Trial Outcomes & Findings for Pegylated Interferon Alpha-2b in Early Primary Myelofibrosis (NCT NCT01758588)

Last Updated: 2018-07-24

Results Overview

Clinical improvement (CI) Requires one of the following in the absence of both disease progression (as outlined below) and Complete Response (CR)/Partial Response (PR) assignment (CI response is validated only if it lasts for no fewer than 8 weeks) i. A minimum 20-g/L increase in hemoglobin level or becoming transfusion independent (applicable only for patients with baseline hemoglobin level of less than 100 g/L). ii. Either a minimum 50% reduction in palpable splenomegaly of a spleen that is at least 10 cm at baseline or a spleen that is palpable at more than 5 cm at baseline becomes not palpable. iii. A minimum 100% increase in platelet count and an absolute platelet count of at least 50 000 109/L (applicable only for patients with baseline platelet count below 50 109/L). iv. A minimum 100% increase in Absolute Neutrophil Count (ANC) and an ANC of at least 0.5 109/L (applicable only for patients with baseline absolute neutrophil count below 1 109/L).

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

One year

Results posted on

2018-07-24

Participant Flow

Patients were randomized to either the treatment arm or the control arm of the study with a 2:1 allocation ratio. 8 patients were enrolled. No patients crossed over from observation to treatment.

Participant milestones

Participant milestones
Measure	Treatment Arm 50mcg peginterferon alfa-2b subcutaneously per week	Observation Arm Patients were observed and did not receive treatment.
Overall Study STARTED	5	3
Overall Study COMPLETED	5	3
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pegylated Interferon Alpha-2b in Early Primary Myelofibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Arm n=5 Participants Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 Participants Patients who did not receive treatment and were observed only.	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Age, Categorical >=65 years	4 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants
Age, Continuous	71.9 years STANDARD_DEVIATION 6.2 • n=5 Participants	69.1 years STANDARD_DEVIATION 5.5 • n=7 Participants	69.9 years STANDARD_DEVIATION 7.1 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States · Weill Cornell Medicine	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Region of Enrollment United States · Emory University	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: One year

Outcome measures

Outcome measures
Measure	Treatment Arm n=5 Participants Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 Participants Patients who did not receive treatment and were observed only.
Clinical Improvement Achieved Clinical Improvement (CI)	1 Participants	0 Participants
Clinical Improvement Did not achieve Clinical Improvement (CI)	2 Participants	2 Participants
Clinical Improvement Not evaluable	2 Participants	1 Participants

SECONDARY outcome

Timeframe: Week 21

Population: Analysis population includes subjects who were evaluable at 21 weeks from time of study entry.

Progression free survival is the measure of subject survival in the absence of disease progression. Disease progression is defined as progression to the next higher International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Dynamic International Prognostic Scoring System (DIPSS) stage from diagnosis. The IWG-MRT DIPSS stratifies primary myelofibrosis (PMF) into four risk categories (low, intermediate 1, intermediate 2, and high risk), based on 5 clinical factors; Age\>65, Hemoglobin \<10gm/dL, white blood cell (WBC)\>25,000/uL, peripheral blasts\>1%, and constitutional symptoms. Progression free survival will be assessed at 21 weeks from time of study entry.

Outcome measures

Outcome measures
Measure	Treatment Arm n=4 Participants Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 Participants Patients who did not receive treatment and were observed only.
Progression Free Survival Disease did not progress	2 Participants	3 Participants
Progression Free Survival Disease progressed	2 Participants	0 Participants

SECONDARY outcome

Timeframe: Week 21

Population: Due to early termination of the trial, there is not enough data to evaluate overall survival.

Overall survival measures subject survival regardless of disease progression. Overall survival will be assessed at 21 weeks from time of study entry.

Outcome measures

Outcome measures
Measure	Treatment Arm n=4 Participants Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 Participants Patients who did not receive treatment and were observed only.
Overall Survival Not deceased	4 Participants	3 Participants
Overall Survival Deceased	0 Participants	0 Participants

Adverse Events

Treatment Arm

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Observation Arm

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment Arm n=5 participants at risk Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 participants at risk Patients on the non-interventional arm.
Respiratory, thoracic and mediastinal disorders Pneumonia	20.0% 1/5 • Number of events 1 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.	0.00% 0/3 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.
Respiratory, thoracic and mediastinal disorders Hypoxia	20.0% 1/5 • Number of events 1 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.	0.00% 0/3 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.

Other adverse events

Other adverse events
Measure	Treatment Arm n=5 participants at risk Patients treated with 50mcg of peginterferon alfa-2b subcutaneously per week.	Observation Arm n=3 participants at risk Patients on the non-interventional arm.
Psychiatric disorders Depression	20.0% 1/5 • Number of events 1 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.	0.00% 0/3 • Adverse events were collected from the date that the first patient was consented until study termination. The definitions of adverse event and/or serious adverse event, used to collect adverse event information, do not differ from the clinicaltrials.gov definitions.

Additional Information

Assistant Director of Leukemia and BMT Clinical Trials

Weill Cornell Medicine

Phone: 646-962-9305

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place