Trial Outcomes & Findings for Pomalidomide, Dexamethasone and Rituximab in Waldenstrom's Macroglobulinemia (NCT NCT01078974)

Last Updated: 2016-07-25

Results Overview

To determine the MTD of pomalidomide administered orally in patients with Waldenstrom's Macroglobulinemia in combination with dexamethasone and rituximab. Because maximum tolerated dose was not determined due to study termination, the highest dose of pomalidomide administered is presented below.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

7 participants

Primary outcome timeframe

2 years

Results posted on

2016-07-25

Participant Flow

A total of 7 participants were recruited at the DFCI medical clinic between 9/22/2010 and 4/3/2012.

Participant milestones

Participant milestones
Measure	Pomalidomide, Dexamethasone, Rituximab Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Overall Study STARTED	7
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Pomalidomide, Dexamethasone, Rituximab Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Overall Study Adverse Event	3

Baseline Characteristics

Pomalidomide, Dexamethasone and Rituximab in Waldenstrom's Macroglobulinemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pomalidomide, Dexamethasone, Rituximab n=7 Participants Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	64 years STANDARD_DEVIATION 7.27 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	7 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	7 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	7 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: The maximum tolerated dose was not determined due to study termination. Three participants experienced IgM flare causing them to be removed from the study early.

Outcome measures

Outcome measures
Measure	Pomalidomide, Dexamethasone, Rituximab n=7 Participants Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Maximum Tolerated Dose of Pomalidomide	1 mg

PRIMARY outcome

Timeframe: 2 years

Number of participants with dose limiting toxicities which resulted in being removed from pomalidomide therapy

Outcome measures

Outcome measures
Measure	Pomalidomide, Dexamethasone, Rituximab n=7 Participants Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Tolerability of Pomalidomide	3 participants

Adverse Events

Pomalidomide, Dexamethasone, Rituximab

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Pomalidomide, Dexamethasone, Rituximab n=7 participants at risk Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Blood and lymphatic system disorders IgM flare	42.9% 3/7 • Number of events 3 • Adverse events were collected over a 3 year period.

Other adverse events

Other adverse events
Measure	Pomalidomide, Dexamethasone, Rituximab n=7 participants at risk Drug: pomalidomide Taken orally once a day Drug: dexamethasone Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 Drug: rituximab Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 pomalidomide: Taken orally once a day dexamethasone: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15 rituximab: Given intravenously on weeks 1, 2, 3 and 4 and weeks 12, 13, 14 and 15
Blood and lymphatic system disorders Anemia	28.6% 2/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Alanine aminotransferase increased	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Anorexia	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Musculoskeletal and connective tissue disorders Arthralgia	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Aspartate aminotransferase increased	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
General disorders Chills	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Eye disorders Conjunctivitis	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
General disorders Cough	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Creatinine increased	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Gastrointestinal disorders Diarrhea	28.6% 2/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Nervous system disorders Dizziness	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Gastrointestinal disorders Dyspepsia	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
General disorders Dyspnea	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
General disorders Fatigue	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Nervous system disorders Headache	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Hyperglycemia	57.1% 4/7 • Number of events 5 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders Hypocalcemia	14.3% 1/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Injury, poisoning and procedural complications Infusion related reaction	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Metabolism and nutrition disorders INR increased	14.3% 1/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Infections and infestations Lung infection	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Musculoskeletal and connective tissue disorders Myalgia	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Gastrointestinal disorders Nausea	28.6% 2/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Musculoskeletal and connective tissue disorders Neck pain	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Blood and lymphatic system disorders Neutrophil count decreased	28.6% 2/7 • Number of events 3 • Adverse events were collected over a 3 year period.
Blood and lymphatic system disorders Platelet count decreased	28.6% 2/7 • Number of events 3 • Adverse events were collected over a 3 year period.
Nervous system disorders Seizure	14.3% 1/7 • Number of events 2 • Adverse events were collected over a 3 year period.
Infections and infestations Tooth Infection	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
Nervous system disorders Tremor	28.6% 2/7 • Number of events 2 • Adverse events were collected over a 3 year period.
General disorders Urinary frequency	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.
General disorders Vomiting	14.3% 1/7 • Number of events 1 • Adverse events were collected over a 3 year period.

Additional Information

Steven P. Treon

Dana-Farber Cancer Institute

Phone: 617-632-2681

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place