Patient-Reported Outcome Questionnaire for Use Among Patients with Myelofibrosis After First Line Treatment Failure

Overview

About this study

The primary goal of this study is to inform and substantiate a measurement strategy and its regulatory documentation pursuant to United States (US) product labeling goals.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Able to understand all pertinent aspects of the study and has signed and dated a written informed consent form
  • At least 18 years of age
  • Able to speak, read, write, and comprehend US English fluently
  • Has been diagnosed with Primary Myelofibrosis (PMF) or secondary Myelofibrosis (postessential thrombocythemia myelofibrosis [PET‐MF] and post‐polycythemia vera myelofibrosis [PPV‐MF]) as per World Health Organization (WHO) 2008 criteria
  • Meets one of the following treatment criteria:
    • Failed prior treatment with a JAKi by investigator judgment (JAKi‐treated patients)
    • No prior JAKi treatment, and Intermediate‐1, Intermediate‐2 or High‐risk myelofibrosis according to the Dynamic International Prognostic Scoring System (DIPSS) (JAKi naïve patients)
  • Has a palpable spleen
  • Reports current symptoms that are common to MF (e.g., abdominal discomfort, bone or muscle pain, etc.); and
  • Is willing and able to complete the study questionnaires and participate in a face‐to‐face or telephone interview session.

Exclusion Criteria:

  • Prior treatment with a licensed or experimental smoothened inhibitor (SMOi)
  • Targeted anti‐cancer therapy up to 14 days prior to enrolment (note prior treatment with best available therapy such as hydroxyurea is allowed)
  • Has ruxolitinib discontinuation syndrome; and
  • Subject has a
    • History of drug or alcohol abuse within the last 6 months,
    • Cognitive impairment,
    • Psychiatric condition or
    • Any other clinically relevant concern (e.g., impaired function due to current use of narcotics for pain treatment), that, in the opinion of the investigator, would interfere with the patient's ability to provide written informed consent and/or participate in the study procedures or interfere with the interpretation of the study results.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Ruben Mesa, M.D.

Closed for enrollment

Contact information:

Cancer Center Clinical Trials Referral Office

(855) 776-0015

More information

Publications

Publications are currently not available
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CLS-20492624

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