Soft Tissue Sarcoma
Conditions
Keywords
Soft Tissue Sarcoma, CC-4047, Pomalidomide
Brief summary
The purpose of the study is to determine the safety and efficacy of single agent CC-4047 (pomalidomide) in patients with advanced soft tissue sarcomas who have relapsed or are refractory to prior anticancer therapy.
Interventions
DRUGPomalidomide
Sponsors
Celgene
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Must be \> 18 years of age * Must have histologically confirmed soft tissue sarcoma * Must have locally recurrent unresectable, or metastatic soft tissue sarcoma, and have failed or relapsed after a minimum of one and a maximum of 3 prior systemic anticancer therapy regimens * Must have measurable or evaluable disease determined as per Response Evaluation Criteria in Solid Tumors (RECIST) criteria * Must have documented disease progression (PD) determined as per RECIST criteria within 3 months prior to study enrollment * Must have Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1
Exclusion criteria
* Pregnant or lactating females * Prior therapy with thalidomide or lenalidomide * Prior use of experimental/investigational drug therapy \< 3 months prior to treatment initiation * Prior chemotherapy, biologic or immunotherapy \< 3 weeks prior to treatment initiation * Prior radiotherapy \< 3 weeks prior to treatment initiation * Prior major surgery \< 3 weeks prior to treatment initiation * Absolute neutrophil count (ANC) \< 1.5 x 109 cells/L * Platelet count \< 100 x 109cells/L * Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase/serum glutamic pyruvate transaminase (ALT/SGPT) \> 3.0 x upper limit of normal (ULN) or \> 5.0 x ULN in the presence of demonstrable liver metastases * Known active central nervous system (CNS) metastases
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | AEs/SAEs were recorded from informed consent to 30 days post treatment discontinuation visit. Median treatment duration was 49 days (range: 3 to 102 days). | An adverse event (AE) is defined as any noxious, unintended, or untoward medical occurrence occurring at any dose that may appear or worsen in a study subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the study subject's health, including laboratory test values, regardless of etiology. A serious adverse event (SAE) 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; constitutes an important medical event. National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Events (CTCAE), Version 3.0, grades: 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, 5 = death. For more details, please see the Adverse Events section of this record. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Tumor Response as Assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee Guidelines | Assessed every 8 weeks for the first 8 months and then every 12 weeks thereafter, and at treatment discontinuation. Median treatment duration was 49 days (range: 3 to 102 days). | Changes in only the longest diameter (LD) of tumor lesions are used in RECIST criteria. Evaluation of target lesions: Complete Response (CR)=Disappearance of all target lesions; Partial Response (PR)=≥30% decrease in sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD; Progressed Disease (PD)=≥20% increase in sum of LD of target lesions taking as reference the smallest sum LD recorded since treatment started or the appearance of ≥1 new lesions; Stable Disease (SD)=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as reference the smallest sum LD since treatment started. For non-target lesions: CR= Disappearance of all non-target lesions and normalization of tumor marker level; Incomplete Response/SD=Persistence of ≥1 non-target lesions and/or maintenance of tumor marker level above normal limits; PD=Appearance of ≥1 new lesions; unequivocal progression of existing non-target lesions. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Pomalidomide 7 mg pomalidomide taken orally once daily (QD) on days 1 through 21 of each 28-day cycle | 7 |
| Total | 7 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Death | 1 |
| Overall Study | Disease Progression | 5 |
Baseline characteristics
| Characteristic | Pomalidomide |
|---|---|
| Age, Continuous | 58.9 years STANDARD_DEVIATION 13.52 |
| Race/Ethnicity, Customized Non-white | 0 participants |
| Race/Ethnicity, Customized White | 7 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 7 / 7 |
| serious Total, serious adverse events | 3 / 7 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs
An adverse event (AE) is defined as any noxious, unintended, or untoward medical occurrence occurring at any dose that may appear or worsen in a study subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the study subject's health, including laboratory test values, regardless of etiology. A serious adverse event (SAE) 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; constitutes an important medical event. National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Events (CTCAE), Version 3.0, grades: 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, 5 = death. For more details, please see the Adverse Events section of this record.
Time frame: AEs/SAEs were recorded from informed consent to 30 days post treatment discontinuation visit. Median treatment duration was 49 days (range: 3 to 102 days).
Population: All participants
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 AE | 7 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 AE suspected to be related to study drug | 6 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 Grade 3 AE | 4 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 Grade 4 AE | 1 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 SAE | 3 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | ≥1 SAE suspected to be related to study drug | 1 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | Death | 4 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | Death within 30 days of last dose of study drug | 1 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | Discontinuation due to AE | 2 participants |
| Pomalidomide | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs | Dose reduction and interruption due to AE | 1 participants |
Secondary
Tumor Response as Assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee Guidelines
Changes in only the longest diameter (LD) of tumor lesions are used in RECIST criteria. Evaluation of target lesions: Complete Response (CR)=Disappearance of all target lesions; Partial Response (PR)=≥30% decrease in sum of the longest diameter (LD) of target lesions taking as reference the baseline sum LD; Progressed Disease (PD)=≥20% increase in sum of LD of target lesions taking as reference the smallest sum LD recorded since treatment started or the appearance of ≥1 new lesions; Stable Disease (SD)=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as reference the smallest sum LD since treatment started. For non-target lesions: CR= Disappearance of all non-target lesions and normalization of tumor marker level; Incomplete Response/SD=Persistence of ≥1 non-target lesions and/or maintenance of tumor marker level above normal limits; PD=Appearance of ≥1 new lesions; unequivocal progression of existing non-target lesions.
Time frame: Assessed every 8 weeks for the first 8 months and then every 12 weeks thereafter, and at treatment discontinuation. Median treatment duration was 49 days (range: 3 to 102 days).
Population: This analysis was not done. Study enrollment was suspended due to a corporate strategic decision unrelated to patient safety, and the protocol was amended to evaluate safety only (efficacy data was stored but not cleaned or analyzed unless related to safety).