A Phase 2 interventional study of Laboratory Biomarker Analysis and Quality-of-Life Assessment in Primary Myelofibrosis and Secondary Myelofibrosis, sponsored by Mayo Clinic. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-27.
Sponsored by Mayo Clinic · Phase 2, Interventional, and Treatment
This pilot phase II trial studies P1101 (polyethyleneglycol [PEG]-proline-interferon alpha-2b) in treating patients with myelofibrosis. PEG-proline-interferon alpha-2b is a substance that can improve the body's natural response and may slow the growth of myelofibrosis.
PRIMARY OBJECTIVE:
I. To evaluate for clinical response (complete remission [CR], partial remission [PR], or clinical improvement [CI]) as defined by International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria in a cohort of intermediate-2/high risk myelofibrosis (MF) patients. Response in a second cohort of early stage MF patients will also be described.
SECONDARY OBJECTIVES:
I. To evaluate the adverse event profile of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).
II. To evaluate the tolerability of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).
EXPLORATORY AND CORRELATIVE RESEARCH OBJECTIVES:
I. To evaluate quality of life (QOL) and patient-reported symptoms using the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) with P1101 for patients with myelofibrosis by cohort (early vs intermediate-2/high risk).
II. To evaluate the impact of P1101 on bone marrow and histological features of myelofibrosis including cytogenetics, blast percentage, fibrosis, and JAK2-V617F allele burden by cohort (early vs intermediate-2/high risk).
OUTLINE:
Patients receive PEG-proline-interferon alpha-2b subcutaneously (SC) on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3-6 months for 3 years.
419 studies on the registry are indexed under Primary Myelofibrosis; 117 are open to participants now.
This study's enrollment of 11 is below the median of 44 across 347 interventional studies indexed under Primary Myelofibrosis.
Browse Primary Myelofibrosis studies →Mayo Clinic is the lead sponsor of 3,218 studies on the registry; 670 are open to participants now.
Of its 445 completed or terminated interventional studies of FDA-regulated products, 313 (70%) have results posted.
Counted across the registry records on this site, refreshed daily.
Evaluable myelofibrosis by IWG-MRT criteria including one or more of the following:
Exclusion Criteria:
Any of the following:
Patients receive PEG-proline-interferon alpha-2b SC on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Other: Laboratory Biomarker Analysis · Other: Quality-of-Life Assessment · Biological: Ropeginterferon Alfa-2B
Correlative studies
Ancillary studies
Also known as: Quality of Life Assessment
Given SC
Also known as: AOP2014, Besremi, P-1101, P1101, PEG-P-IFN-Alfa-2b, PEG-P-IFN-Alpha-2b, PEG-Proline-Interferon Alfa-2b
Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria
Patients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.
Time frame: Up to 3 years
Progression-free Survival Time
The distribution of survival time will be estimated using the method of Kaplan-Meier.
Time frame: Up to 3 years
Number of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4)
The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
Time frame: Up to 3 years
Changes in Patient-reported Symptoms and QOL as Measured by MPN-SAF
Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range. Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated. Graphical procedures will include stream plots of individual patient scores and plots of average values over time. Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.
Time frame: Baseline to up to 3 years
| Milestone | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Started | 11 |
| Completed | 0 |
| Not completed | 11 |
| Withdrew: Alternate treatment | 1 |
| Withdrew: Adverse event | 1 |
| Withdrew: Disease progression | 3 |
| Withdrew: Physician decision | 2 |
| Withdrew: Study closure | 4 |
Patients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.
| Participants | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Clinical Improvement | 7 |
| Stable Disease | 3 |
| Progression | 1 |
The distribution of survival time will be estimated using the method of Kaplan-Meier.
| months | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Progression-free Survival Time | NA (31.9 to NA) |
The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
| Participants | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Number of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4) | 10 |
Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range. Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated. Graphical procedures will include stream plots of individual patient scores and plots of average values over time. Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.
Results for this outcome have not been posted.
Collected over 3 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (PEG-proline-interferon alpha-2b) | 3/11 (27.3%) | 8/11 (72.7%) | 11/11 (100%) |
| Event | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| White blood cell decreasedInvestigations | 2/11 |
| Atrial fibrillationCardiac disorders | 1/11 |
| Chest pain - cardiacCardiac disorders | 1/11 |
| Heart failureCardiac disorders | 1/11 |
| Sinus bradycardiaCardiac disorders | 1/11 |
| Gastrointestinal disorders - Oth specGastrointestinal disorders | 1/11 |
| Allergic reactionImmune system disorders | 1/11 |
| AppendicitisInfections and infestations | 1/11 |
| SepsisInfections and infestations | 1/11 |
| BruisingInjury, poisoning and procedural complications | 1/11 |
| Event | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| AnemiaBlood and lymphatic system disorders | 9/11 |
| White blood cell decreasedInvestigations | 8/11 |
| Neutrophil count decreasedInvestigations | 6/11 |
| DiarrheaGastrointestinal disorders | 5/11 |
| Lymphocyte count decreasedInvestigations | 5/11 |
| FatigueGeneral disorders and administration site conditions | 4/11 |
| NauseaGastrointestinal disorders | 3/11 |
| Platelet count decreasedInvestigations | 3/11 |
| Weight lossInvestigations | 3/11 |
| DizzinessNervous system disorders | 2/11 |
| Age, Continuous(years) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Median | 69 (39 to 88) |
| Sex: Female, Male(Participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Female | 5 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 10 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 11 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| United States | 11 |
| Myelofibrosis Risk Stage(Participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| Low/Intermediate-1 risk stage | 4 |
| Intermediate-2/high risk stage | 7 |
| ECOG Performance Status(Participants) | Treatment (PEG-proline-interferon alpha-2b) |
|---|---|
| 0 | 8 |
| 1 | 2 |
| 2 | 1 |
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