Trial Outcomes & Findings for Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis (NCT NCT02590588)

NCT ID: NCT02590588

Last Updated: 2017-09-21

Results Overview

Evaluate hematologic response according to standard criteria

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

3 months

Results posted on

2017-09-21

Participant Flow

Participant milestones

Participant milestones
Measure	Idelalisib 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 Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Idelalisib 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 Study Adverse Event	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Idelalisib n=1 only patient enrolled 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.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	0 Participants n=93 Participants
Sex: Female, Male Male	1 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	1 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants

PRIMARY outcome

Timeframe: 3 months

Population: 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.

Evaluate hematologic response according to standard criteria

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: 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.

Evaluate time to progression

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Population: Number of patients with organ response using standard AL amyloidosis criteria.

Number of patients with organ response using standard AL amyloidosis criteria.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Population: There was only one patient enrolled and he did experience treatment-related adverse events.

Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0

Outcome measures

Outcome measures
Measure	Idelalisib n=1 Participants 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.
Evaluate Safety and Tolerability of Agent	1 Participants

SECONDARY outcome

Timeframe: 3 months

Population: 'There was only one patient enrolled and he did not remain on study long enough for his first protocol-specified quality of life assessment

Evaluate quality of life according to Functional Assessment of Cancer Therapy Lymphoma Subscale (FACT-Lym) assessment tool

Outcome measures

Outcome data not reported

Adverse Events

Idelalisib

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Idelalisib n=1 participants at risk 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.
Investigations increased	100.0% 1/1 • Number of events 1 • 2 months

Other adverse events

Other adverse events
Measure	Idelalisib n=1 participants at risk 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.
Gastrointestinal disorders nausea	100.0% 1/1 • Number of events 1 • 2 months
Gastrointestinal disorders diarrhea	100.0% 1/1 • Number of events 1 • 2 months
Gastrointestinal disorders vomiting	100.0% 1/1 • Number of events 1 • 2 months
Gastrointestinal disorders diminished appetite	100.0% 1/1 • Number of events 1 • 2 months
Eye disorders swollen eyelids	100.0% 1/1 • Number of events 1 • 2 months
Investigations discolored urine	100.0% 1/1 • Number of events 1 • 2 months

Additional Information

Mark Sloan MD

Boston Medical Center

Phone: 617-638-2367

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place