitinib
Timepoint [1] 0 0
Baseline Visit and every 12 weeks until the data cut-off date (up to 14 months).
Secondary outcome [2] 0 0
Duration of Maintenance of a = 35% Reduction From Baseline in Spleen Volume Among Patients Initially Randomized to Receive Ruxolitinib
Timepoint [2] 0 0
Baseline Visit and every 12 weeks until the data cut-off date (up to 14 months).
Secondary outcome [3] 0 0
Number of Participants With a = 50% Reduction in Total Symptom Score From Baseline to Week 24
Timepoint [3] 0 0
Baseline and Week 24. Baseline total score was the average of the daily total scores for the last 7 days prior to randomization. The Week 24 total score was the average of daily total scores from the 28 days prior to the Week 24 visit.
Secondary outcome [4] 0 0
Change From Baseline to Week 24 in Total Symptom Score
Timepoint [4] 0 0
Baseline and Week 24. Baseline total score was the average of the daily total scores for the last 7 days prior to randomization. The Week 24 total score was the average of daily total scores from the 28 days prior to the Week 24 visit.
Secondary outcome [5] 0 0
Overall Survival
Timepoint [5] 0 0
From randomization to the data cut-off date (up to 14 months).
Secondary outcome [6] 0 0
Overall Survival Time
Timepoint [6] 0 0
From randomization to the data cut-off date (up to 14 months).
Secondary outcome [7] 0 0
Overall Survival - Extended Data
Timepoint [7] 0 0
From randomization to 4 months after the data cut-off date (up to 18 months).
Secondary outcome [8] 0 0
Overall Survival Time - Extended Data
Timepoint [8] 0 0
From randomization to 4 months after the data cut-off date (up to 18 months).
Secondary outcome [9] 0 0
Overall Survival at Week 144
Timepoint [9] 0 0
Week 144
Secondary outcome [10] 0 0
Overall Survival Time at Week 144
Timepoint [10] 0 0
Week 144

Eligibility
Key inclusion criteria
* Subjects must be diagnosed with primary myelofibrosis (PMF), post-polycythemia vera-myelofibrosis (PPV-MF) or post-essential thrombocythemia-myelofibrosis (PET-MF) according to the 2008 World Health Organization criteria
* Subjects with myelofibrosis requiring therapy must be classified as high risk OR intermediate risk level 2 according to the prognostic factors defined by the International Working Group
* Subjects with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, 2 or 3
* Subjects who have not previously received treatment with a Janus kinase (JAK) inhibitor
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Subjects with a life expectancy of less than 6 months
* Subjects with inadequate bone marrow reserve as demonstrated by specific clinical laboratory counts
* Subjects with inadequate liver or renal function
* Subjects with clinically significant bacterial, fungal, parasitic or viral infection which require therapy
* Subjects with an active malignancy over the previous 5 years except specific skin cancers.
* Subjects with severe cardiac conditions
* Subjects who have had splenic irradiation within 12 months

