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Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia

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ClinicalTrials.gov Identifier: NCT02439138
Recruitment Status : Terminated (Safety issues from trials in CLL)
First Posted : May 8, 2015
Results First Posted : January 11, 2017
Last Update Posted : January 11, 2017
Sponsor:
Collaborator:
Gilead Sciences
Information provided by (Responsible Party):
Jorge J. Castillo, MD, Dana-Farber Cancer Institute

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Waldenstrom's Macroglobulinemia
Intervention Drug: GS-1101
Enrollment 5
Recruitment Details  
Pre-assignment Details  
Arm/Group Title GS-1101
Hide Arm/Group Description

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

Period Title: Overall Study
Started 5
Completed 2
Not Completed 3
Reason Not Completed
Adverse Event             1
PI decision to terminate the study             2
Arm/Group Title GS-1101
Hide Arm/Group Description

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 Number of Baseline Participants 5
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
<=18 years
0
   0.0%
Between 18 and 65 years
2
  40.0%
>=65 years
3
  60.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 5 participants
66
(57 to 80)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
1
  20.0%
Male
4
  80.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 5 participants
5
1.Primary Outcome
Title Overall Response Rate (ORR)
Hide Description ORR measured by decrease in serum IgM level by at least 25% from baseline.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
4 of 5 participants returned for at least 1 follow-up to assess disease response.
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with response
0
2.Secondary Outcome
Title Percentage of Participants With Adverse Events
Hide Description Assess the safety and tolerability of idelalisib
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: percentage of participants with AEs
100
3.Secondary Outcome
Title Rate of Complete Response (CR)
Hide Description 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.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with CR
0
4.Secondary Outcome
Title Rate of Very Good Partial Response (VGPR)
Hide Description VGPR measured by decrease in serum IgM levels of at least 90% from baseline.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with VGPR
0
5.Secondary Outcome
Title Rate of Partial Response (PR)
Hide Description PR measured by decrease in serum IgM levels of between 25% and 50% from baseline.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with PR
0
6.Secondary Outcome
Title Rate of Minimal Response
Hide Description Minimal response measured by decrease in serum IgM levels of between 25% and 50%.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with MR
0
7.Secondary Outcome
Title Rate of Stable Disease
Hide Description Stable disease measured by serum IgM levels <25% reduced from baseline.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: percentage of participants with SD
100
8.Secondary Outcome
Title Rate of Progressive Disease
Hide Description 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.
Time Frame Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title GS-1101
Hide Arm/Group Description:

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 Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: percentage of participants with PD
20
Time Frame Adverse events were collected from the time of screening until 30 days post treatment discontinuation, up to 16 weeks (median 4 weeks).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GS-1101
Hide Arm/Group Description

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

All-Cause Mortality
GS-1101
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
GS-1101
Affected / at Risk (%) # Events
Total   4/5 (80.00%)    
Blood and lymphatic system disorders   
INR elevation * 1  1/5 (20.00%)  1
Febrile Neutropenia * 1  1/5 (20.00%)  1
Investigations   
ALT elevation * 1  3/5 (60.00%)  3
Metabolism and nutrition disorders   
Hyponatremia * 1  1/5 (20.00%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Death due to disease progression * 1  1/5 (20.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
GS-1101
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Gastrointestinal disorders   
Flatulence * 1  1/5 (20.00%)  1
Mouth sores * 1  1/5 (20.00%)  1
General disorders   
Sweats * 1  1/5 (20.00%)  1
Shaking chills * 1  1/5 (20.00%)  1
Painful gums * 1  1/5 (20.00%)  1
Investigations   
Thrush * 1  1/5 (20.00%)  1
AST elevation * 1  3/5 (60.00%)  3
Musculoskeletal and connective tissue disorders   
Joint pain * 1  1/5 (20.00%)  1
Nervous system disorders   
Headache * 1  1/5 (20.00%)  1
Respiratory, thoracic and mediastinal disorders   
Dyspnea * 1  1/5 (20.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
This study was terminated early due to safety concerns from the previously untreated CLL experience. All participants who remained on study at that time were permanently removed from protocol therapy. The median time on protocol therapy was 1 cycle
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Jorge J. Castillo
Organization: Dana-Farber Cancer Institute
Phone: 617-632-6045
Responsible Party: Jorge J. Castillo, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT02439138     History of Changes
Other Study ID Numbers: 15-040
ISR IN-US-313-1609 ( Other Grant/Funding Number: Gilead Sciences, Inc. )
First Submitted: April 29, 2015
First Posted: May 8, 2015
Results First Submitted: September 26, 2016
Results First Posted: January 11, 2017
Last Update Posted: January 11, 2017