Trial Outcomes & Findings for AUY922 for Advanced ALK-positive NSCLC (NCT NCT01752400)

Last Updated: 2018-03-29

Results Overview

The number of participants that achieved either a complete response (CR) or a partial response (PR) as assessed by Response Evaluation Criteria in Solid Tumors (RECIST v1.1). * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \< 10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

Baseline and then every six weeks (± 7 days), until the time of disease progression

Results posted on

2018-03-29

Participant Flow

Participant milestones

Participant milestones
Measure	AUY922 Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes AUY922
Overall Study STARTED	6
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

AUY922 for Advanced ALK-positive NSCLC

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes AUY922
Age, Continuous	52.5 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	6 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants
Smoking Status Never Smoker	2 Participants n=5 Participants
Smoking Status Light Smoker (≤ 10 pack years)	2 Participants n=5 Participants
Smoking Status Heavy Smoker (>10 pack years)	2 Participants n=5 Participants
Median Number of Prior Lines of Therapy	3 Prior Lines of Therapy n=5 Participants
Previous ALK Inhibitors Crizotinib	5 participants n=5 Participants
Previous ALK Inhibitors Alectinib	1 participants n=5 Participants
Previous ALK Inhibitors Certinib	2 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and then every six weeks (± 7 days), until the time of disease progression

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Objective Response Rate	0 Participants

SECONDARY outcome

Timeframe: From the start of treatment until the time of death or progression

Progression free survival is measured as the number of months from date of study entry to date of progression or death, whichever comes first. Progression is assessed using RECIST v1.1. Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study with at least a 5 mm absolute increase in the sum of all lesions. The appearance of one or more new lesions denotes disease progression.

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Progression-free Survival	1.43 Months Interval 1.3 to 2.8

SECONDARY outcome

Timeframe: Baseline and then every six weeks (± 7 days), until the time of disease progression

The number of participants that achieved disease control, which includes complete responses, partial responses or stable disease as assessed by RECIST v1.1 * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \< 10 mm. * Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Disease Control Rate CR	0 Participants
Disease Control Rate PR	3 Participants
Disease Control Rate SD	3 Participants

SECONDARY outcome

Timeframe: From the start of treatment until 30 days after last dose was received

The number of participants that developed any grade adverse event as assessed by Common Terminology Criteria for Adverse Events (CTCAE v4)

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Number of Participants Who Develop Adverse Events on AUY922	6 Participants

SECONDARY outcome

Timeframe: Baseline

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Number of Participants With Concurrent KRAS Mutations Unknown Mutation Status	4 Participants
Number of Participants With Concurrent KRAS Mutations KRAS Mutation	0 Participants
Number of Participants With Concurrent KRAS Mutations Negative for KRAS Mutation	2 Participants

SECONDARY outcome

Timeframe: Baseline

Population: Data unavailable for all six participants

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, end of treatment

Population: Data not available for the other 5 participants

The number of secondary ALK mutations or ALK amplification as a mechanism of resistance in pre-treatment and post-treatment biopsies.

Outcome measures

Outcome measures
Measure	AUY922 n=1 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
ALK Mutation Status ALK Mutation	1 Participants
ALK Mutation Status No ALK Mutation	0 Participants

SECONDARY outcome

Timeframe: From the start of treatment until death or withdrawal from the study

The median duration of time from the start of treatment until the time of death or until the participant withdraws participation in the trial.

Outcome measures

Outcome measures
Measure	AUY922 n=6 Participants Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Median Overall Survival	26.87 Months Interval 9.03 to 26.87

Adverse Events

AUY922

Serious events: 2 serious events

Other events: 6 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	AUY922 n=6 participants at risk Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Gastrointestinal disorders Abdominal Pain	16.7% 1/6 • Number of events 2 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, progressive disease	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.

Other adverse events

Other adverse events
Measure	AUY922 n=6 participants at risk Via intravenous infusion on Days 1, 8 and 15 of each 21 day cycle (once per week). Infusion lasts approximately 60 minutes
Gastrointestinal disorders Abdominal pain	33.3% 2/6 • Number of events 3 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Investigations Alanine aminotransferase increased	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Investigations Alkaline phosphatase increased	33.3% 2/6 • Number of events 2 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Metabolism and nutrition disorders Alkalosis	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Blood and lymphatic system disorders Anemia	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Metabolism and nutrition disorders Anorexia	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Psychiatric disorders Anxiety	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Investigations Aspartate aminotransferase increased	16.7% 1/6 • Number of events 2 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Gastrointestinal disorders Constipation	33.3% 2/6 • Number of events 2 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Gastrointestinal disorders Diarrhea	66.7% 4/6 • Number of events 5 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Skin and subcutaneous tissue disorders Dry skin	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Eye disorders Eye disorders - Other, specify	66.7% 4/6 • Number of events 5 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
General disorders Fatigue	50.0% 3/6 • Number of events 3 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Metabolism and nutrition disorders Hypomagnesemia	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Metabolism and nutrition disorders Hyponatremia	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Infections and infestations Infections and infestations - Other, specify	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Investigations INR increased	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Investigations Investigations - Other, specify	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Infections and infestations Lung infection	16.7% 1/6 • Number of events 2 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, specify	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Musculoskeletal and connective tissue disorders Myalgia	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Gastrointestinal disorders Nausea	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.
Blood and lymphatic system disorders Thrombotic thrombocytopenic purpura	16.7% 1/6 • Number of events 1 • From the start of treatment until 30 days after the last dose of the study drug was received. Treatment is continued until disease progression, unacceptable toxicity, participant withdrawal, death, or discontinuation from the study for any other reason.

Additional Information

Alice Shaw, MD, PhD

Massachusetts General Hospital

Phone: 617-724-4000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place