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
- Darlinghurst
Recruitment hospital [2] 0 0
- Kogarah
Recruitment hospital [3] 0 0
- Randwick
Recruitment hospital [4] 0 0
- St Leonards
Recruitment hospital [5] 0 0
- Brisbane
Recruitment hospital [6] 0 0
- Douglas
Recruitment hospital [7] 0 0
- Herston
Recruitment hospital [8] 0 0
- Milton
Recruitment hospital [9] 0 0
- Woolloongabba
Recruitment hospital [10] 0 0
- Bedford Park
Recruitment hospital [11] 0 0
- Box Hill
Recruitment hospital [12] 0 0
- Clayton
Recruitment hospital [13] 0 0
- Frankston
Recruitment hospital [14] 0 0
- Ringwood East
Recruitment hospital [15] 0 0
- Fremantle
Recruitment hospital [16] 0 0
- Perth
Recruitment postcode(s) [1] 0 0
- Darlinghurst
Recruitment postcode(s) [2] 0 0
- Kogarah
Recruitment postcode(s) [3] 0 0
- Randwick
Recruitment postcode(s) [4] 0 0
- St Leonards
Recruitment postcode(s) [5] 0 0
- Brisbane
Recruitment postcode(s) [6] 0 0
- Douglas
Recruitment postcode(s) [7] 0 0
- Herston
Recruitment postcode(s) [8] 0 0
- Milton
Recruitment postcode(s) [9] 0 0
- Woolloongabba
Recruitment postcode(s) [10] 0 0
- Bedford Park
Recruitment postcode(s) [11] 0 0
- Box Hill
Recruitment postcode(s) [12] 0 0
- Clayton
Recruitment postcode(s) [13] 0 0
- Frankston
Recruitment postcode(s) [14] 0 0
- Ringwood East
Recruitment postcode(s) [15] 0 0
- Fremantle
Recruitment postcode(s) [16] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Colorado
Country [5] 0 0
United States of America
State/province [5] 0 0
Connecticut
Country [6] 0 0
United States of America
State/province [6] 0 0
District of Columbia
Country [7] 0 0
United States of America
State/province [7] 0 0
Florida
Country [8] 0 0
United States of America
State/province [8] 0 0
Georgia
Country [9] 0 0
United States of America
State/province [9] 0 0
Hawaii
Country [10] 0 0
United States of America
State/province [10] 0 0
Idaho
Country [11] 0 0
United States of America
State/province [11] 0 0
Illinois
Country [12] 0 0
United States of America
State/province [12] 0 0
Indiana
Country [13] 0 0
United States of America
State/province [13] 0 0
Iowa
Country [14] 0 0
United States of America
State/province [14] 0 0
Kentucky
Country [15] 0 0
United States of America
State/province [15] 0 0
Louisiana
Country [16] 0 0
United States of America
State/province [16] 0 0
Maryland
Country [17] 0 0
United States of America
State/province [17] 0 0
Michigan
Country [18] 0 0
United States of America
State/province [18] 0 0
Minnesota
Country [19] 0 0
United States of America
State/province [19] 0 0
Mississippi
Country [20] 0 0
United States of America
State/province [20] 0 0
Missouri
Country [21] 0 0
United States of America
State/province [21] 0 0
Montana
Country [22] 0 0
United States of America
State/province [22] 0 0
New Jersey
Country [23] 0 0
United States of America
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New Mexico
Country [24] 0 0
United States of America
State/province [24] 0 0
New York
Country [25] 0 0
United States of America
State/province [25] 0 0
North Carolina
Country [26] 0 0
United States of America
State/province [26] 0 0
North Dakota
Country [27] 0 0
United States of America
State/province [27] 0 0
Ohio
Country [28] 0 0
United States of America
State/province [28] 0 0
Oregon
Country [29] 0 0
United States of America
State/province [29] 0 0
Pennsylvania
Country [30] 0 0
United States of America
State/province [30] 0 0
South Carolina
Country [31] 0 0
United States of America
State/province [31] 0 0
Tennessee
Country [32] 0 0
United States of America
State/province [32] 0 0
Texas
Country [33] 0 0
United States of America
State/province [33] 0 0
Utah
Country [34] 0 0
United States of America
State/province [34] 0 0
Vermont
Country [35] 0 0
United States of America
State/province [35] 0 0
Washington
Country [36] 0 0
United States of America
State/province [36] 0 0
Wisconsin
Country [37] 0 0
Canada
State/province [37] 0 0
British Columbia
Country [38] 0 0
Canada
State/province [38] 0 0
Newfoundland and Labrador
Country [39] 0 0
Canada
State/province [39] 0 0
Nova Scotia
Country [40] 0 0
Canada
State/province [40] 0 0
Ontario
Country [41] 0 0
Canada
State/province [41] 0 0
Quebec

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Incyte Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Srdan Verstovsek, MD, PhD
Address 0 0
M.D. Anderson Cancer Center
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents