Trial Outcomes & Findings for PD-1 Inhibition in Advanced Myeloproliferative Neoplasms (NCT NCT03065400)
NCT ID: NCT03065400
Last Updated: 2021-06-21
Results Overview
The proportion of treated MF-CP patients (primary cohort) that achieve at least a clinical improvement (CI, PR, CR) by combined European Leukemia Net -International Working Group (ELN-IWG) criteria after 6 cycles of pembrolizumab therapy. Complete Remission - CR: Bone marrow: Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF and Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥ 1 × 109/L and \<UNL; Platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH Partial Remission (PR): Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH or Bone marrow: Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF, and Hemoglobin ≥85 but \<100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; plat
COMPLETED
PHASE2
10 participants
18 weeks
2021-06-21
Participant Flow
Participant milestones
| Measure |
Pembolizumab
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
| Measure |
Pembolizumab
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Physician Decision
|
3
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Completed 6 cycles but did not meet criteria to continue past cycle 6
|
5
|
Baseline Characteristics
PD-1 Inhibition in Advanced Myeloproliferative Neoplasms
Baseline characteristics by cohort
| Measure |
Pembolizumab
n=10 Participants
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 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
|
|
Age, Continuous
|
70 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 18 weeksThe proportion of treated MF-CP patients (primary cohort) that achieve at least a clinical improvement (CI, PR, CR) by combined European Leukemia Net -International Working Group (ELN-IWG) criteria after 6 cycles of pembrolizumab therapy. Complete Remission - CR: Bone marrow: Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF and Peripheral blood: Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥ 1 × 109/L and \<UNL; Platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH Partial Remission (PR): Hemoglobin ≥100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; platelet count ≥100 × 109/L and \<UNL; \<2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH or Bone marrow: Age-adjusted normocellularity; \<5% blasts; ≤grade 1 MF, and Hemoglobin ≥85 but \<100 g/L and \<UNL; neutrophil count ≥1 × 109/L and \<UNL; plat
Outcome measures
| Measure |
Pembolizumab
n=10 Participants
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
European Leukemia Net -International Working Group (ELN-IWG) Criteria
|
0 Participants
|
SECONDARY outcome
Timeframe: 18 weeksThe proportion of treated MPN-AP/BP patients (exploratory cohort) that achieve at least a complete morphologic remission of the leukemic blasts (CR, Cri) by Acute Myeloid Leukemia Response Criteria within 6 cycles of pembrolizumab therapy. Acute Myeloid Leukemia Response Assessment Criteria: Complete Response (CR) - The subject must be free of all symptoms related to leukemia and have an absolute neutrophil count of greater than 1 x 109/L, no need for red blood cell transfusion, platelet count greater than 100x 109/L, and normal marrow differential (\<5% blasts) in a normo- or hypercellular marrow Complete Remission with Incomplete Hematologic Recovery (Cri) - As per CR but incomplete count recovery Partial Response - CR with 6-25% abnormal cells in the marrow or 50% decrease in bone marrow blasts
Outcome measures
| Measure |
Pembolizumab
n=10 Participants
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
Acute Myeloid Leukemia Response Criteria
|
0 Participants
|
Adverse Events
Pembolizumab
Serious adverse events
| Measure |
Pembolizumab
n=10 participants at risk
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
Nervous system disorders
Weakness
|
10.0%
1/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
10.0%
1/10 • 18 weeks
|
|
Cardiac disorders
Supraventricular Tachycardia
|
10.0%
1/10 • 18 weeks
|
|
Reproductive system and breast disorders
High grade pleomorphic sarcoma of left breast.
|
10.0%
1/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiraitory Failure
|
10.0%
1/10 • 18 weeks
|
Other adverse events
| Measure |
Pembolizumab
n=10 participants at risk
200 mg of Pembolizumab administered via intravenous infusion over 30 mins given every 3 weeks
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
90.0%
9/10 • 18 weeks
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
50.0%
5/10 • 18 weeks
|
|
Blood and lymphatic system disorders
Leukopenia
|
40.0%
4/10 • 18 weeks
|
|
Blood and lymphatic system disorders
Lymphopenia
|
50.0%
5/10 • 18 weeks
|
|
Blood and lymphatic system disorders
WBC Decreeased
|
40.0%
4/10 • 18 weeks
|
|
Hepatobiliary disorders
AST Increased
|
30.0%
3/10 • 18 weeks
|
|
Endocrine disorders
Hyperglycemia
|
50.0%
5/10 • 18 weeks
|
|
Renal and urinary disorders
Hyperuricemia
|
40.0%
4/10 • 18 weeks
|
|
Hepatobiliary disorders
Hypoalbuminemia
|
30.0%
3/10 • 18 weeks
|
|
Hepatobiliary disorders
ALP Increased
|
20.0%
2/10 • 18 weeks
|
|
Renal and urinary disorders
Hypocalcaemia
|
20.0%
2/10 • 18 weeks
|
|
Renal and urinary disorders
Hypernatremia
|
20.0%
2/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
50.0%
5/10 • 18 weeks
|
|
General disorders
Fatigue
|
50.0%
5/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
50.0%
5/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Chest Pain
|
30.0%
3/10 • 18 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
30.0%
3/10 • 18 weeks
|
|
Nervous system disorders
Dizziness
|
30.0%
3/10 • 18 weeks
|
|
General disorders
Fever
|
30.0%
3/10 • 18 weeks
|
|
Nervous system disorders
Headache
|
40.0%
4/10 • 18 weeks
|
|
Gastrointestinal disorders
Nausea
|
30.0%
3/10 • 18 weeks
|
|
Nervous system disorders
Weakness
|
30.0%
3/10 • 18 weeks
|
|
Nervous system disorders
Blurred Vision
|
20.0%
2/10 • 18 weeks
|
|
Gastrointestinal disorders
Constipation
|
20.0%
2/10 • 18 weeks
|
|
Gastrointestinal disorders
Dyspepsia
|
20.0%
2/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Productive Cough
|
20.0%
2/10 • 18 weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
20.0%
2/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Sore Throat
|
20.0%
2/10 • 18 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
20.0%
2/10 • 18 weeks
|
|
General disorders
Weight Loss
|
20.0%
2/10 • 18 weeks
|
Additional Information
Mikaela Dougherty
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place