Trial Outcomes & Findings for Melanoma Treatment With White Blood Cells That Destroy MART Expressing Tumor Cells (NCT NCT01495572)
NCT ID: NCT01495572
Last Updated: 2015-10-15
Results Overview
Complete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10mm. Partial response (PR) is at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum diameters. Progressive disease (PD) is at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study) or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum diameters while on study.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
One year
Results posted on
2015-10-15
Participant Flow
Due to slow accrual, the investigator decided to close the study.
Participant milestones
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x10\^11 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
|---|---|---|
|
Overall Study
STARTED
|
0
|
5
|
|
Overall Study
COMPLETED
|
0
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Melanoma Treatment With White Blood Cells That Destroy MART Expressing Tumor Cells
Baseline characteristics by cohort
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x1011 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
n=5 Participants
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days(day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
—
|
48.4 years
STANDARD_DEVIATION 10.89 • n=7 Participants
|
48.4 years
STANDARD_DEVIATION 10.89 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
—
|
4 participants
n=7 Participants
|
4 participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
—
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Gender
Female
|
—
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Gender
Male
|
—
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
—
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
—
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
—
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
—
|
5 participants
n=7 Participants
|
5 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: One yearComplete response (CR) is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10mm. Partial response (PR) is at least a 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum diameters. Progressive disease (PD) is at least a 20% increase in the sum of diameters of target lesions taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study) or the appearance of one or more new lesions. Stable disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD taking as references the smallest sum diameters while on study.
Outcome measures
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x10\^11 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
n=5 Participants
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
|---|---|---|
|
Clinical Tumor Response
Complete Response
|
—
|
0 participants
|
|
Clinical Tumor Response
Partial Response
|
—
|
0 participants
|
|
Clinical Tumor Response
Progressive Disease
|
—
|
5 participants
|
|
Clinical Tumor Response
Stable Disease
|
—
|
0 participants
|
PRIMARY outcome
Timeframe: 10 monthsHere is the number of participants with adverse events. For the detailed list of adverse events, see the adverse event module.
Outcome measures
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x10\^11 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
n=5 Participants
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
|---|---|---|
|
Number of Participants With Adverse Events
|
—
|
5 participants
|
Adverse Events
High Dose (HD) Aldesleukin
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Peripheral Blood Lymphocytes (PBL)
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x1011 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
n=5 participants at risk
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days(day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
|---|---|---|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
—
0/0
|
20.0%
1/5 • Number of events 1
|
Other adverse events
| Measure |
High Dose (HD) Aldesleukin
Pts receiving high dose aldesleukin: Cyclophosphamide 60 mg/kg intravenous (IV) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus melanoma antigen recognized by T cells (MART)-127-35 reactive CD8+ peripheral blood lymphocytes (PBL) up to 3x1011 IV over 20-30 minutes on day 0, plus aldesleukin 720,000 IU/kg IV over 15 minutes, every 8 hrs for up to 5 days.
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days (day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
Aldesleukin: Only given to patients assigned to high dose (HD) Arm - 720,000 IU/kg IV over 15 minutes, every 8 hrs (+/- 1 hr) for up to 5 days (max 15 doses).-6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
Peripheral Blood Lymphocytes (PBL)
n=5 participants at risk
Cyclophosphamide 60 mg/kg intravenous (IV ) for days -7 and -6, Fludarabine 25 mg/m\^2 IV for days -5 to -1, plus MART-127-35 reactive CD8+ PBL up to 3x10\^11 IV over 20-30 minutes on day 0
Fludarabine: 25 mg/m\^2 IV (in the vein) for 5 days(day -5 to -1)
Cyclophosphamide: 60 mg/kg IV (in the vein) for days -7 and -6
MART-1 Reactive CD8+ PBL: IV over 30 minutes on day 0
|
|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
—
0/0
|
80.0%
4/5 • Number of events 4
|
|
Infections and infestations
Catheter related infection
|
—
0/0
|
20.0%
1/5 • Number of events 2
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
—
0/0
|
80.0%
4/5 • Number of events 4
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
—
0/0
|
20.0%
1/5 • Number of events 1
|
|
Investigations
Lymphocyte count decreased
|
—
0/0
|
100.0%
5/5 • Number of events 5
|
|
Investigations
Neutrophil count decreased
|
—
0/0
|
100.0%
5/5 • Number of events 5
|
|
Investigations
Platelet count decreased
|
—
0/0
|
100.0%
5/5 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
—
0/0
|
20.0%
1/5 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash maculopapular
|
—
0/0
|
20.0%
1/5 • Number of events 1
|
|
Investigations
White blood cell decreased
|
—
0/0
|
100.0%
5/5 • Number of events 5
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place