Trial Outcomes & Findings for Phase I/II Study to Assess the Safety and Activity of Enhanced TCR Transduced Autologous T Cells in Metastatic Melanoma (NCT NCT01350401)
NCT ID: NCT01350401
Last Updated: 2019-01-10
Results Overview
Number of Participants with NCI CTC V.4 Adverse Events related to study treatment greater than or equal to Grade 3
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
4 participants
Primary outcome timeframe
Up to 12 months
Results posted on
2019-01-10
Participant Flow
Participant milestones
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase I/II Study to Assess the Safety and Activity of Enhanced TCR Transduced Autologous T Cells in Metastatic Melanoma
Baseline characteristics by cohort
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 Participants
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
49 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
4 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 12 monthsPopulation: Participants who received cytoreductive chemotherapy followed by IV infusion of NY-ESO-1ᶜ²⁵⁹T
Number of Participants with NCI CTC V.4 Adverse Events related to study treatment greater than or equal to Grade 3
Outcome measures
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 Participants
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
Subject 1 - Day 60
|
Subject 2 - Manufactured Product
|
Subject 2 - Day 60
|
|---|---|---|---|---|
|
Adverse Events Related to Study Treatment
|
3 Participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Change from Baseline, every 4 weeks until Month 5 and then every other month through Month 11Number of participants with response as assessed by RECIST (version 1.1) criteria.
Outcome measures
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 Participants
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
Subject 1 - Day 60
|
Subject 2 - Manufactured Product
|
Subject 2 - Day 60
|
|---|---|---|---|---|
|
Tumor Response
|
0 Participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: 8 Weeks post T-cell infusionPopulation: Participants who received cytoreductive chemotherapy followed by infusion of NY-ESO-1ᶜ²⁵⁹T with functionality data
Measurement of functionality of NY-ESO-1ᶜ²⁵⁹T cells in the blood and tumor sites.
Outcome measures
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=7 T-cell sub population
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
Subject 1 - Day 60
n=7 T-cell sub population
|
Subject 2 - Manufactured Product
n=7 T-cell sub population
|
Subject 2 - Day 60
n=7 T-cell sub population
|
|---|---|---|---|---|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%Stem Cell Memory
|
31.1354 percentage of T cell sub population
|
61.545 percentage of T cell sub population
|
18.65934 percentage of T cell sub population
|
6.9 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%Central Memory
|
14.62221 percentage of T cell sub population
|
3.42 percentage of T cell sub population
|
6.85214 percentage of T cell sub population
|
6.9 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%Effector Memory RA
|
36.329 percentage of T cell sub population
|
32.475 percentage of T cell sub population
|
34.50644 percentage of T cell sub population
|
82.7 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%Effector Memory
|
17.8215 percentage of T cell sub population
|
2.56 percentage of T cell sub population
|
39.99068 percentage of T cell sub population
|
3.45 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%LAG-3+
|
0.40391 percentage of T cell sub population
|
0 percentage of T cell sub population
|
1.23488 percentage of T cell sub population
|
0 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%PD-1+
|
1.24694 percentage of T cell sub population
|
0.855 percentage of T cell sub population
|
4.11214 percentage of T cell sub population
|
0 percentage of T cell sub population
|
|
Determine the Functional Properties and Phenotype of Modified T-cells From Peripheral Blood and Tumor Sites.
%TIM-3+
|
97.74411 percentage of T cell sub population
|
5.985 percentage of T cell sub population
|
95.2126 percentage of T cell sub population
|
10.35 percentage of T cell sub population
|
SECONDARY outcome
Timeframe: Days 1, 5-9, 12-16, weekly thereafter through Week 12, monthly thereafter through Month 12, and during LTFUPopulation: Participants who received cytoreductive chemotherapy followed by infusion of NY-ESO-1ᶜ²⁵⁹T with persistence data
Measurement of NY-ESO-1ᶜ²⁵⁹T cells in blood (copies of WPRE per µg of genomic PBMC DNA)
Outcome measures
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 Participants
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
Subject 1 - Day 60
|
Subject 2 - Manufactured Product
|
Subject 2 - Day 60
|
|---|---|---|---|---|
|
Peak Persistence of Modified T-cells in the Peripheral Blood
|
56561.22 copies per μg of DNA
Interval 24984.1 to 126679.0
|
—
|
—
|
—
|
Adverse Events
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
Serious events: 4 serious events
Other events: 4 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 participants at risk
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
|---|---|
|
General disorders
Pyrexia
|
25.0%
1/4 • Number of events 4 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Investigations
Platelet count decreased
|
25.0%
1/4 • Number of events 4 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
50.0%
2/4 • Number of events 4 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Neutropenia
|
50.0%
2/4 • Number of events 4 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
Other adverse events
| Measure |
NYESO-1ᶜ²⁵⁹T Cells Administered IV as a Split Dose Over 2 Days
n=4 participants at risk
Participants who received cytoreductive chemotherapy followed by infusion of lentivirus-mediated genetically engineered NY-ESO-1ᶜ²⁵⁹T (Target dose: 5-10 billion cells)
|
|---|---|
|
General disorders
Fatigue
|
100.0%
4/4 • Number of events 4 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Gastrointestinal disorders
Nausea
|
75.0%
3/4 • Number of events 3 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Skin and subcutaneous tissue disorders
Rash
|
75.0%
3/4 • Number of events 3 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
50.0%
2/4 • Number of events 2 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Anemia
|
50.0%
2/4 • Number of events 2 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Gastrointestinal disorders
Constipation
|
50.0%
2/4 • Number of events 2 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
General disorders
Pyrexia
|
50.0%
2/4 • Number of events 2 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Investigations
Alanine aminotransferase increased
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Psychiatric disorders
Anxiety
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Investigations
Aspartate aminotransferase increased
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Infections and infestations
Candida infection
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Infections and infestations
Cellulitis
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
General disorders
Chills
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Psychiatric disorders
Depression
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Nervous system disorders
Dizziness
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Renal and urinary disorders
Dysuria
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Renal and urinary disorders
Hematuria
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Psychiatric disorders
Insomnia
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Nervous system disorders
Neuropathy peripheral
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Neutropenia decreased
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Pancytopenia
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Renal and urinary disorders
Pollakiuria
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Skin and subcutaneous tissue disorders
Skin Fissures
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Cardiac disorders
Supraventricular tachycardia
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Infections and infestations
Urinary Tract Infection
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Skin and subcutaneous tissue disorders
Vitiligo
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • Number of events 1 • Start of lymphodepletion to end of intervention phase (up to 12 months post T-cell infusion)
Participants who received NY-ESO-1ᶜ²⁵⁹T
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60