Trial Outcomes & Findings for HSP90 Inhibitor, AUY922, in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET (NCT NCT01668173)

Last Updated: 2016-05-23

Results Overview

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

7 participants

Primary outcome timeframe

6 months

Results posted on

2016-05-23

Participant Flow

Participant milestones

Participant milestones
Measure	All Patients HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
Overall Study STARTED	7
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
Overall Study Adverse Event	3
Overall Study Trial suspension	4

Baseline Characteristics

HSP90 Inhibitor, AUY922, in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=7 Participants HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	All Patients n=1 Participants HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
Overall Objective Response	1 participant with Stable Disease

Adverse Events

All Patients

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=7 participants at risk HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
Gastrointestinal disorders Colonic hemorrhage	14.3% 1/7 • Number of events 1
Gastrointestinal disorders Diarrhea	14.3% 1/7 • Number of events 1
Gastrointestinal disorders Ileal ulcer	14.3% 1/7 • Number of events 1
Gastrointestinal disorders Nausea	14.3% 1/7
Psychiatric disorders Psychiatric disorders - Other	14.3% 1/7 • Number of events 1
Gastrointestinal disorders Vomiting	14.3% 1/7

Other adverse events

Other adverse events
Measure	All Patients n=7 participants at risk HSP90 Inhibitor, AUY922, in Patients with Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), Post-Essential Thrombocythemia Myelofibrosis (Post-ET MF), and Refractory PV/ET
General disorders Pain	14.3% 1/7
Blood and lymphatic system disorders Anemia	100.0% 7/7
Gastrointestinal disorders Nausea	42.9% 3/7
Gastrointestinal disorders Colitis	14.3% 1/7
General disorders Fatigue	71.4% 5/7
Musculoskeletal and connective tissue disorders Myalgia	14.3% 1/7
General disorders Anorexia	14.3% 1/7
General disorders Bloating	14.3% 1/7
Gastrointestinal disorders Diarrhea	71.4% 5/7
General disorders Insomnia	14.3% 1/7
Skin and subcutaneous tissue disorders Pruritus	14.3% 1/7
Gastrointestinal disorders Vomiting	28.6% 2/7
General disorders Back pain	14.3% 1/7
General disorders Bone pain	14.3% 1/7
Musculoskeletal and connective tissue disorders Arthralgia	14.3% 1/7
Gastrointestinal disorders Dehydration	14.3% 1/7
Metabolism and nutrition disorders Hypokalemia	14.3% 1/7
Skin and subcutaneous tissue disorders Erythroderma	14.3% 1/7
Metabolism and nutrition disorders Hyperkalemia	28.6% 2/7
Metabolism and nutrition disorders Hypocalcemia	71.4% 5/7
Metabolism and nutrition disorders CPK increased	14.3% 1/7
Metabolism and nutrition disorders Hyperglycemia	28.6% 2/7
Skin and subcutaneous tissue disorders Rash pustular	14.3% 1/7
General disorders Abdominal pain	28.6% 2/7
General disorders Blurred vision	14.3% 1/7
Metabolism and nutrition disorders Hypoalbuminemia	14.3% 1/7
General disorders Night blindness	42.9% 3/7
Metabolism and nutrition disorders Hypophosphatemia	28.6% 2/7
Metabolism and nutrition disorders Lipase increased	14.3% 1/7
Skin and subcutaneous tissue disorders Papulopustular rash	14.3% 1/7
Skin and subcutaneous tissue disorders Rash maculo-papular	28.6% 2/7
Metabolism and nutrition disorders Creatinine increased	28.6% 2/7
Metabolism and nutrition disorders Serum amylase increased	14.3% 1/7
Blood and lymphatic system disorders Platelet count decreased	28.6% 2/7
Metabolism and nutrition disorders Blood bilirubin increased	14.3% 1/7
Nervous system disorders Peripheral sensory neuropathy	14.3% 1/7
Metabolism and nutrition disorders Alkaline phosphatase increased	28.6% 2/7
Gastrointestinal disorders Lower gastrointestinal hemorrhage	14.3% 1/7
Psychiatric disorders Psychiatric disorders - Other	14.3% 1/7

Additional Information

Dr. Raajit Rampal

Memorial Sloan Kettering Cancer Center

Phone: 212-639-2194

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place