Trial Outcomes & Findings for A Phase II Study of SGI-110 in Philadelphia-Negative Myeloproliferative Neoplasms (NCT NCT03075826)

Last Updated: 2022-05-05

Results Overview

The response to treatment will be measured from baseline through 12 cycles. A response assessment will be performed after cycle 3, cycle 6 and cycle 12. The response will be measured by IWG-MDS (International working group - Myelodysplastic Syndrome), which is a set of criteria that dictates what response a patient with MDS has had to a treatment; IWG-MF (International Working Group - Myelofibrosis) which is a set of criteria that dictates what response a patient with MF has had to a treatment. Both sets of criteria utilize the following parameters to asses response: Results from blood tests; bone marrow biopsies; physical exams to look at spleen and liver size; and symptoms. All responses will be recorded in our database and the best response to treatment will be reported. A cycle is at least 28 days, which can be extended past day 28 in order to allow physicians decision to delay next cycle based on the patients clinical needs.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

At the end of cycles 3, 6 and 12 - up to approximately 336 days

Results posted on

2022-05-05

Participant Flow

22 subjects were treated with SGI-110. Of the 22 that were treated 16 were evaluable for response to study drug.

Participant milestones

Participant milestones
Measure	Open Label-Single Arm This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Overall Study STARTED	22
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Open Label-Single Arm This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Overall Study Withdrawal by Subject	1
Overall Study Received less than 3 cycles of study drug	5

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open Label-Single Arm n=22 Participants This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Age, Categorical <=18 years	0 Participants n=22 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=22 Participants
Age, Categorical >=65 years	16 Participants n=22 Participants
Sex: Female, Male Female	10 Participants n=22 Participants
Sex: Female, Male Male	12 Participants n=22 Participants
Region of Enrollment United States	22 participants n=22 Participants

PRIMARY outcome

Timeframe: At the end of cycles 3, 6 and 12 - up to approximately 336 days

Population: 6 patients were inevaluable - 1 patient withdrew consent and 5 patients received less than 3 cycles of study drug.

Outcome measures

Outcome measures
Measure	Open Label-Single Arm n=16 Participants This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Complete remission	2 Participants
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Partial remission	0 Participants
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Complete cytogenetic remission	0 Participants
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Marrow response	3 Participants
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Clinical benefit	2 Participants
Number of Participants With Hematological Response, as Measured by Any One or More of the Following Response Assessments: IWG-MDS and IWG-MF Criteria as Accurate and Appropriate. Stable disease	9 Participants

SECONDARY outcome

Timeframe: Baseline, at the beginning of each new cycle - approximately every 28 days for up to 700 days

Population: Out of 22 patients treated, 21 patients were analyzed for at least one section of the MPN-SAF questionnaire (1 patient came off treatment after 1 cycle). 5 patients did not complete a baseline QOL.

Symptom improvement response is defined as a number of subjects with improvement in symptoms response as defined by Myeloproliferative Neoplasms Symptoms Assessment Form (MPN-SAF). The MPN-SAF is a questionnaire that measures certain symptoms and how they improve throughout the study, from baseline. The questionnaire is divided into 4 sections - 1: 16 questions about the most common symptoms in these disease types, rated from 0 (lowest impact) to 10 (biggest impact); 2: Highest grade of fever; 3: Unintentional weight loss; 4. Quality of life, rated from 0 (as good as it can be) to 10 (as bad as it can be).

Outcome measures

Outcome measures
Measure	Open Label-Single Arm n=16 Participants This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Number of Participants With Change in Quality of Life (QoL) Scores as Defined by Myeloproliferative Neoplasms Symptoms Assessment Form (MPN-SAF). Showed overall improvement in mean QOL from baseline	7 Participants
Number of Participants With Change in Quality of Life (QoL) Scores as Defined by Myeloproliferative Neoplasms Symptoms Assessment Form (MPN-SAF). Showed no change in mean QOL from baseline	4 Participants
Number of Participants With Change in Quality of Life (QoL) Scores as Defined by Myeloproliferative Neoplasms Symptoms Assessment Form (MPN-SAF). Showed overall decrease in mean QOL from baseline	5 Participants

Adverse Events

Open Label-Single Arm

Serious events: 17 serious events

Other events: 22 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Open Label-Single Arm n=22 participants at risk This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Gastrointestinal disorders Nausea	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Right hand MSSA cellulitis	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Febrile neutropenia	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Cellulitis	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Cardiac disorders Atrial fibrillation	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic lymphocytic leukemia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations COVID-19 infection	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Delirium	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Dyspnea	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations E.Coli Sepsis	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fall	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fatigue	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fever	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Gastrointestinal bleed	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations Legionella pnemonia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Myositis	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Peripheral ischemia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Platelet transfusion reaction	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations Pneumonia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Progression to AML	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations Salmonella diarrhea	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Severe anemia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Splenic pain	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Metabolism and nutrition disorders Hypoglycemia	4.5% 1/22 • Number of events 1 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.

