Trial Outcomes & Findings for Sotatercept in Treating Patients With Myeloproliferative Neoplasm-Associated Myelofibrosis or Anemia (NCT NCT01712308)
NCT ID: NCT01712308
Last Updated: 2023-10-10
Results Overview
Defined as an increase in hemoglobin level equal to or greater than 1.5 g/L without red blood cell-transfusion OR becoming red blood cell-transfusion-independent in a patient who is red blood cell-transfusion-dependent. Will be based on the intent-to-treat principle.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
56 participants
Primary outcome timeframe
Up to 84 days
Results posted on
2023-10-10
Participant Flow
Participant milestones
| Measure |
Treatment Monotherapy (Sotatercept)
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
|---|---|---|
|
Overall Study
STARTED
|
35
|
21
|
|
Overall Study
COMPLETED
|
35
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Sotatercept in Treating Patients With Myeloproliferative Neoplasm-Associated Myelofibrosis or Anemia
Baseline characteristics by cohort
| Measure |
Treatment Monotherapy (Sotatercept)
n=35 Participants
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
n=21 Participants
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
Total
n=56 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
|
14 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
21 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
38 Participants
n=5 Participants
|
|
Age, Continuous
|
66 years
n=5 Participants
|
71 years
n=7 Participants
|
67 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
35 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
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 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
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
43 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
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
35 participants
n=5 Participants
|
21 participants
n=7 Participants
|
56 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 84 daysPopulation: Of the 35 participants on the monotherapy arm, 27 were evaluable for response. Of the 21 participants on the Combination Therapy arm, 19 were evaluable for response.
Defined as an increase in hemoglobin level equal to or greater than 1.5 g/L without red blood cell-transfusion OR becoming red blood cell-transfusion-independent in a patient who is red blood cell-transfusion-dependent. Will be based on the intent-to-treat principle.
Outcome measures
| Measure |
Treatment Monotherapy (Sotatercept)
n=27 Participants
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
n=19 Participants
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
|---|---|---|
|
Anemia-response
|
8 Participants
|
6 Participants
|
PRIMARY outcome
Timeframe: Up to 9 yearsPopulation: Of the 35 participants on the monotherapy arm, 27 were evaluable for response. Of the 21 participants on the Combination Therapy arm, 19 were evaluable for response.
Response date to loss of response or last follow up.
Outcome measures
| Measure |
Treatment Monotherapy (Sotatercept)
n=27 Participants
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
n=19 Participants
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
|---|---|---|
|
Duration of Response
|
23.3 Months
Interval 3.9 to 68.4
|
19.9 Months
Interval 3.7 to 56.8
|
Adverse Events
Treatment Monotherapy (Sotatercept)
Serious events: 11 serious events
Other events: 30 other events
Deaths: 0 deaths
Treatment Combination (Ruxolitinib + Sotatercept)
Serious events: 10 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment Monotherapy (Sotatercept)
n=35 participants at risk
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
n=21 participants at risk
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
|---|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Blood and lymphatic system disorders
Anemia
|
8.6%
3/35 • Number of events 3 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
General disorders
Chest wall pain
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
General disorders
Chills
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Nervous system disorders
Dizziness
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Gastrointestinal disorders
Enterocolitis
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
General disorders
Fever
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Nervous system disorders
Headache
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Renal and urinary disorders
Hematuria
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Infections and infestations
Infections and infestations
|
0.00%
0/35 • Up to 9 years
|
9.5%
2/21 • Number of events 3 • Up to 9 years
|
|
Infections and infestations
Lung infection
|
0.00%
0/35 • Up to 9 years
|
14.3%
3/21 • Number of events 5 • Up to 9 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
General disorders
Non cardiac chest pain
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
General disorders
Pain in extremity
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Infections and infestations
Sepsis
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Infections and infestations
Skin infection
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
4.8%
1/21 • Number of events 2 • Up to 9 years
|
|
Gastrointestinal disorders
Vomiting
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Infections and infestations
Wound infection
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
Other adverse events
| Measure |
Treatment Monotherapy (Sotatercept)
n=35 participants at risk
Patients receive sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
|
Treatment Combination (Ruxolitinib + Sotatercept)
n=21 participants at risk
patients that are already on therapy with ruxolitinib (for at least for 6 months, and on stable dose for last 2 months) will continue ruxolitinib in addition to sotatercept SC once every 3 weeks. Cycles repeat every 3 weeks in the absence of disease progression or unacceptable toxicity. Patients unable to achieve anemia-response after 8 cycles will be taken off study.
