PFS was defined as the time from the baseline CT scan to disease progression per RECIST or death for any cause. Progressive disease (PD) was defined as

• at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or
• the appearance of 1 or more new lesions or
• the unequivocal progression of existing nontarget lesions

Overall survival was defined as the time from first dose to death or the date last known alive.

PET imaging was completed on selected patients only from one investigative site. Treatment phase PET and biopsy was completed on any day from Cycle 1 Day 2 through Day 10. PET imaging data were analyzed utilizing the European Organization for Research and Treatment of Cancer (EORTC) PET Study Group guidelines [Young H, Eur J Cancer, 1999]. Tumor response was considered a complete response (CR) or a partial response (PR).

Treatment-emergent AEs were defined as AEs that occurred from the time of first dose through 30 days after the last dose of study medication. The Investigator graded the severity of AEs according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria:

Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Life threatening Grade 5 = Death

A Serious AE is defined as any AE which results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or constitutes an important medical event.

Dose modification includes dose delay and dose reduction.

N = 25 participants

N = 20 participants GIST = gastrointestinal stromal tumor

The ECOG scale is as follows:

• Grade 0: Fully active, able to carry on all pre-disease activities without restriction;
• Grade 1: Restricted in physically strenuous activity, ambulatory and able to carry out work of a light nature;
• Grade 2: Ambulatory and capable of all self-care but unable to work. Up and about more than 50% of waking hours;
• Grade 3: Capable of only limited self-care, confined to bed or chair > 50% of waking hours;
• Grade 4: Completely disabled. Cannot carry on any self-care. Confined to bed or Chair.

Target Lesion Response Criteria, RECIST (version 1.0)

• Complete response - disappearance of all target lesions
• Partial response - at least a 30% decrease in the sum of the longest diameter of target lesions
• Stable disease - neither sufficient shrinkage to qualify for partial response nor sufficient increase to qualify for progressive disease
• Progressive disease - at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or the appearance of 1 or more new lesions

Objective response included participants whose best response with confirmation was a complete response (CR) or partial response (PR) from first dose until progression or end of study.

• CR: disappearance of all target lesions and non-target lesions and no new lesions
• PR: at least a 30% decrease in the sum of the longest diameter of target lesions, no disease progression for non-target lesions, and no new lesions

Full analysis set

PFS was defined as the time from the baseline CT scan to disease progression per RECIST or death for any cause. Progressive disease (PD) was defined as

• at least a 20% increase in the sum of the longest diameter of target lesions, taking as reference the smallest sum longest diameter recorded since the treatment started or
• the appearance of 1 or more new lesions or
• the unequivocal progression of existing nontarget lesions

Full analysis set

Overall survival was defined as the time from first dose to death or the date last known alive.

Full analysis set

PET imaging was completed on selected patients only from one investigative site. Treatment phase PET and biopsy was completed on any day from Cycle 1 Day 2 through Day 10. PET imaging data were analyzed utilizing the European Organization for Research and Treatment of Cancer (EORTC) PET Study Group guidelines [Young H, Eur J Cancer, 1999]. Tumor response was considered a complete response (CR) or a partial response (PR).

Participants from one investigative site who provided consent for the PET/CT imaging.

Treatment-emergent AEs were defined as AEs that occurred from the time of first dose through 30 days after the last dose of study medication. The Investigator graded the severity of AEs according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria:

Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Life threatening Grade 5 = Death

A Serious AE is defined as any AE which results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect or constitutes an important medical event.

Dose modification includes dose delay and dose reduction.

Full analysis set