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Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation
ClinicalTrials.gov Identifier:
NCT02252159
First received: September 23, 2014
Last updated: February 28, 2017
Last verified: February 2017
  Purpose
This is a Phase IV, multicenter, non-interventional, non-randomized, prospective, observational study in an adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV and are being followed in either community or academic medical centers in the United States who will be enrolled over a 12-month period and observed for 36 months from the date the last patient is enrolled.

Condition
Polycythemia Vera

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective, Non-interventional Study of Disease Progression and Treatment of Patients With Polycythemia Vera in United States Academic or Community Clinical Practices

Resource links provided by NLM:


Further study details as provided by Incyte Corporation:

Primary Outcome Measures:
  • Description of patterns of the disease Polycythemia Vera (PV), and associations of such patterns with patients' exposures or treatments [ Time Frame: Every 3 months for approximately 36 months ]
  • Incidence (frequency) of disease progression [ Time Frame: Every 3 months for approximately 36 months ]
  • Incidence (frequency) of healthcare resources utilization [ Time Frame: Every 3 months for approximately 36 months ]
    Healthcare resources were defined as a. Medical visits (including Office visits, ER visits, and hospitalizations), b. phlebotomy (PHL) procedures and c. prescriptions (including PV-related prescriptions, PV-related over-the-counter (OTC) medications, and prescription medications for co-morbid conditions).

  • Incidence (frequency) of complications due to PV [ Time Frame: Every 3 months for approximately 36 months ]
  • Incidence (frequency) and description of PV-related symptoms [ Time Frame: Every 3 months for approximately 36 months ]

Secondary Outcome Measures:
  • Incidence (frequency) of adverse events (for those patients on active therapies, including PHL) [ Time Frame: Baseline through end of study. Approximately 36 months ]
  • Patient-reported outcomes as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) for assessment of the severity of symptoms [ Time Frame: Every 3 months for approximately 36 months ]
    The MPN-SAF TSS is a validated 10-item instrument that efficiently assesses the prevalence and severity of PV symptoms in both clinical practice and trial settings. Higher scores indicate more severe symptoms and greater inactivity with a possible total score range of 0-100.

  • Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP) score for assessment of work productivity and activity impairment [ Time Frame: Every 3 months for approximately 36 months ]
    WPAI-SHP is a validated 6-item instrument that measures the effect of overall health and specific symptoms on productivity at work and outside of it. All outcomes are presented as a proportion, with 0% representing minimal/no impairment and 100% representing maximal impairment or productivity loss.

  • European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) for Health Related Quality of Life (HRQoL) [ Time Frame: Every 3 months for approximately 36 months ]
  • Caregiver burden [ Time Frame: Every 3 months for approximately 36 months ]
    The amount of health care received by a paid healthcare worker or by an unpaid caregiver

  • Burden of phlebotomy (PHL) [ Time Frame: Every 3 months for approximately 36 months ]
    A patients self-reported assessment of burden including pain at site, difficulty and inconvenience of scheduling and cost factors.


Biospecimen Retention:   Samples With DNA
Blood sample(s), two vials, one with heparin, another with ethylenediaminetetraacetic acid (EDTA) will be collected at enrollment and then annually thereafter until the end of study for serum analysis of proteins and assessment of allele burden and mutations.

Enrollment: 2544
Study Start Date: July 2014
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Groups/Cohorts
Cohort A

Patients with clinically overt PV (and not exhibiting any of the characteristics listed for Cohort B), managed with:

  1. Watchful waiting (with or without aspirin)*, or
  2. Phlebotomy (PHL) alone (with or without aspirin)* - or
  3. HU alone (without concomitant PHL, with or without aspirin).

(*Unless patient has a history of intolerance or clinical resistance/ refractoriness to hydroxyurea [HU] (as assessed by the treating physician) - in which case, s/he belongs to Cohort B)

Cohort B

Patients with clinically overt PV, with one or more of the following disease characteristics:

  1. Treatment with HU and PHL in combination or
  2. Treatment with any agent other than HU or aspirin (e.g., recombinant interferon (IFN) or pegylated IFN preparations, busulfan, anagrelide) or
  3. A history of thrombosis (venous or arterial) or
  4. A history of intolerance or clinical resistance/ refractoriness to HU (as assessed by the treating physician) or
  5. Presence of documented splenomegaly (clinically assessed by palpation) or
  6. Presence of one or more of the following uncontrolled symptoms related to PV despite therapy (Symptoms deemed uncontrolled as per physician's judgment)

    1. Tiredness
    2. Difficulty sleeping
    3. Itching
    4. Muscle aches and/or bone pain
    5. Night sweats
    6. Sweats while awake
    7. Other

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
An adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV will be enrolled. Sites will include both community and academic centers across the United States that are representative of where patients with PV are diagnosed and treated.
Criteria

Inclusion Criteria:

  • Age ≥18 years
  • Diagnosis of Polycythemia Vera (PV)
  • Willing and able to provide written informed consent
  • Willing and able to complete patient assessment questionnaires either alone or with minimal assistance from a caregiver and/or trained site personnel
  • Under the supervision of a physician for the current care of PV including but not limited to watchful waiting, acetylsalicylic acid (ASA) 81mg or greater, antithrombotic therapy, Phlebotomy (PHL), Hydroxyurea (HU), interferon (recombinant or pegylated), busulfan, anagrelide

Exclusion Criteria:

  • Participation in an active clinical trial in which the study treatment is blinded
  • Life expectancy <6 months
  • Diagnosis of myelofibrosis (MF) [including primary MF, post-PV MF, or post-essential thrombocythemia MF (post-ET MF)]
  • Diagnosis of secondary Acute Myeloid Leukemia (AML)
  • Diagnosis of Myelodysplastic Syndrome (MDS)
  • History of or active plan to proceed to allogeneic hematopoietic stem cell transplant in next 3 months
  • Splenectomy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02252159

  Show 266 Study Locations
Sponsors and Collaborators
Incyte Corporation
Investigators
Study Director: Ahmad Naim, MD Incyte Corporation
  More Information

Additional Information:
Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT02252159     History of Changes
Other Study ID Numbers: INCB-MA-PV-401
Study First Received: September 23, 2014
Last Updated: February 28, 2017

Keywords provided by Incyte Corporation:
Polycythemia
Polycythemia Vera
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Hydroxyurea
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors

Additional relevant MeSH terms:
Polycythemia
Polycythemia Vera
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Hydroxyurea
Nucleic Acid Synthesis Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors

ClinicalTrials.gov processed this record on March 23, 2017