Trial Outcomes & Findings for Pegasys® in Patients With Myeloproliferative Diseases (NCT NCT00452023)

NCT ID: NCT00452023

Last Updated: 2024-12-11

Results Overview

CR = Reduction of PLT to \<440x109/l and disappearance of thromboembolic events, without the use of anagrelide or hydroxyurea PR = Reduction of PLT by 50% but still \>440x109/l or reduction of thromboembolic events by 50%, without the use of anagrelide or hydroxyurea

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

83 participants

Primary outcome timeframe

Up to 18 years

Results posted on

2024-12-11

Participant Flow

Participant milestones

Participant milestones
Measure
IFN-alpha2a 450 Microgram (mcg) Injection
Starting dose 450 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 450 microgram (mcg) injection under the skin once a week
IFN-alpha2a 360 Microgram (mcg) Injection
Starting dose 360 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 360 microgram (mcg) injection under the skin once a week
IFN-alpha2a 270 Microgram (mcg) Injection
Starting dose 270 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 270 microgram (mcg) injection under the skin once a week
IFN-alpha2a 180 Microgram (mcg) Injection
Starting dose 180 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 180 microgram (mcg) injection under the skin once a week
IFN-alpha2a 90 Microgram (mcg) Injection
Starting dose 90 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 90 microgram (mcg) injection under the skin once a week
Overall Study
STARTED
3
3
19
26
32
Overall Study
COMPLETED
3
3
19
26
32
Overall Study
NOT COMPLETED
0
0
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pegasys® in Patients With Myeloproliferative Diseases

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
IFN-alpha2a 450 Microgram (mcg)
n=3 Participants
Starting dose 450 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 450 microgram (mcg) injection under the skin once a week
IFN-alpha2a 360 Microgram (mcg)
n=3 Participants
Starting dose 360 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 360 microgram (mcg) injection under the skin once a week
IFN-alpha2a 270 Microgram (mcg)
n=19 Participants
Starting dose 270 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 270 microgram (mcg) injection under the skin once a week
IFN-alpha2a 180 Microgram (mcg)
n=26 Participants
Starting dose 180 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 180 microgram (mcg) injection under the skin once a week
IFN-alpha2a 90 Microgram (mcg)
n=32 Participants
Starting dose 90 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 90 microgram (mcg) injection under the skin once a week
Total
n=83 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
0 Participants
n=30 Participants
Age, Categorical
Between 18 and 65 years
3 Participants
n=99 Participants
3 Participants
n=107 Participants
15 Participants
n=206 Participants
19 Participants
n=7 Participants
27 Participants
n=31 Participants
67 Participants
n=30 Participants
Age, Categorical
>=65 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
4 Participants
n=206 Participants
7 Participants
n=7 Participants
5 Participants
n=31 Participants
16 Participants
n=30 Participants
Age, Continuous
47 years
n=99 Participants
33 years
n=107 Participants
58 years
n=206 Participants
54 years
n=7 Participants
52 years
n=31 Participants
53 years
n=30 Participants
Sex: Female, Male
Female
2 Participants
n=99 Participants
2 Participants
n=107 Participants
12 Participants
n=206 Participants
17 Participants
n=7 Participants
21 Participants
n=31 Participants
54 Participants
n=30 Participants
Sex: Female, Male
Male
1 Participants
n=99 Participants
1 Participants
n=107 Participants
7 Participants
n=206 Participants
9 Participants
n=7 Participants
11 Participants
n=31 Participants
29 Participants
n=30 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
1 Participants
n=7 Participants
0 Participants
n=31 Participants
1 Participants
n=30 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
1 Participants
n=7 Participants
1 Participants
n=31 Participants
2 Participants
n=30 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
0 Participants
n=30 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
1 Participants
n=7 Participants
2 Participants
n=31 Participants
3 Participants
n=30 Participants
Race (NIH/OMB)
White
3 Participants
n=99 Participants
3 Participants
n=107 Participants
19 Participants
n=206 Participants
23 Participants
n=7 Participants
29 Participants
n=31 Participants
77 Participants
n=30 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
0 Participants
n=30 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
0 Participants
n=7 Participants
0 Participants
n=31 Participants
0 Participants
n=30 Participants
Region of Enrollment
United States
3 participants
n=99 Participants
3 participants
n=107 Participants
19 participants
n=206 Participants
26 participants
n=7 Participants
32 participants
n=31 Participants
83 participants
n=30 Participants

PRIMARY outcome

Timeframe: Up to 18 years

CR = Reduction of PLT to \<440x109/l and disappearance of thromboembolic events, without the use of anagrelide or hydroxyurea PR = Reduction of PLT by 50% but still \>440x109/l or reduction of thromboembolic events by 50%, without the use of anagrelide or hydroxyurea

Outcome measures

Outcome measures
Measure
IFN-alpha2a 450 Microgram (mcg)
n=3 Participants
Starting dose 450 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 450 microgram (mcg) injection under the skin once a week
IFN-alpha2a 360 Microgram (mcg)
n=3 Participants
Starting dose 360 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 360 microgram (mcg) injection under the skin once a week
IFN-alpha2a 270 Microgram (mcg)
n=19 Participants
Starting dose 270 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 270 microgram (mcg) injection under the skin once a week
IFN-alpha2a 180 Microgram (mcg)
n=26 Participants
Starting dose 180 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 180 microgram (mcg) injection under the skin once a week
IFN-alpha2a 90 Microgram (mcg)
n=32 Participants
Starting dose 90 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 90 microgram (mcg) injection under the skin once a week
Number of Patients With Complete Response (CR) or Partial Response (PR)
3 Participants
3 Participants
14 Participants
23 Participants
23 Participants

