Trial Outcomes & Findings for Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia (NCT NCT02439138)
NCT ID: NCT02439138
Last Updated: 2017-01-11
Results Overview
ORR measured by decrease in serum IgM level by at least 25% from baseline.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Results posted on
2017-01-11
Participant Flow
Participant milestones
| Measure |
GS-1101
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Overall Study
STARTED
|
5
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
GS-1101
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
PI decision to terminate the study
|
2
|
Baseline Characteristics
Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
Baseline characteristics by cohort
| Measure |
GS-1101
n=5 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
|
Age, Continuous
|
66 years
n=5 Participants
|
|
Gender
Female
|
1 Participants
n=5 Participants
|
|
Gender
Male
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.Population: 4 of 5 participants returned for at least 1 follow-up to assess disease response.
ORR measured by decrease in serum IgM level by at least 25% from baseline.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Overall Response Rate (ORR)
|
0 percentage of participants with response
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.Assess the safety and tolerability of idelalisib
Outcome measures
| Measure |
GS-1101
n=5 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Percentage of Participants With Adverse Events
|
100 percentage of participants with AEs
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.CR measured by decrease in serum IgM levels to normal range, disappearnace of monoclonal protein by immunofixation, no evidence of bone marrow involvement, and resolution of any extramedullary disease by CT scan.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Complete Response (CR)
|
0 percentage of participants with CR
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.VGPR measured by decrease in serum IgM levels of at least 90% from baseline.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Very Good Partial Response (VGPR)
|
0 percentage of participants with VGPR
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.PR measured by decrease in serum IgM levels of between 25% and 50% from baseline.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Partial Response (PR)
|
0 percentage of participants with PR
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.Minimal response measured by decrease in serum IgM levels of between 25% and 50%.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Minimal Response
|
0 percentage of participants with MR
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.Stable disease measured by serum IgM levels \<25% reduced from baseline.
Outcome measures
| Measure |
GS-1101
n=4 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Stable Disease
|
100 percentage of participants with SD
|
SECONDARY outcome
Timeframe: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.Progressive disease measured by an 25% increase in serum IgM level with an absolute increase of at least 500mg/dL from the lowest attained IgM on therapy.
Outcome measures
| Measure |
GS-1101
n=5 Participants
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Rate of Progressive Disease
|
20 percentage of participants with PD
|
Adverse Events
GS-1101
Serious events: 4 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GS-1101
n=5 participants at risk
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Blood and lymphatic system disorders
INR elevation
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Investigations
ALT elevation
|
60.0%
3/5 • Number of events 3 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Metabolism and nutrition disorders
Hyponatremia
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Death due to disease progression
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
Other adverse events
| Measure |
GS-1101
n=5 participants at risk
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
\-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
GS-1101
|
|---|---|
|
Nervous system disorders
Headache
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Gastrointestinal disorders
Flatulence
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Investigations
Thrush
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
General disorders
Sweats
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
General disorders
Shaking chills
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
General disorders
Painful gums
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Investigations
AST elevation
|
60.0%
3/5 • Number of events 3 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
|
Gastrointestinal disorders
Mouth sores
|
20.0%
1/5 • Number of events 1 • Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place