Trial Outcomes & Findings for Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia (NCT NCT00233454)
NCT ID: NCT00233454
Last Updated: 2018-09-20
Results Overview
Clinical Response \[PR + CR\] will be assessed after 2 cycles of treatment, with each cycle being 28 days (4 weeks) in length. Except as otherwise noted, the minimum criteria for PR is improvement by at least 50% from the baseline value towards the indicated value for one or more of the criteria below: BONE MARROW \& BLOOD * ANC \<1000/uL * Hb \<10 g/dL * Platelets \>100,000/uL LIVER * If hepatomegaly with ascites, decrease in frequency of paracenteses by 50% * Elevated enzyme levels \> upper limit of normal (ULN) * Hypoalbuminemia \< ULN * Portal hypertension \> ULN SPLEEN * If palpable splenomegaly with hypersplenism/thrombocytopenia, hypersplenism markers improved GI TRACT * If malabsorption with hypoalbuminemia and/or weight loss, albumin improved BONES * If huge osteolyses or/and severe osteoporosis with pathologic fractures, partial resolution of osteolyses Subjects with PR or greater continue, those without response discontinue.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
26 participants
Primary outcome timeframe
2 months
Results posted on
2018-09-20
Participant Flow
Participant milestones
| Measure |
Midostaurin
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Overall Study
STARTED
|
26
|
|
Overall Study
2 Months (for Response Rate)
|
26
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Midostaurin
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Overall Study
Death
|
6
|
Baseline Characteristics
Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia
Baseline characteristics by cohort
| Measure |
Midostaurin
n=26 Participants
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
13 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=5 Participants
|
|
Age, Continuous
|
65 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
25 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
26 Participants
n=5 Participants
|
|
Disease Sub-type
Aggressive systemic mastocytosis (ASM)
|
3 Participants
n=5 Participants
|
|
Disease Sub-type
SM with associated hematologic neoplasm (SM-AHN)
|
17 Participants
n=5 Participants
|
|
Disease Sub-type
Mast cell leukemia (MCL)
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 monthsClinical Response \[PR + CR\] will be assessed after 2 cycles of treatment, with each cycle being 28 days (4 weeks) in length. Except as otherwise noted, the minimum criteria for PR is improvement by at least 50% from the baseline value towards the indicated value for one or more of the criteria below: BONE MARROW \& BLOOD * ANC \<1000/uL * Hb \<10 g/dL * Platelets \>100,000/uL LIVER * If hepatomegaly with ascites, decrease in frequency of paracenteses by 50% * Elevated enzyme levels \> upper limit of normal (ULN) * Hypoalbuminemia \< ULN * Portal hypertension \> ULN SPLEEN * If palpable splenomegaly with hypersplenism/thrombocytopenia, hypersplenism markers improved GI TRACT * If malabsorption with hypoalbuminemia and/or weight loss, albumin improved BONES * If huge osteolyses or/and severe osteoporosis with pathologic fractures, partial resolution of osteolyses Subjects with PR or greater continue, those without response discontinue.
Outcome measures
| Measure |
Midostaurin
n=26 Participants
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Subjects With Clinical Response [Partial Response (PR) + Complete Response (CR)]
|
25 participants
|
SECONDARY outcome
Timeframe: 11 monthsOverall survival will be assessed after 12 cycles of treatment, with each cycle being 28 days (4 weeks) in length. 12 cycles of treatment is considered to be about 11 months.
Outcome measures
| Measure |
Midostaurin
n=26 Participants
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Overall Survival (OS)
|
20 participants
|
SECONDARY outcome
Timeframe: 40 monthsOverall survival was assessed as the median duration of survival at the time of data cut-off, and reported with 95% confidence interval.
