Trial Outcomes & Findings for Dasatinib In Waldenström Macroglobulinemia (NCT NCT04115059)

NCT ID: NCT04115059

Last Updated: 2024-03-21

Results Overview

Count of participants who experience a treatment-emergent adverse event

• Recruitment status: TERMINATED
• Study phase: PHASE1
• Target enrollment: 3 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2024-03-21

Participant Flow

Participant milestones

Measure	Dasatinib \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure	Dasatinib \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle
Overall Study Withdrawal by Subject	1
Overall Study Lack of Efficacy	2

Baseline Characteristics

Dasatinib In Waldenström Macroglobulinemia

Baseline characteristics by cohort

Measure	Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	73 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	1 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	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Count of participants who experience a treatment-emergent adverse event

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Number of Participants With at Least One Treatment-Emergent Adverse Event

3 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with an Overall Response. Overall Response Rate= Minor response (>25%-50% reduction in serum IgM from baseline) + Partial Response (>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Overall Response Rate

0 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with Complete Response (CR). Complete Response requires resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, and resolution of any adenopathy or splenomegaly.

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Complete Response Rate

0 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with very good partial response (VGPR) to therapy. (VGPR is >90% reduction in serum IgM from baseline)

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Very Good Partial Response Rate

0 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with partial response (PR) to therapy. (PR is 50-89% reduction in serum IgM from baseline)

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Partial Response Rate

0 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with Minor Responses to therapy. (MR is 25-49% reduction in serum IgM from baseline)

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Minimal Response Rate

0 Participants

SECONDARY outcome

Timeframe: 2 years

Percentage of patients with Stable disease to therapy. (SD is <25% reduction in serum IgM from baseline).

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Stable Disease Rate

3 Participants

SECONDARY outcome

Timeframe: From first dose through disease progression, up to 2 years from baseline

Percentage of patients with who experienced disease progression on study. PD is >25% increase in serum IgM from baseline with an absolute increase of at least 500 mg/dL, or progression of clinically significant disease related symptoms. Death from any cause or initiation of a new anti-neoplastic therapy will also be considered a progression event. An increase of 1 cm in any axis for adenopathy, or 2 cm in the craniocaudal axis of the spleen will be considered evidence of progression of extramedullary disease. Development of Bing Neel syndrome, or other extramedullary disease manifestations, as well as disease transformation will be considered as progressive events.

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Progressive Disease Rate

2 Participants

SECONDARY outcome

Timeframe: From first dose through disease progression, up to 2 years from baseline

The amount of time between starting treatment and experiencing disease progression. PD is >25% increase in serum IgM from baseline with an absolute increase of at least 500 mg/dL, or progression of clinically significant disease related symptoms. Death from any cause or initiation of a new anti-neoplastic therapy will also be considered a progression event. An increase of 1 cm in any axis for adenopathy, or 2 cm in the craniocaudal axis of the spleen will be considered evidence of progression of extramedullary disease. Development of Bing Neel syndrome, or other extramedullary disease manifestations, as well as disease transformation will be considered as progressive events.

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Progression Free Survival

5 months Interval 0.0 to 5.0

SECONDARY outcome

Timeframe: From first dose through initiation of new therapy, up to 2 years from baseline

The amount of time between starting study treatment and initiating a new therapy

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Time to Next Therapy (TTNT)

6 months Interval 1.0 to 6.0

SECONDARY outcome

Timeframe: From first dose through death, up to 2 years from baseline

The number of participants who are still alive at the end of follow-up. Participants were observed for up to 2 years after discontinuing study therapy for survival status.

Outcome measures

Measure

Dasatinib n=3 Participants \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle

Overall Survival

2 Participants

Adverse Events

Dasatinib

Serious events: 2 serious events

Other events: 3 other events

Deaths: 1 deaths

Serious adverse events

Measure	Dasatinib n=3 participants at risk \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle
Respiratory, thoracic and mediastinal disorders Metapneumovirus	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Blood and lymphatic system disorders Febrile neutropenia	33.3% 1/3 • Number of events 3 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Cardiac disorders Arrhythmia	33.3% 1/3 • Number of events 3 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.

Other adverse events

Measure	Dasatinib n=3 participants at risk \-- After the screening procedures confirm participation in the research study: The participant will be given a study drug-dosing calendar for each treatment cycle. Dasatinib: Oral Study Drug(s): * Each study treatment cycle lasts 4 weeks during which time you will be taking the study drug one time per day. * This will continue for up to 24 cycles. Dasatinib: Oral, daily, dosing per protocol, once a day for cycle
Blood and lymphatic system disorders Anemia	66.7% 2/3 • Number of events 2 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Cardiac disorders Congestive heart failure	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Cardiac disorders Heart failure	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Gastrointestinal disorders Abdominal distention	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Gastrointestinal disorders Constipation	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Gastrointestinal disorders Mouth sores	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Edema	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Fatigue	66.7% 2/3 • Number of events 2 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Fever	66.7% 2/3 • Number of events 2 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Fluid overload	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Leg edema	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
General disorders Withdrawal symptoms	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Investigations Neutropenia	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Investigations Thrombocytopenia	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Musculoskeletal and connective tissue disorders Body Aches	66.7% 2/3 • Number of events 2 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Musculoskeletal and connective tissue disorders Bone pain	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Nervous system disorders Dizziness	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Nervous system disorders Migraines	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Psychiatric disorders Insomnia	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Respiratory, thoracic and mediastinal disorders Dyspnea	66.7% 2/3 • Number of events 2 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Respiratory, thoracic and mediastinal disorders Post nasal drip	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.
Respiratory, thoracic and mediastinal disorders Sinus congestion	33.3% 1/3 • Number of events 1 • Adverse events were collected after dasatinib initiation, through 30 days after the last dose of dasatinib (e.g. 6 months). Participants were observed for overall survival for up to 2 years, or until withdrawal of consent or death.

Additional Information

Jorge Castillo

Dana-Farber Cancer Institute

Phone: 617-632-2681

Email: jorgej_castillo@dfci.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place