Sotatercept: Given SC
Ruxolitinib
|
|---|---|---|
|
Immune system disorders
Allergic reaction
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Cardiac disorders
Cardiac disorders
|
14.3%
5/35 • Number of events 7 • Up to 9 years
|
23.8%
5/21 • Number of events 5 • Up to 9 years
|
|
Gastrointestinal disorders
Constipation
|
14.3%
5/35 • Number of events 5 • Up to 9 years
|
23.8%
5/21 • Number of events 5 • Up to 9 years
|
|
Gastrointestinal disorders
Diarrhea
|
25.7%
9/35 • Number of events 9 • Up to 9 years
|
38.1%
8/21 • Number of events 8 • Up to 9 years
|
|
Nervous system disorders
Dizziness
|
17.1%
6/35 • Number of events 8 • Up to 9 years
|
19.0%
4/21 • Number of events 5 • Up to 9 years
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/35 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
17.1%
6/35 • Number of events 6 • Up to 9 years
|
14.3%
3/21 • Up to 9 years
|
|
General disorders
Edema limbs
|
25.7%
9/35 • Number of events 9 • Up to 9 years
|
14.3%
3/21 • Number of events 3 • Up to 9 years
|
|
Investigations
Elevated Albumin/Creatinine Ratio
|
11.4%
4/35 • Number of events 4 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Investigations
elevated transaminase levels
|
2.9%
1/35 • Number of events 2 • Up to 9 years
|
14.3%
3/21 • Number of events 3 • Up to 9 years
|
|
Injury, poisoning and procedural complications
Fall
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
14.3%
3/21 • Number of events 4 • Up to 9 years
|
|
General disorders
Fatigue
|
17.1%
6/35 • Number of events 6 • Up to 9 years
|
14.3%
3/21 • Number of events 3 • Up to 9 years
|
|
General disorders
Fever
|
8.6%
3/35 • Number of events 3 • Up to 9 years
|
14.3%
3/21 • Number of events 5 • Up to 9 years
|
|
General disorders
Flu like symptoms
|
25.7%
9/35 • Number of events 28 • Up to 9 years
|
66.7%
14/21 • Number of events 31 • Up to 9 years
|
|
Vascular disorders
Flushing
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
28.6%
10/35 • Number of events 17 • Up to 9 years
|
42.9%
9/21 • Number of events 14 • Up to 9 years
|
|
General disorders
Headache
|
5.7%
2/35 • Number of events 3 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Vascular disorders
Hot flashes
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Vascular disorders
Hypertension
|
14.3%
5/35 • Number of events 7 • Up to 9 years
|
23.8%
5/21 • Number of events 5 • Up to 9 years
|
|
Endocrine disorders
Hypothyroidism
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Infections and infestations
Infection
|
11.4%
4/35 • Number of events 7 • Up to 9 years
|
14.3%
3/21 • Number of events 3 • Up to 9 years
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
5.7%
2/35 • Number of events 5 • Up to 9 years
|
4.8%
1/21 • Number of events 2 • Up to 9 years
|
|
Psychiatric disorders
Insomnia
|
11.4%
4/35 • Number of events 4 • Up to 9 years
|
28.6%
6/21 • Number of events 6 • Up to 9 years
|
|
Metabolism and nutrition disorders
Iron overload
|
0.00%
0/35 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Vascular disorders
Lymphedema
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 2 • Up to 9 years
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/35 • Up to 9 years
|
19.0%
4/21 • Number of events 5 • Up to 9 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder
|
25.7%
9/35 • Number of events 16 • Up to 9 years
|
28.6%
6/21 • Number of events 11 • Up to 9 years
|
|
Gastrointestinal disorders
Nausea
|
8.6%
3/35 • Number of events 3 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
General disorders
Pain
|
45.7%
16/35 • Number of events 34 • Up to 9 years
|
42.9%
9/21 • Number of events 22 • Up to 9 years
|
|
Nervous system disorders
Paresthesia
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
Nervous system disorders
Peripheral neuropathy
|
11.4%
4/35 • Number of events 4 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/35 • Up to 9 years
|
19.0%
4/21 • Number of events 4 • Up to 9 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo papular
|
11.4%
4/35 • Number of events 4 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Renal and urinary disorders
Renal and urinary disorders
|
28.6%
10/35 • Number of events 13 • Up to 9 years
|
33.3%
7/21 • Number of events 9 • Up to 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/35 • Up to 9 years
|
4.8%
1/21 • Number of events 2 • Up to 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
Infections and infestations
Sinusitis
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
0.00%
0/21 • Up to 9 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
22.9%
8/35 • Number of events 9 • Up to 9 years
|
23.8%
5/21 • Number of events 10 • Up to 9 years
|
|
Ear and labyrinth disorders
Tinnitus
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
9.5%
2/21 • Number of events 2 • Up to 9 years
|
|
Nervous system disorders
Tremor
|
2.9%
1/35 • Number of events 1 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Gastrointestinal disorders
Vomiting
|
8.6%
3/35 • Number of events 5 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
Investigations
Weight loss
|
5.7%
2/35 • Number of events 2 • Up to 9 years
|
4.8%
1/21 • Number of events 1 • Up to 9 years
|
|
General disorders
Bleeding Hemorrhage
|
22.9%
8/35 • Number of events 13 • Up to 9 years
|
28.6%
6/21 • Number of events 10 • Up to 9 years
|
|
Psychiatric disorders
Psychiatric Disorders
|
11.4%
4/35 • Number of events 4 • Up to 9 years
|
33.3%
7/21 • Number of events 7 • Up to 9 years
|
Additional Information
Prithviraj Bose MD/Associate Professor
The University of Texas MD Anderson Cancer
Phone: 713-792-7747
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place