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Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis

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ClinicalTrials.gov Identifier: NCT02590588
Recruitment Status : Terminated (Poor accrual)
First Posted : October 29, 2015
Results First Posted : May 10, 2017
Last Update Posted : September 21, 2017
Sponsor:
Collaborator:
Gilead Sciences
Information provided by (Responsible Party):
John Mark Sloan, Boston Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Amyloidosis
Intervention Drug: Idelalisib
Enrollment 1
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Idelalisib
Hide Arm/Group Description

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Period Title: Overall Study
Started 1
Completed 0
Not Completed 1
Reason Not Completed
Adverse Event             1
Arm/Group Title Idelalisib
Hide Arm/Group Description

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Baseline Participants 1
Overall Number of Units Analyzed
Type of Units Analyzed: Only patient enrolled
1
Hide Baseline Analysis Population Description
'There was only one patient enrolled
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
<=18 years
0
   0.0%
Between 18 and 65 years
1
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Female
0
   0.0%
Male
1
 100.0%
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
1
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
[1]
Measure Description: Unit of measure based on patient identification
1.Primary Outcome
Title Overall Response
Hide Description Evaluate hematologic response according to standard criteria
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
Number of participants with hematologic response is zero. There was only one patient enrolled and he did not remain on study long enough for his first 3 month response evaluation.
Arm/Group Title Idelalisib
Hide Arm/Group Description:

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Secondary Outcome
Title Progression Free Survival
Hide Description Evaluate time to progression
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
The evaluation of progression-free survival requires that a patient responds, and then progresses. There was only one patient enrolled and he did not remain on study long enough for his first 3 month response evaluation.
Arm/Group Title Idelalisib
Hide Arm/Group Description:

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Organ Response
Hide Description Number of patients with organ response using standard AL amyloidosis criteria.
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
Number of patients with organ response using standard AL amyloidosis criteria.
Arm/Group Title Idelalisib
Hide Arm/Group Description:

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Evaluate Safety and Tolerability of Agent
Hide Description Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
There was only one patient enrolled and he did experience treatment-related adverse events.
Arm/Group Title Idelalisib
Hide Arm/Group Description:

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Participants Analyzed 1
Measure Type: Count of Participants
Unit of Measure: Participants
1
 100.0%
5.Secondary Outcome
Title Quality of Life
Hide Description Evaluate quality of life according to Functional Assessment of Cancer Therapy Lymphoma Subscale (FACT-Lym) assessment tool
Time Frame 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
'There was only one patient enrolled and he did not remain on study long enough for his first protocol-specified quality of life assessment
Arm/Group Title Idelalisib
Hide Arm/Group Description:

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 2 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Idelalisib
Hide Arm/Group Description

Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion.

Idelalisib: Idelalisib daily until unacceptable toxicity or disease progression.

All-Cause Mortality
Idelalisib
Affected / at Risk (%)
Total   0/1 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Idelalisib
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Investigations   
increased  1 [1]  1/1 (100.00%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
[1]
alanine-aminotransferase
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Idelalisib
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Eye disorders   
swollen eyelids  1  1/1 (100.00%)  1
Gastrointestinal disorders   
nausea  1  1/1 (100.00%)  1
diarrhea  1  1/1 (100.00%)  1
vomiting  1  1/1 (100.00%)  1
diminished appetite  1  1/1 (100.00%)  1
Investigations   
discolored urine  1  1/1 (100.00%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Early termination leading to small numbers of subjects analyzed.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Mark Sloan MD
Organization: Boston Medical Center
Phone: 617-638-2367
Responsible Party: John Mark Sloan, Boston Medical Center
ClinicalTrials.gov Identifier: NCT02590588     History of Changes
Other Study ID Numbers: H-34318
First Submitted: September 9, 2015
First Posted: October 29, 2015
Results First Submitted: March 30, 2017
Results First Posted: May 10, 2017
Last Update Posted: September 21, 2017