Trial Outcomes & Findings for Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia (NCT NCT00976248)

Results Overview

Overall Response = Complete Response + Near Complete Response + Very Good Partial Response + Partial Response + Minor Response Complete Response: resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. A near CR (nCR) is defined as fulfilling all CR criteria in the presence of positive immunofixation test for an IgM paraprotein. Very Good Partial Response: \> 90% reduction in serum IgM levels. Partial Response: \> 50% reduction in serum IgM levels. Minor Response: 25-49% reduction in serum IgM levels Progressive Disease: greater than 25% increase in serum IgM level occurs from the lowest attained response value or progression of clinically significant disease related symptom(s). Stable Disease: \< 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

33 participants

Primary outcome timeframe

End of Treatment, an average of 16 months

Results posted on

2018-10-03

Participant Flow

Participant milestones

Participant milestones
Measure	RAD001 RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Overall Study STARTED	33
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RAD001 n=33 Participants RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	21 Participants n=5 Participants
Age, Categorical >=65 years	12 Participants n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants
Region of Enrollment United States	33 participants n=5 Participants

PRIMARY outcome

Timeframe: End of Treatment, an average of 16 months

Overall Response = Complete Response + Near Complete Response + Very Good Partial Response + Partial Response + Minor Response Complete Response: resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. A near CR (nCR) is defined as fulfilling all CR criteria in the presence of positive immunofixation test for an IgM paraprotein. Very Good Partial Response: \> 90% reduction in serum IgM levels. Partial Response: \> 50% reduction in serum IgM levels. Minor Response: 25-49% reduction in serum IgM levels Progressive Disease: greater than 25% increase in serum IgM level occurs from the lowest attained response value or progression of clinically significant disease related symptom(s). Stable Disease: \< 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM

Outcome measures

Outcome measures
Measure	RAD001 n=33 Participants RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Overall Response Rate of RAD001 in Patients With Previously Untreated WM	22 participants

PRIMARY outcome

Timeframe: End of Treatment, an average of 16 months

Population: 13 participants were excluded from this analysis due to treatment and follow-up ending prior to progression.

Progression is defined as a 25% increase in serum IgM from the lowest attained response value or progression of clinically significant disease related symptoms.

Outcome measures

Outcome measures
Measure	RAD001 n=20 Participants RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Time to Progression With Single Agent RAD001 Therapy in Previously Untreated WM.	21 Months Interval 1.0 to 64.0

PRIMARY outcome

Timeframe: End of follow-up, an average of 18 months

Population: 13 participants were censored due to follow-up ending prior to new therapy initiation.

Outcome measures

Outcome measures
Measure	RAD001 n=20 Participants RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Time to Next Therapy With Single Agent RAD001 Therapy in Previously Untreated WM	15 Months Interval 1.0 to 69.0

Adverse Events

RAD001

Serious events: 7 serious events

Other events: 33 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	RAD001 n=33 participants at risk RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Musculoskeletal and connective tissue disorders Pain	3.0% 1/33 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonitis	6.1% 2/33 • Number of events 2
Respiratory, thoracic and mediastinal disorders Pneumonia	3.0% 1/33 • Number of events 1
Blood and lymphatic system disorders Hyperviscosity syndrome	3.0% 1/33 • Number of events 1
Musculoskeletal and connective tissue disorders Fracture	3.0% 1/33 • Number of events 1
Gastrointestinal disorders Swollen tongue	3.0% 1/33 • Number of events 1

Other adverse events

Other adverse events
Measure	RAD001 n=33 participants at risk RAD001, oral, 10 mg, daily RAD001 : Taken orally once a day
Blood and lymphatic system disorders Anemia	24.2% 8/33 • Number of events 8
Blood and lymphatic system disorders Thrombocytopenia	15.2% 5/33 • Number of events 5
Blood and lymphatic system disorders Neutropenia	15.2% 5/33 • Number of events 5
Metabolism and nutrition disorders Hyperglycemia	6.1% 2/33 • Number of events 2
Gastrointestinal disorders Oral ulcerations	21.2% 7/33 • Number of events 7
Respiratory, thoracic and mediastinal disorders Pneumonitis	15.2% 5/33 • Number of events 5
General disorders Fatigue	12.1% 4/33 • Number of events 4
Skin and subcutaneous tissue disorders Rash	6.1% 2/33 • Number of events 2
Infections and infestations Cellulitis	6.1% 2/33 • Number of events 2

Additional Information

Steven P. Treon, MD, PhD

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