Trial Outcomes & Findings for Anti-NY ESO-1 mTCR Peripheral Blood Lymphocytes (NCT NCT02774291)
NCT ID: NCT02774291
Last Updated: 2023-08-04
Results Overview
Toxicity graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 4.0.
Recruitment status
TERMINATED
Study phase
EARLY_PHASE1
Target enrollment
3 participants
Primary outcome timeframe
Up to 15 years
Results posted on
2023-08-04
Participant Flow
Participant milestones
| Measure |
Treatment (mTCR, Aldesleukin)
Patients receive standard cyclophosphamide IV over 1 hour on days -7 to -6 and fludarabine phosphate via IVPB over 30 minutes on days -5 to -1 followed by anti-ESO (cancer/test antigen) mTCR-transduced autologous peripheral blood lymphocytes IV over 20-30 minutes on day 0 and aldesleukin IV over 15 minutes approximately every 8 hours on days 0-4. Patients also receive filgrastim SC on days 1-4.
Aldesleukin: Given IV
Anti-thyroglobulin mTCR-transduced Autologous Peripheral Blood Lymphocytes: Given IV
Cyclophosphamide: Given IV
Filgrastim: Given SC
Fludarabine Phosphate: Given IVPB
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Treatment (mTCR, Aldesleukin)
Patients receive standard cyclophosphamide IV over 1 hour on days -7 to -6 and fludarabine phosphate via IVPB over 30 minutes on days -5 to -1 followed by anti-ESO (cancer/test antigen) mTCR-transduced autologous peripheral blood lymphocytes IV over 20-30 minutes on day 0 and aldesleukin IV over 15 minutes approximately every 8 hours on days 0-4. Patients also receive filgrastim SC on days 1-4.
Aldesleukin: Given IV
Anti-thyroglobulin mTCR-transduced Autologous Peripheral Blood Lymphocytes: Given IV
Cyclophosphamide: Given IV
Filgrastim: Given SC
Fludarabine Phosphate: Given IVPB
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Anti-NY ESO-1 mTCR Peripheral Blood Lymphocytes
Baseline characteristics by cohort
| Measure |
Treatment (mTCR, Aldesleukin)
n=3 Participants
Patients receive standard cyclophosphamide IV over 1 hour on days -7 to -6 and fludarabine phosphate via IVPB over 30 minutes on days -5 to -1 followed by anti-ESO (cancer/test antigen) mTCR-transduced autologous peripheral blood lymphocytes IV over 20-30 minutes on day 0 and aldesleukin IV over 15 minutes approximately every 8 hours on days 0-4. Patients also receive filgrastim SC on days 1-4.
Aldesleukin: Given IV
Anti-thyroglobulin mTCR-transduced Autologous Peripheral Blood Lymphocytes: Given IV
Cyclophosphamide: Given IV
Filgrastim: Given SC
Fludarabine Phosphate: Given IVPB
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 15 yearsPopulation: The study was officially closed on 7/1/2020 as documented on the final progress report dated 8/4/2020. Data for this Outcome Measure limited to 3 participants
Toxicity graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 4.0.
Outcome measures
| Measure |
Treatment (mTCR, Aldesleukin)
n=3 Participants
Patients receive standard cyclophosphamide IV over 1 hour on days -7 to -6 and fludarabine phosphate via IVPB over 30 minutes on days -5 to -1 followed by anti-ESO (cancer/test antigen) mTCR-transduced autologous peripheral blood lymphocytes IV over 20-30 minutes on day 0 and aldesleukin IV over 15 minutes approximately every 8 hours on days 0-4. Patients also receive filgrastim SC on days 1-4.
Aldesleukin: Given IV
Anti-thyroglobulin mTCR-transduced Autologous Peripheral Blood Lymphocytes: Given IV
Cyclophosphamide: Given IV
Filgrastim: Given SC
Fludarabine Phosphate: Given IVPB
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Number of Participants With Toxicity Graded According to NCI-CTCAE Version 4.0
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 weeks post evaluations scan and at 3, 6, and 12 months and annually thereafterPopulation: Data was never collected/aggregated and in vivo survival of TCR gene-engineered cells outcome measure was never analyzed due to the limited number of participants and early termination. The study was officially closed on 7/1/2020 as documented on the final IRB progress report dated 8/4/2020.
TCR and vector presence will be quantitated in PBMC samples (5-10mL) using established PCR techniques. This will provide data to estimate the in vivo survival of lymphocytes derived from the infused cells. In addition, measurement of CD4+ and CD8+ T-cells will be conducted and studies of these T-cell subsets in the circulation will be determined by using specific PCR assays capable of detecting the unique DNA sequence for each retroviral vector engineered T-cell. Descriptive statistics will be used to determine the in vivo survival of TCR gene-engineered cells.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 15 yearsPopulation: Data to support the Objective Response Rate outcome measure was never collected and analyzed due to the limited number of participants enrolled and early termination. The study was officially closed on 7/1/2020 as documented on the final progress report dated 8/4/2020.
Objective Response Rate will be graded based on the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 criteria. Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD): 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). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study Descriptive statistics will be used to determine the objective response rate.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (mTCR, Aldesleukin)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 2 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment (mTCR, Aldesleukin)
n=3 participants at risk
Patients receive standard cyclophosphamide IV over 1 hour on days -7 to -6 and fludarabine phosphate via IVPB over 30 minutes on days -5 to -1 followed by anti-ESO (cancer/test antigen) mTCR-transduced autologous peripheral blood lymphocytes IV over 20-30 minutes on day 0 and aldesleukin IV over 15 minutes approximately every 8 hours on days 0-4. Patients also receive filgrastim SC on days 1-4.
Aldesleukin: Given IV
Anti-thyroglobulin mTCR-transduced Autologous Peripheral Blood Lymphocytes: Given IV
Cyclophosphamide: Given IV
Filgrastim: Given SC
Fludarabine Phosphate: Given IVPB
Laboratory Biomarker Analysis: Correlative studies
|
|---|---|
|
Nervous system disorders
Headache
|
66.7%
2/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
General disorders
Edema limbs
|
33.3%
1/3 • Number of events 2 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Investigations
Weight Gain
|
66.7%
2/3 • Number of events 2 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Gastrointestinal disorders
Vomiting
|
66.7%
2/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
General disorders
Chills
|
66.7%
2/3 • Number of events 2 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Vascular disorders
Hypotension
|
66.7%
2/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Cardiac disorders
Sinus Tachycardia
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Investigations
Neutrophil Count Decreased
|
66.7%
2/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
33.3%
1/3 • Number of events 2 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Nervous system disorders
Lethargy
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Psychiatric disorders
Confusion
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
General disorders
Fatigue
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
General disorders
Fever
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Investigations
Creatinine Increased
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Investigations
Platelet Count Decreased
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
General disorders
Chest Pain
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Vascular disorders
Flushing
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Musculoskeletal and connective tissue disorders
Bone Pain
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Appetite
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep apnea
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Metabolism and nutrition disorders
Acidosis
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Investigations
Blood Bilirubin Increased
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Renal and urinary disorders
Urinary Frequency
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Cardiac disorders
Tachycardia
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
|
Gastrointestinal disorders
Mucositis oral
|
33.3%
1/3 • Number of events 1 • 9 months
The study was closed via final progress report on 08/03/2020 and no further AE data were collected.
|
Additional Information
Braunschweig, Ira
Albert Einstein College of Medicine/Montefiore medical center
Phone: 718-798-7474
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place