Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01178281




Registration number
NCT01178281
Ethics application status
Date submitted
15/07/2010
Date registered
10/08/2010
Date last updated
17/07/2019

Titles & IDs
Public title
Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
Scientific title
A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence
Secondary ID [1] 0 0
2010-018965-42
Secondary ID [2] 0 0
CC-4047-MF-002
Universal Trial Number (UTN)
Trial acronym
RESUME
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Myelofibrosis 0 0
MPN-associated Myelofibrosis 0 0
Condition category
Condition code
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 - Pomalidomide 0.5 mg
Treatment: Drugs - Placebo
Treatment: Drugs - Pomalidomide

Experimental: Pomalidomide 0.5 mg - Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.

Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of = 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

Placebo comparator: Placebo - Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression.

Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC- transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied.

Experimental: China Extension: Pomalidomide 0.5 mg - Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.

Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.


Treatment: Drugs: Pomalidomide 0.5 mg
Pomalidomide 0.5 mg capsule taken by mouth once daily. Immunomodulatory agent with demonstrated efficacy in the treatment of subjects with RBC-transfusion-dependence associated with MNP-associated myelofibrosis.

Treatment: Drugs: Placebo
Placebo Comparator to active drug; Placebo capsule taken by mouth once daily

Treatment: Drugs: Pomalidomide
Pomalidomide 0.5 mg capsule taken by mouth once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Who Achieved RBC-Transfusion Independence
Timepoint [1] 0 0
168 days
Primary outcome [2] 0 0
China Extension: Number of Participants Achieving a Hemoglobin Increase of = 15 g/L Compared to Baseline for = 84 Consecutive Days
Timepoint [2] 0 0
From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.
Secondary outcome [1] 0 0
Overall Survival
Timepoint [1] 0 0
From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm.
Secondary outcome [2] 0 0
Duration of RBC-Transfusion Independence
Timepoint [2] 0 0
From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.
Secondary outcome [3] 0 0
Time to RBC-Transfusion Independence
Timepoint [3] 0 0
168 days
Secondary outcome [4] 0 0
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Timepoint [4] 0 0
From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm.
Secondary outcome [5] 0 0
Healthcare Resource Utilization
Timepoint [5] 0 0
From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm.
Secondary outcome [6] 0 0
Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score
Timepoint [6] 0 0
Baseline and Days 85 and 169
Secondary outcome [7] 0 0
Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale
Timepoint [7] 0 0
Baseline and Days 85 and 169
Secondary outcome [8] 0 0
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score
Timepoint [8] 0 0
Baseline and Days 85 and 169

Eligibility
Key inclusion criteria
* Age = 18 years
* Myeloproliferative-neoplasm (MPN)-associated myelofibrosis
* RBC-transfusion-dependence (global study):

* Average RBC-transfusion frequency = 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval > 42 days without = 1 RBC-transfusion.
* Only RBC-transfusions given when the hemoglobin = 90 g/L³ are scored in

determining eligibility.

* RBC-transfusions due to bleeding are not scored in determining eligibility.
* RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.

* Severe anemia (China-specific extension):
* = 2 hemoglobin concentrations = 80 g/L for = 84 days immediately before the day of enrollment.
* No RBC-transfusion within 6 months prior to enrollment.

* Hemoglobin = 130 g/L at randomization (global study); = 80 g/L at enrollment in the China-specific extension.
* Bone marrow biopsy within 6 months (global study only).
* Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids
* Eastern Cooperative Oncology Group (ECOG) performance status = 2.
* Agree to follow pregnancy precautions as required by the protocol.
* Agree to receive counseling related to teratogenic and other risks of pomalidomide.
* Agree not to donate blood or semen.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Prior blood cell or bone marrow allotransplant.
* Use of drugs to treat MPN-associated myelofibrosis = 30 days before starting study drug.
* Treatment with erythropoietin or androgenic steroids = 84 days before starting study drug.
* Anemia due to reasons other than MPN-associated myelofibrosis.
* Pregnant or lactating females.
* More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks
* Prior history of malignancies,other than the disease being studied, unless the subject has been free of the malignancy for = 5 years with the following exceptions:

* Carcinoma in situ of the cervix
* Carcinoma in situ of the breast
* Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)
* Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.
* Prior treatment with pomalidomide.
* Allergic reaction or rash after treatment with thalidomide or lenalidomide
* Any of the following laboratory abnormalities:

* Neutrophils < 0.5x10^9 /L
* Platelets < 25 x 10^9 /L
* Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²
* Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)
* Total bilirubin = 4 x ULN;
* Uncontrolled hyperthyroidism or hypothyroidism.
* Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug
* Clinically-important heart disease within the past 6 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
8/09/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/05/2018
Sample size
Target
Accrual to date
Final
267
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Gosford Hospital - Gosford
Recruitment hospital [2] 0 0
Royal North Shore Hospital - St. Leonards
Recruitment hospital [3] 0 0
Frankston Hospital - Frankston
Recruitment hospital [4] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2250 - Gosford
Recruitment postcode(s) [2] 0 0
2065 - St. Leonards
Recruitment postcode(s) [3] 0 0
3199 - Frankston
Recruitment postcode(s) [4] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
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New York
Country [8] 0 0
United States of America
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Ohio
Country [9] 0 0
United States of America
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Pennsylvania
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United States of America
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South Dakota
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Texas
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United States of America
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Utah
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United States of America
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Washington
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Austria
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Graz
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Austria
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Innsbruck
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Austria
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Vienna
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Belgium
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Brugge
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Belgium
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Charleroi
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Belgium
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Leuven
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Canada
State/province [20] 0 0
Alberta
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Montreal,
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China
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Beijing
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China
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Jiangsu
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China
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Shanghai
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China
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Sichuan
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China
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Tianjin
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France
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La Tronche
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France
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Lille
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France
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Limoges
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France
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Paris
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Pessac
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Strasbourg
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France
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Toulouse
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Villejuif
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Germany
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Aachen
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Germany
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Hannover
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Germany
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Leipzig
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Germany
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Minden
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Germany
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Ulm
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Italy
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Bari
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Italy
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Bergamo
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Italy
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Firenze
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Italy
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Napoli
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Italy
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Orbassano
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Italy
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Pavia
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Italy
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Varese
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Japan
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Bunkyou-ku
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Japan
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Fukuoka City
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Japan
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Isehara City
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Japan
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Kyoto City
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Japan
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Nagasaki City
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Japan
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Shinjuku
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Netherlands
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Amsterdam
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Netherlands
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Rotterdam
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Netherlands
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Utrecht
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Poland
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Rzeszow
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Poland
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Szczecin
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Poland
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Warsaw
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Russian Federation
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Moscow
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Russian Federation
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Saint-Petersburg
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Spain
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Barcelona
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Spain
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Majadahonda
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Spain
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Salamanca
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Spain
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Valencia
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Sweden
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Lund
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Sweden
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Stockholm
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United Kingdom
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Belfast
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United Kingdom
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Glasgow
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Headington
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London
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United Kingdom
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Newcastle upon Tyne
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United Kingdom
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Sheffield

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Robert Peter P Gale, MD, Ph.D.
Address 0 0
Celgene Corporation
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

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01178281