Outcome measures
| Measure |
Midostaurin
n=26 Participants
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Overall Survival (OS)
|
40 months
Interval 27.3 to 52.7
|
Adverse Events
Midostaurin
Serious events: 12 serious events
Other events: 26 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Midostaurin
n=26 participants at risk
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, hand swelling
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Diarrhea
|
11.5%
3/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
vomiting
|
3.8%
1/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
nausea
|
3.8%
1/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Dehydration
|
3.8%
1/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Small intestinal perforation
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Death NOS
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Death NOS-refactory sepsis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Fatigue
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Fever
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders - Other, increased fatigue with "cloudy thinkin
|
3.8%
1/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, Liver lesions-neuroendocrine tumor
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, Urosepsis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Lung infection
|
7.7%
2/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Scrotal infection
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Injury, poisoning and procedural complications
Fracture
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Lipase increased
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Platelet count decreased
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Blood bilirubin increased
|
3.8%
1/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
3.8%
1/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Cognitive disturbance
|
3.8%
1/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Nervous system disorders - Other, transient ischemic attack/ cerebrovascular accident
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, Hematuria
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Reproductive system and breast disorders
Scrotal pain
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
3.8%
1/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, pneumonia
|
3.8%
1/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, blistery lesions on hands and lower arms
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
Other adverse events
| Measure |
Midostaurin
n=26 participants at risk
100 mg midostaurin twice daily as oral capsules
Midostaurin: Midostaurin is a broad-spectrum protein kinase inhibitor, acting on conventional PKC isoforms (α, β, γ); PDFRβ; VEGFR2; Syk; PKCη; Flk-1; Flt3; Cdk1/B; PKA; c-Kit; c-Fgr; c-Src; VEGFR1; and EGFR
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
42.3%
11/26 • Number of events 12 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, lymphadenopathy
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
15.4%
4/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Cardiac disorders
Atrial fibrillation
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Cardiac disorders
Atrioventricular block first degree
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Cardiac disorders
Sinus bradycardia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Eye disorders
Blurred vision
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Eye disorders
Cataract
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Abdominal distension
|
26.9%
7/26 • Number of events 7 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Abdominal pain
|
19.2%
5/26 • Number of events 6 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Ascites
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Constipation
|
46.2%
12/26 • Number of events 12 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Diarrhea
|
38.5%
10/26 • Number of events 10 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Enterocolitis
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, increased saliva
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, bowel obstruction
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Nausea
|
92.3%
24/26 • Number of events 24 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Gastrointestinal disorders
Vomiting
|
69.2%
18/26 • Number of events 18 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Chills
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Edema limbs
|
34.6%
9/26 • Number of events 9 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Fatigue
|
50.0%
13/26 • Number of events 14 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Fever
|
23.1%
6/26 • Number of events 6 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Flu like symptoms
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Gait disturbance
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, altitude sickness
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, failure to thrive
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, fluid retention
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, malnutrition
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, transfusion reaction
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, weakness
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other, thrush
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
General disorders and administration site conditions - Other animal or insect bite
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Localized edema
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
General disorders
Pain
|
11.5%
3/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other, increased Hepatosplenomegaly
|
19.2%
5/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Hepatobiliary disorders
Cholecystitis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, (tooth, peritoneal catheter)
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, herpes lesion(nasal/oral)
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, herpes zoster
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, Lyme's disease
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, peritonitis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, skin (cellulitis)
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, staph infection
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, Urosepsis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Infections and infestations - Other, viral
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Lung infection
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Sepsis
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Sinusitis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Infections and infestations
Urinary tract infection
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Injury, poisoning and procedural complications
Bruising
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Injury, poisoning and procedural complications
Fracture
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Activated partial thromboplastin time prolonged
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Alanine aminotransferase increased
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Alkaline phosphatase increased
|
23.1%
6/26 • Number of events 6 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Aspartate aminotransferase increased
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Blood bilirubin increased
|
3.8%
1/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Creatinine increased
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Investigations - Other, tryptase increased
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Lipase increased
|
23.1%
6/26 • Number of events 9 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Platelet count decreased
|
30.8%
8/26 • Number of events 8 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Weight gain
|
15.4%
4/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Weight loss
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
White blood cell decreased
|
7.7%
2/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Investigations
Serum Amylase Increased
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Acidosis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Dehydration
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Glucose intolerance
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
23.1%
6/26 • Number of events 7 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
15.4%
4/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, rotator cuff tear
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, basal cell CA
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, squamous cell CA
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Treatment related secondary malignancy, lung CA, malignant neoplasm
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Cognitive disturbance
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Headache
|
30.8%
8/26 • Number of events 8 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Nervous system disorders - Other, lightheadedness
|
23.1%
6/26 • Number of events 6 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Nervous system disorders - Other, presyncope
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Nervous system disorders - Other, vertigo
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Nervous system disorders
Tremor
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Psychiatric disorders
Confusion
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Psychiatric disorders
Depression
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Psychiatric disorders
Insomnia
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Psychiatric disorders
Psychosis
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Psychiatric disorders
Anxiety
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Renal and urinary disorders
Acute kidney injury
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Renal and urinary disorders
Hematuria
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Renal and urinary disorders
Urinary frequency
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
26.9%
7/26 • Number of events 7 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, bronchitis
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other, Upper Respiratory Infection
|
30.8%
8/26 • Number of events 8 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
19.2%
5/26 • Number of events 5 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, dermatitis (blisters)
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, injury (abrasion)
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, rosacea
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, not specified
|
7.7%
2/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, skin nodule
|
7.7%
2/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, sunburn
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
3.8%
1/26 • Number of events 1 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Vascular disorders
Flushing
|
15.4%
4/26 • Number of events 4 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Vascular disorders
Hematoma
|
11.5%
3/26 • Number of events 3 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Vascular disorders
Hypotension
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
|
Vascular disorders
Vascular disorders - Other, conjunctival hemorrhage
|
7.7%
2/26 • Number of events 2 • This study includes open-ended maintenance treatment. AEs are reported for the duration of treatment through follow-up to death or lost-to-follow-up.
|
Additional Information
Jason R Gotlib, MD, Associate Professor of Medicine (Hematology)
Stanford University Medical Center
Phone: 650-867-2823
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place