HOPE-PMF: Pre-fibrotic/Early Primary Myelofibrosis or Overt Primary Myelofibrosis

Purpose of this Study

We are doing this study to find the most effective, safe dose of an experimental drug called P1101 (the study drug) in adults with early or low risk primary myelofibrosis (PMF).

Who Can Participate?

Eligibility

Adults ages 18+ who:
  • Are diagnosed with pre-fibrotic/early PMF or overt PMF at low or intermediate-1 risk according to DIPSS plus risk calculator
  • Have good liver function
  • Have a hemoglobin level of 10.0 g/dL at start of study
  • Do not have any hypersensitivity or contraindication to interferon
  • Are not eligible for JAK inhibitor therapy
For more information, contact the study team at erin.murphy@duke.edu.

Age Range

18-110

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

If you are able to take part in this research study, you will be randomly assigned to receive P1101 or a placebo (a liquid without the medication). Screening Visit (visit window: -28 days to -1 day) - tests to determine your eligibility to participate. Baseline Visit (Week 0) Dose Escalation Period (Week 2 to Week 4 visits) Study medication injection during the Core Study Drug Period: During the Baseline Visit and Dose Escalation Period, you will receive P1101 (study group) or placebo (placebo group) during your clinic visit on the following schedule: Baseline visit (Day 0) = 250 mcg; Week 2 = 350 mcg; Week 4 = 500mcg Core Study Drug Period (Week 8 to Week 76 visits) - visits will occur roughly every 4 weeks during the 76-week Core Study Drug Period End of Core Study Drug Visit (Week 80 of receiving P1101/placebo) Safety Follow-up Visit for the Core Study Phase (~28 days after the last dose of P1101/placebo) - If you participate in the Blinded Extension Phase, the Safety Follow-up Visit will also serve as the Screening Visit for the Blinded Extension Phase. On-site biweekly study procedures for visits for whom self-injection is not possible (every 2 weeks) Early Termination Visit During the study visits the following tests may be done physical examinations, echocardiogram, eye exam, vitals, blood and urine samples collected, electrocardiogram (ECG), chest x-ray, questionnaires, spleen measurements, and bone marrow samples. You are expected to be in this research study for roughly 2.5 years (80 weeks for the Core Study Phase, 4 weeks of Safety Follow-up Visit, and 60 weeks for the Blinded Extension Phase [separate consent form]) or longer, depending on disease progression. If you are found to be positive for drug antibodies at your safety follow-up (end of study), we will need to check again in 12 weeks and 24 weeks or until you test negative, whichever comes first.

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

No

Spanish Materials Available

No

Study Details

Full Title

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase 3 Clinical Study to Assess Efficacy and Safety of Ropeginterferon alfa-2b (P1101) in Adult Patients with Pre-fibrotic/Early Primary Myelofibrosis or Overt Primary Myelofibrosis at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

Principal Investigator

Lindsay
Rein

Protocol Number

PRO00118102

NCT ID

NCT06468033

Phase

III

Enrollment Status

Pending Open to Enrollment