Trial Outcomes & Findings for P1101 in Treating Patients With Myelofibrosis (NCT NCT02370329)
NCT ID: NCT02370329
Last Updated: 2025-10-27
Results Overview
Patients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Up to 3 years
Results posted on
2025-10-27
Participant Flow
Participant milestones
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Overall Study
Alternate treatment
|
1
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Disease progression
|
3
|
|
Overall Study
Physician Decision
|
2
|
|
Overall Study
Study closure
|
4
|
Baseline Characteristics
P1101 in Treating Patients With Myelofibrosis
Baseline characteristics by cohort
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 Participants
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Age, Continuous
|
69 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=5 Participants
|
|
Myelofibrosis Risk Stage
Low/Intermediate-1 risk stage
|
4 Participants
n=5 Participants
|
|
Myelofibrosis Risk Stage
Intermediate-2/high risk stage
|
7 Participants
n=5 Participants
|
|
ECOG Performance Status
0
|
8 Participants
n=5 Participants
|
|
ECOG Performance Status
1
|
2 Participants
n=5 Participants
|
|
ECOG Performance Status
2
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 3 yearsPatients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.
Outcome measures
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 Participants
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria
Clinical Improvement
|
7 Participants
|
|
Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria
Stable Disease
|
3 Participants
|
|
Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria
Progression
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 3 yearsThe distribution of survival time will be estimated using the method of Kaplan-Meier.
Outcome measures
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 Participants
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Progression-free Survival Time
|
NA months
Interval 31.9 to
The median and upper limit of the confidence interval were not reached due to a low number of events.
|
SECONDARY outcome
Timeframe: Up to 3 yearsThe 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.
Outcome measures
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 Participants
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
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 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline to up to 3 yearsPatient-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.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (PEG-proline-interferon alpha-2b)
Serious events: 8 serious events
Other events: 11 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 participants at risk
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Cardiac disorders
Chest pain - cardiac
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Cardiac disorders
Heart failure
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Cardiac disorders
Sinus bradycardia
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Oth spec
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Immune system disorders
Allergic reaction
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Infections and infestations
Appendicitis
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Infections and infestations
Sepsis
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Injury, poisoning and procedural complications
Bruising
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Investigations
CPK increased
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Investigations
Neutrophil count decreased
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Investigations
White blood cell decreased
|
18.2%
2/11 • Number of events 3 • 3 years
|
|
Metabolism and nutrition disorders
Dehydration
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Metabolism and nutrition disorders
Tumor lysis syndrome
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Renal and urinary disorders
Renal and urinary disorders - Oth spec
|
9.1%
1/11 • Number of events 1 • 3 years
|
Other adverse events
| Measure |
Treatment (PEG-proline-interferon alpha-2b)
n=11 participants at risk
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. All patients will be started at a dose level of 100 micrograms (mcg) every other week, and then dose will be titrated up by 50mcg increments to a maximum dose of 300mcg every other week as tolerated.
\>
\> Laboratory Biomarker Analysis: Correlative studies
\>
\> Quality-of-Life Assessment: Ancillary studies
\>
\> Ropeginterferon Alfa-2B: Given SC
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
81.8%
9/11 • Number of events 53 • 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
45.5%
5/11 • Number of events 39 • 3 years
|
|
Gastrointestinal disorders
Nausea
|
27.3%
3/11 • Number of events 17 • 3 years
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
General disorders and administration site conditions
Fatigue
|
36.4%
4/11 • Number of events 21 • 3 years
|
|
Infections and infestations
Sinusitis
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Injury, poisoning and procedural complications
Fracture
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Investigations
Alkaline phosphatase increased
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Investigations
CD4 lymphocytes decreased
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Investigations
Lymphocyte count decreased
|
45.5%
5/11 • Number of events 36 • 3 years
|
|
Investigations
Lymphocyte count increased
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Investigations
Neutrophil count decreased
|
54.5%
6/11 • Number of events 57 • 3 years
|
|
Investigations
Platelet count decreased
|
27.3%
3/11 • Number of events 40 • 3 years
|
|
Investigations
Weight loss
|
27.3%
3/11 • Number of events 4 • 3 years
|
|
Investigations
White blood cell decreased
|
72.7%
8/11 • Number of events 133 • 3 years
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Nervous system disorders
Dizziness
|
18.2%
2/11 • Number of events 5 • 3 years
|
|
Nervous system disorders
Nervous system disorders - Oth spec
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Psychiatric disorders
Depression
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Psychiatric disorders
Insomnia
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
9.1%
1/11 • Number of events 2 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Resp, thoracic, mediastinal - Oth spec
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnea
|
9.1%
1/11 • Number of events 16 • 3 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcut tissue disord - Oth spec
|
9.1%
1/11 • Number of events 1 • 3 years
|
|
Vascular disorders
Hypertension
|
9.1%
1/11 • Number of events 2 • 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place