Adverse Events

IFN-alpha2a 450 Microgram (mcg)

Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths

IFN-alpha2a 360 Microgram (mcg)

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

IFN-alpha2a 270 Microgram (mcg)

Serious events: 3 serious events
Other events: 9 other events
Deaths: 0 deaths

IFN-alpha2a 180 Microgram (mcg)

Serious events: 3 serious events
Other events: 8 other events
Deaths: 2 deaths

IFN-alpha2a 90 Microgram (mcg)

Serious events: 7 serious events
Other events: 7 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
IFN-alpha2a 450 Microgram (mcg)
n=3 participants at risk
Starting dose 450 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 450 microgram (mcg) injection under the skin once a week
IFN-alpha2a 360 Microgram (mcg)
n=3 participants at risk
Starting dose 360 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 360 microgram (mcg) injection under the skin once a week
IFN-alpha2a 270 Microgram (mcg)
n=19 participants at risk
Starting dose 270 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 270 microgram (mcg) injection under the skin once a week
IFN-alpha2a 180 Microgram (mcg)
n=26 participants at risk
Starting dose 180 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 180 microgram (mcg) injection under the skin once a week
IFN-alpha2a 90 Microgram (mcg)
n=32 participants at risk
Starting dose 90 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 90 microgram (mcg) injection under the skin once a week
Blood and lymphatic system disorders
Anemia
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Injury, poisoning and procedural complications
Ankle Fracture
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Cardiac disorders
Aortic Aneurism
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
carcinoma-facial
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Cardiac disorders
Cardiac Condition
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Surgical and medical procedures
Cardiac Surgery
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Injury, poisoning and procedural complications
Fatal Traffic Accident
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
General disorders
Fever
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Vascular disorders
Hypertension
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Metabolism and nutrition disorders
Invasive adenocarcinoma of the colon
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Injury, poisoning and procedural complications
Leg wound
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 3 • Up to 18 years, one month and 23 days
Infections and infestations
Lung Infection
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Infections and infestations
Pneumonia
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Surgical and medical procedures
Surgery for post- menopausal bleeding
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Surgical and medical procedures
Resection of Small Intestine
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Nervous system disorders
Seizure
33.3%
1/3 • Number of events 4 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Vascular disorders
Thromboembolic event
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Vascular disorders
Vasculitis
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days

Other adverse events

Other adverse events
Measure
IFN-alpha2a 450 Microgram (mcg)
n=3 participants at risk
Starting dose 450 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 450 microgram (mcg) injection under the skin once a week
IFN-alpha2a 360 Microgram (mcg)
n=3 participants at risk
Starting dose 360 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 360 microgram (mcg) injection under the skin once a week
IFN-alpha2a 270 Microgram (mcg)
n=19 participants at risk
Starting dose 270 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 270 microgram (mcg) injection under the skin once a week
IFN-alpha2a 180 Microgram (mcg)
n=26 participants at risk
Starting dose 180 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 180 microgram (mcg) injection under the skin once a week
IFN-alpha2a 90 Microgram (mcg)
n=32 participants at risk
Starting dose 90 microgram (mcg) injection under the skin once a week. IFN-alpha2a: Starting dose 90 microgram (mcg) injection under the skin once a week
Blood and lymphatic system disorders
Neutropenia
0.00%
0/3 • Up to 18 years, one month and 23 days
100.0%
3/3 • Number of events 3 • Up to 18 years, one month and 23 days
31.6%
6/19 • Number of events 6 • Up to 18 years, one month and 23 days
15.4%
4/26 • Number of events 4 • Up to 18 years, one month and 23 days
6.2%
2/32 • Number of events 2 • Up to 18 years, one month and 23 days
Investigations
Elevated transaminase
66.7%
2/3 • Number of events 2 • Up to 18 years, one month and 23 days
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 2 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
6.2%
2/32 • Number of events 2 • Up to 18 years, one month and 23 days
General disorders
Fatigue
0.00%
0/3 • Up to 18 years, one month and 23 days
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
General disorders
Pain
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Infections and infestations
Infection
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Psychiatric disorders
Depression
0.00%
0/3 • Up to 18 years, one month and 23 days
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
6.2%
2/32 • Number of events 2 • Up to 18 years, one month and 23 days
Eye disorders
Blur Vision
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Nervous system disorders
Dizziness
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Gastrointestinal disorders
Mucositis
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
5.3%
1/19 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Skin and subcutaneous tissue disorders
Rash
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Metabolism and nutrition disorders
Hyponatremia
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
3.8%
1/26 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days
Blood and lymphatic system disorders
Anemia
0.00%
0/3 • Up to 18 years, one month and 23 days
33.3%
1/3 • Number of events 1 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
0.00%
0/26 • Up to 18 years, one month and 23 days
3.1%
1/32 • Number of events 1 • Up to 18 years, one month and 23 days
Respiratory, thoracic and mediastinal disorders
Dyspnea
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/3 • Up to 18 years, one month and 23 days
0.00%
0/19 • Up to 18 years, one month and 23 days
7.7%
2/26 • Number of events 2 • Up to 18 years, one month and 23 days
0.00%
0/32 • Up to 18 years, one month and 23 days

Additional Information

Lucia Masarova MD/Assistant Professor

The University of Texas MD Anderson Cancer Center

Phone: 832-750-4211

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place