Other adverse events

Other adverse events
Measure	Open Label-Single Arm n=22 participants at risk This is a single arm, open-label study of SGI-110 in patients with MPN. SGI-110 will be administered subcutaneously at a dose of 60 mg/m2 on days 1-5, repeated every 28 days. Toxicity will be evaluated using the NCI Common Terminology Criteria for Adverse Events Active Version 4. The frequency of toxicities per organ system will be tabulated using descriptive statistics. All patients who receive any amount of the study drug will be evaluable for toxicity SGI-110: subcutaneously at a dose of 60 mg/m2
Gastrointestinal disorders Abdominal cramping	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Abdominal discomfort	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Abdominal pain	18.2% 4/22 • Number of events 6 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Anorexia	13.6% 3/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Appetite change	27.3% 6/22 • Number of events 13 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Back pain	18.2% 4/22 • Number of events 4 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Edema	22.7% 5/22 • Number of events 16 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Musculoskeletal and connective tissue disorders Bone/joint pain	59.1% 13/22 • Number of events 14 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Bruising/bleeds easily	22.7% 5/22 • Number of events 11 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Musculoskeletal and connective tissue disorders Calf cramps/pain	13.6% 3/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Mouth sores	18.2% 4/22 • Number of events 7 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Chills/rigors	27.3% 6/22 • Number of events 8 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Respiratory, thoracic and mediastinal disorders Congestion	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Constipation	40.9% 9/22 • Number of events 11 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Respiratory, thoracic and mediastinal disorders Cough	45.5% 10/22 • Number of events 11 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Diaphoresis	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Diarrhea	36.4% 8/22 • Number of events 13 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Diffuse achiness	9.1% 2/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Nervous system disorders Dizziness	40.9% 9/22 • Number of events 23 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Dry mouth	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Reproductive system and breast disorders Dyspnea	45.5% 10/22 • Number of events 19 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Renal and urinary disorders Dysuria	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Respiratory, thoracic and mediastinal disorders Epistaxis	27.3% 6/22 • Number of events 8 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fall	9.1% 2/22 • Number of events 4 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fatigue	68.2% 15/22 • Number of events 34 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Fecal urgency	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Fever	18.2% 4/22 • Number of events 5 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Headache	27.3% 6/22 • Number of events 7 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Hemangioma	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Hemorrhagic bullae	9.1% 2/22 • Number of events 5 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Metabolism and nutrition disorders Hypocalcemia	9.1% 2/22 • Number of events 6 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Itching	18.2% 4/22 • Number of events 4 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Musculoskeletal and connective tissue disorders Muscle cramps	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Gastrointestinal disorders Nausea	36.4% 8/22 • Number of events 19 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Night sweats	22.7% 5/22 • Number of events 5 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations Pneumonia	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Musculoskeletal and connective tissue disorders Arthralgia	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Pallor	22.7% 5/22 • Number of events 8 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Splenomegaly	13.6% 3/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Cardiac disorders Palpitations	9.1% 2/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Petechiae	18.2% 4/22 • Number of events 4 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Progression to AML	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Skin and subcutaneous tissue disorders Rash	18.2% 4/22 • Number of events 4 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Shoulder pain	13.6% 3/22 • Number of events 5 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Sore throat	13.6% 3/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Infections and infestations Upper respiratory infection	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Weakness	27.3% 6/22 • Number of events 6 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
General disorders Weight loss	13.6% 3/22 • Number of events 3 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Respiratory, thoracic and mediastinal disorders Wheezes	9.1% 2/22 • Number of events 2 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.
Blood and lymphatic system disorders Anemia	77.3% 17/22 • Number of events 47 • All adverse events (AE's), and deaths recorded from initiation of investigational product (IP), until 30 days of the last study intervention, should be followed to their resolution, or until the investigator assesses them as stable, determines the event to be irreversible, or the participant is lost to follow-up. AE's will be recorded for each patient, from the first day of treatment with IP until 30 days after the last dose of IP. Approximately up to 1840 days.

Additional Information

Dr.Pinkal Desai

Weill Cornell Medical College

Phone: 646-962-2700

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place