Trial Outcomes & Findings for IPI-504 in NSCLC Patients With ALK Translocations (NCT NCT01228435)

Results Overview

The response rate was defined as the number of patients achieving a RECIST 1.0 defined response divided by the number of patients treated and was to be calculated separately for each arm. A response by RECIST criteria means that the pre-defined target lesions (sum of the longest diameters) had to decrease by 30% or more and this response needed to be confirmed on a second scan at least 4 weeks later.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

2 years

Results posted on

2017-12-04

Participant Flow

Patients were recruited from the oncology clinic at Mass General from 6/10/10 to 9/26/11.

The study was closed to accrual after 3 patients were enrolled over a 1-year period. It was determined that there were too many competing protocols and completion of the study as designed was not feasible.

Participant milestones

Participant milestones
Measure	ALK-inhibitor Naive Patients in this arm has no prior exposure to ALK-inhibitor and were treated with IPI-504 at 225 mg/m2 twice a week for 2 weeks followed by 10 days off therapy, cycles repeated every 21 days.	ALK-inhibitor Pre-treated Patients in this arm had prior exposure to an ALK inhibitor and were treated with IPI-504 at 225 mg/m2 twice a week for 2 weeks followed by 10 days off therapy, cycles repeated every 21 days.
Overall Study STARTED	1	2
Overall Study COMPLETED	1	2
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

IPI-504 in NSCLC Patients With ALK Translocations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ALK-inhibitor Naive n=1 Participants No prior exposure to ALK-inhibitor	ALK-inhibitor Pre-treated n=2 Participants Prior exposure to ALK inhibitor	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Continuous	79 years n=5 Participants	67.5 years STANDARD_DEVIATION 14.8 • n=7 Participants	71.3 years STANDARD_DEVIATION 12.4 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

The response rate was defined as the number of patients achieving a RECIST 1.0 defined response divided by the number of patients treated and was to be calculated separately for each arm. A response by RECIST criteria means that the pre-defined target lesions (sum of the longest diameters) had to decrease by 30% or more and this response needed to be confirmed on a second scan at least 4 weeks later.

Outcome measures

Outcome measures
Measure	ALK-inhibitor Naive n=1 Participants Patients in this arm has no prior exposure to ALK-inhibitor and were treated with IPI-504 at 225mg/m2 twice a week for 2 weeks followed by 10 days off with cycles repeating every 21 days.	ALK-inhibitor Pre-treated n=2 Participants Patients in this arm had received prior exposure to ALK-inhibitor and were treated with IPI-504 at 225mg/m2 twice a week for 2 weeks followed by 10 days off with cycles repeating every 21 days.
Response Rate	0 participants	0 participants

SECONDARY outcome

Timeframe: 2 years

Further document the safety of this regimen. Treatment-emergent adverse events will be summarized by MedDRA coding terms and separate tabulations will be produced for treatment-emergent adverse events, treatment-emergent serious adverse events, discontinuations due to adverse events, and treatment-emergent events of at least Grade 3 severity. A treatment-emergent adverse event is defined as an adverse event that was deemed to be related to the study intervention.

Outcome measures

Outcome measures
Measure	ALK-inhibitor Naive n=1 Participants Patients in this arm has no prior exposure to ALK-inhibitor and were treated with IPI-504 at 225mg/m2 twice a week for 2 weeks followed by 10 days off with cycles repeating every 21 days.	ALK-inhibitor Pre-treated n=2 Participants Patients in this arm had received prior exposure to ALK-inhibitor and were treated with IPI-504 at 225mg/m2 twice a week for 2 weeks followed by 10 days off with cycles repeating every 21 days.
Number of Participants With Treatment-Emergent Adverse Events discontinuations due to adverse events	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events Treatment-emergent adverse events	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events Treatment-emergent serious adverse events	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events treatment-emergent events ≥ grade 3	0 participants	0 participants

Adverse Events

ALK-inhibitor Naive

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

ALK-inhibitor Pre-treated

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	ALK-inhibitor Naive n=1 participants at risk No prior exposure to ALK-inhibitor	ALK-inhibitor Pre-treated n=2 participants at risk Prior exposure to ALK inhibitor
General disorders Fatigue	100.0% 1/1 • 1 Year	0.00% 0/2 • 1 Year
Musculoskeletal and connective tissue disorders Arthralgias	0.00% 0/1 • 1 Year	50.0% 1/2 • 1 Year
Hepatobiliary disorders Elevated AST	0.00% 0/1 • 1 Year	50.0% 1/2 • 1 Year
Blood and lymphatic system disorders Anemia	100.0% 1/1 • 1 Year	50.0% 1/2 • 1 Year

Additional Information

Lecia Sequist

Massachusetts General Hospital

Phone: 617-726-7812

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place