Trial Outcomes & Findings for A Phase II Study of Oral Cyclophosphamide and Sirolimus (OCR) in Advanced Sarcoma (NCT NCT00743509)
NCT ID: NCT00743509
Last Updated: 2015-09-28
Results Overview
Patients who were evaluable for response to therapy, alive and without evidence of sarcoma disease progression. Target lesions followed were lesions that had progressed by World Health Organization (WHO) criteria. Disease progression is defined as a greater than or equal to 25% increase in the sum of the product of target lesions, or unequivocal progression of non-target lesions or the appearance of new tumor lesions \>10mm.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
49 participants
Primary outcome timeframe
6 months
Results posted on
2015-09-28
Participant Flow
Participants came in to the University of Michigan Health System outpatient Hematology/Oncology clinic and were consented to participate in this research project after a description of the research was presented by their physician. Forty-nine eligible patients were enrolled from September 2008 to December 2009.
Eligible participants previously treated with chemotherapy underwent screening. Fifty-one patients were consented, 49 enrolled.
Participant milestones
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Overall Study
STARTED
|
49
|
|
Overall Study
COMPLETED
|
47
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
A Phase II Study of Oral Cyclophosphamide and Sirolimus (OCR) in Advanced Sarcoma
Baseline characteristics by cohort
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
n=49 Participants
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
40 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=99 Participants
|
|
Age, Continuous
|
57 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
49 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: Patients that tolerated and completed at least one 28 day cycle of therapy were considered evaluable
Patients who were evaluable for response to therapy, alive and without evidence of sarcoma disease progression. Target lesions followed were lesions that had progressed by World Health Organization (WHO) criteria. Disease progression is defined as a greater than or equal to 25% increase in the sum of the product of target lesions, or unequivocal progression of non-target lesions or the appearance of new tumor lesions \>10mm.
Outcome measures
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
n=47 Participants
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Number of Patients Alive Without Disease Progression
|
10 participants
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: All patients who completed at least one 28 day cycle
Median overall duration of survival.
Outcome measures
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
n=47 Participants
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Median Overall Survival Time
|
298 days
Interval 193.0 to 408.0
|
Adverse Events
Oral Cyclophosphamide and Sirolimus (OCR)
Serious events: 17 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
n=49 participants at risk
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Cardiac disorders
Hypotension
|
2.0%
1/49 • Number of events 1
|
|
General disorders
Fatigue
|
2.0%
1/49 • Number of events 1
|
|
General disorders
Fever
|
8.2%
4/49 • Number of events 4
|
|
General disorders
Death
|
4.1%
2/49 • Number of events 2
|
|
Gastrointestinal disorders
Dehydration
|
2.0%
1/49 • Number of events 1
|
|
Gastrointestinal disorders
Distension/bloating, abdominal
|
2.0%
1/49 • Number of events 1
|
|
Gastrointestinal disorders
Enteritis (inflammation of the small bowel)
|
2.0%
1/49 • Number of events 1
|
|
Gastrointestinal disorders
Ileus, GI (functional obstruction of bowel, i.e., neuroconstipation)
|
2.0%
1/49 • Number of events 1
|
|
Vascular disorders
Hemorrhage, CNS
|
2.0%
1/49 • Number of events 1
|
|
Vascular disorders
Hemorrhage, pulmonary/upper respiratory
|
2.0%
1/49 • Number of events 1
|
|
Hepatobiliary disorders
Cholecystitis
|
2.0%
1/49 • Number of events 1
|
|
Infections and infestations
Infection with Grade 3 or 4 neutrophils
|
4.1%
2/49 • Number of events 2
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils
|
2.0%
1/49 • Number of events 1
|
|
Metabolism and nutrition disorders
Potassium, serum-high (hyperkalemia)
|
2.0%
1/49 • Number of events 1
|
|
Metabolism and nutrition disorders
Potassium, serum-low (hypokalemia)
|
2.0%
1/49 • Number of events 1
|
|
Nervous system disorders
Confusion
|
2.0%
1/49 • Number of events 1
|
|
Gastrointestinal disorders
Pain
|
2.0%
1/49 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain
|
2.0%
1/49 • Number of events 1
|
|
General disorders
Pain - Other
|
2.0%
1/49 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
4.1%
2/49 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.0%
1/49 • Number of events 1
|
|
Renal and urinary disorders
Urinary retention (including neurogenic bladder)
|
2.0%
1/49 • Number of events 1
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
4.1%
2/49 • Number of events 2
|
Other adverse events
| Measure |
Oral Cyclophosphamide and Sirolimus (OCR)
n=49 participants at risk
Sarcoma patients were given oral Cyclophosphamide and Sirolimus (OCR) in 28 day cycles.
Cyclophosphamide and Sirolimus : The dose of cyclophosphamide will start at 200 mg (4 tablets) per day on day 1 and will be taken for 7 days every other week of a 28 day cycle.
Subjects will take 12 mg (12 tablets) of sirolimus on day 1 of treatment as a loading dose followed by 4 mg (4 tablets) daily continuously
|
|---|---|
|
Blood and lymphatic system disorders
Hemoglobin
|
38.8%
19/49 • Number of events 31
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
20.4%
10/49 • Number of events 17
|
|
Blood and lymphatic system disorders
Lymphopenia
|
51.0%
25/49 • Number of events 42
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
|
16.3%
8/49 • Number of events 9
|
|
Blood and lymphatic system disorders
Platelets
|
12.2%
6/49 • Number of events 10
|
|
General disorders
Fatigue
|
22.4%
11/49 • Number of events 12
|
|
General disorders
Fever
|
10.2%
5/49 • Number of events 5
|
|
Metabolism and nutrition disorders
Weight loss
|
6.1%
3/49 • Number of events 3
|
|
Gastrointestinal disorders
Diarrhea
|
8.2%
4/49 • Number of events 4
|
|
Investigations
Mucositis/stomatitis (clinical exam)
|
6.1%
3/49 • Number of events 3
|
|
Gastrointestinal disorders
Nausea
|
16.3%
8/49 • Number of events 8
|
|
Gastrointestinal disorders
Vomiting
|
8.2%
4/49 • Number of events 4
|
|
Investigations
Creatinine
|
8.2%
4/49 • Number of events 6
|
|
Metabolism and nutrition disorders
Glucose, serum-high (hyperglycemia)
|
12.2%
6/49 • Number of events 9
|
|
Investigations
Triglyceride, serum-high (hypertriglyceridemia)
|
6.1%
3/49 • Number of events 4
|
|
General disorders
Pain
|
6.1%
3/49 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
10.2%
5/49 • Number of events 5
|
|
Renal and urinary disorders
Cystitis
|
6.1%
3/49 • Number of events 3
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
6.1%
3/49 • Number of events 3
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place