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NCT04035434

A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON)

Terminated Phase 1, PHASE2 Results posted Last updated 24 October 2025
What this trial tests

Phase 1, PHASE2 trial testing CTX110 in B-cell Malignancy in 93 participants. Terminated before completion.

Timeline
22 July 2019
Primary endpoint
4 October 2024
4 October 2024

Quick facts

Lead sponsorCRISPR Therapeutics AG
PhasePhase 1, PHASE2
StatusTerminated
Study typeINTERVENTIONAL
Allocationna
Designsequential
Maskingnone
Primary purposetreatment
Enrollment93
Start date22 July 2019
Primary completion4 October 2024
Estimated completion4 October 2024
Sites33 locations across France, Germany, Canada, Australia, United States, Spain

Drugs / interventions tested

Conditions studied

Sponsor

CRISPR Therapeutics AG — full company profile →

Who can join

18 and older, any sex, with B-cell Malignancy or Non-Hodgkin Lymphoma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Dose Escalation Phase 1: Number of Participants With Dose-limiting Toxicities (DLT) in NHL and B Cell ALL Population Primary · Up to 28 days

A DLT is defined as any of the following events occurring during the DLT evaluation period that persisted beyond the specified duration from onset: Grade ≥2 graft-versus-host disease (GvHD) that was steroid-refractory (e.g., progressive disease after 3 days of steroid treatment \[e.g., 1 mg/kg/day\], stable disease after 7 days, or partial response after 14 days of treatment); death during the DLT period, unless due to disease progression; Grade 4 neurotoxicity of any duration that was related or possibly related to CTX110; or any CTX110-related grade 3 or 4 toxicity deemed clinically signific

GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)0
Dose Expansion Phase 1 and Phase 2: Percentage of Participants With Objective Response Rate in NHL Population Primary · Up to 5 years

The objective response rate (complete response + partial response) was analyzed as per Lugano Response Criteria for Malignant Lymphoma, as determined by independent central radiology review. For participants who received the second course of treatment, the response assessments before and after the second course are combined for the derivation of objective response. Percentages are calculated with the number of participants in the specific analysis set in each column as the denominator. Confidence intervals (CI) of percentage are calculated with Clopper-Pearson exact method.

GroupValue95% CI
Phase 1: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)60.938.5 – 80.3
Phase 2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)75.034.9 – 96.8
Dose Escalation Phase 1: Percentage of Participants With Objective Response Rate in B Cell ALL Population Secondary · Up to 5 years

The objective response rate (complete response + complete remission with incomplete blood count recovery) was analyzed as per response criteria adapted from the National Comprehensive Cancer Network guidelines for treatment of acute lymphoblastic leukemia Version 2.2021, as determined by independent central radiology review. For -participants who receive the second course of treatment, the response assessments before and after the second course are combined for the derivation of objective response. Percentages are calculated with the number of participants in the specific analysis set in each

GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)25.00.6 – 80.6
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)40.05.3 – 85.3
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)85.742.1 – 99.6
Dose Escalation and Expansion Phase 1: Duration of Response in NHL Population Secondary · Up to 5 years

Duration of response was only reported for the participants who showed objective response events. This was calculated as the time between first objective response and date of disease progression or death due to any cause. Median duration of response was calculated with the Kaplan-Meier method. Confidence intervals of median duration of response were calculated with the Brookmeyer and Crowley method with log-log transformation.

GroupValue95% CI
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)9.20NA – NA
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)3.751.68 – NA
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)3.520.95 – NA
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)1.020.46 – 1.31
Phase 1: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)3.601.91 – 8.57
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)NANA – NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2.202.00 – NA
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)1.561.05 – NA
Dose Escalation and Expansion Phase 1: Progression Free Survival (PFS) in NHL Population Secondary · Up to 5 years

Progression-free survival (PFS) and event-free survival were calculated as the difference between date of CTX110 infusion and date of disease progression or death due to any cause. The median of PFS was calculated with the Kaplan-Meier method. The CIs of median PFS were calculated with the Brookmeyer and Crowley method with log-log transformation.

GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)0.920.79 – NA
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)1.910.85 – NA
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)2.430.95 – NA
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)1.540.82 – NA
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)1.770.76 – 2.10
Phase 1: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)2.831.02 – 3.94
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)NANA – NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2.920.79 – NA
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)1.940.92 – NA
Dose Escalation and Expansion Phase 1: Median Overall Survival (OS) in NHL Population Secondary · Up to 5 years

Overall survival was calculated as the time between date of first dose of CTX110 and death due to any cause. Participants who are alive at the data cutoff date will be censored at their last date known to be alive. Median overall survival was calculated with the Kaplan-Meier method. CIs of median overall survival were calculated with the Brookmeyer and Crowley method with log-log transformation.

GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)5.032.73 – NA
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)37.161.05 – NA
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)7.772.96 – NA
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)NA1.61 – NA
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)5.161.71 – 27.73
Phase 1: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)14.264.47 – NA
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)12.12NA – NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)7.391.18 – NA
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)7.621.71 – NA
Dose Escalation and Expansion: Number of Participants With Treatment Emergent Adverse Events (TEAEs) in NHL and B Cell ALL Population Secondary · Up to 5 years

A TEAE was any untoward medical occurrence or worsening of a pre-existing condition in a clinical trial participant who received the investigational medicinal product, regardless of a causal relationship with the treatment. An AE had to be classified as a serious adverse event (SAE) if it resulted in death, was life-threatening, required or prolonged hospitalization, caused persistent or significant disability or incapacity, led to a congenital anomaly or birth defect in a newborn, or was deemed a significant medical event by the investigator based on medical judgment.

GroupValue95% CI
Phase1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)10
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)34
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)4
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)7
Dose Escalation and Expansion: Number of Participants With Clinically Significant Laboratory Abnormalities in NHL and B Cell ALL Population Secondary · Up to 5 years

Blood samples were collected for the analysis of laboratory parameters including hematology, clinical chemistry and coagulation parameters. The hematology parameters included: Lymphocyte count decreased, Neutrophil count decreased, White blood cell decreased, Anemia, Platelet count decreased, leukocytosis and Lymphocyte count increased. Chemistry parameters included: Hypoalbuminemia, Aspartate aminotransferase increased, Alanine aminotransferase increased, Hypokalemia, Chronic kidney disease, Blood bilirubin increased, Creatinine increased, Hypermagnesemia, Hypernatremia, Hypercalcemia, Hyperk

Lymphocyte count decreased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)10
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)35
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)4
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)7
Neutrophil count decreased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)10
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)35
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)4
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)7
White blood cell decreased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)6
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)10
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)35
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)4
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)7
Anemia
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)4
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)8
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)17
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)3
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)2
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)3
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)3
Platelet count decreased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)4
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)6
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)19
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)2
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)3
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)3
Lymphocyte count increased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)0
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)1
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)0
Hypoalbuminemia
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)0
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)1
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)1
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)0
Aspartate aminotransferase increased
GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)0
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)0
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)1
Dose Escalation and Expansion: Mean Concentration of CTX110 in Blood Over Time Following the First CTX110 Infusion in NHL Population Secondary · Day 1 pre-infusion, Day 1 post-infusion, Day 2, Day 3, Day 5, Day 8, Day 10, Day 14, Day 21, Day 28, Month 2, Month 3, Month 6, Month 9, Month 12 and Month 24

Blood samples were collected for the analysis of mean concentration of CTX110 in blood (copies per micrograms deoxyribose nucleic acid \[DNA\]) over time following the first CTX110 infusion. Cohort A DL4a and DL4b were combined, as the same dose was administered. Mean concentration values of CTX110 have been presented by dose level.

Day 1 pre-infusion
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)2.90± 0.000
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)3.55± 1.592
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)2.90± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2.90± 0.000
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)2.90± 0.000
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)3.08± 0.934
Day 1 post-infusion
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)144.95± 147.856
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)829.37± 1552.483
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)472.20± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)105.22± 23.028
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)636.29± 352.700
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)979.25± 1658.499
Day 2
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)7.27± 6.403
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)10.77± 13.999
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)28.57± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)35.62± 22.359
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)72.39± 107.323
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)42.39± 101.461
Day 3
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)8.36± 5.310
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)12.46± 9.656
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)22.03± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)16.76± 13.050
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)204.43± 300.219
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)35.80± 98.739
Day 5
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)25.47± 21.898
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)220.78± 246.411
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)311.67± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)80.40± 33.878
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)487.13± 262.303
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)770.14± 3312.713
Day 8
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)3763.68± 8138.642
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)4337.42± 7721.088
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)13269.43± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)5844.98± 9600.631
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)719.13± 231.318
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)10277.87± 27526.126
Day 10
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)180.87± 209.131
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)452.03± 941.853
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)25719.13± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)19018.53± 42303.294
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)297.91± 248.623
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)4056.03± 19737.607
Day 14
GroupValue95% CI
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)2.90± 0.000
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)3.55± 1.592
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)9.93± NA
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)2435.23± 5423.525
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)51.73± 87.882
Phase 1/2: NHL: Cohort A: DL 4a + DL 4b(6x10^8 CAR+ T Cells)2186.81± 13766.282
Dose Escalation and Expansion Phase 1: Mean Concentration of CTX110 in Blood Over Time Following the First CTX110 Infusion in B Cell ALL Population Secondary · Day 1 pre-infusion, Day 1 post-infusion, Day 2, Day 3, Day 5, Day 8, Day 10, Day 14, Day 21, Day 28, and Month 2

Blood samples were collected for the analysis of mean concentration of CTX110 in blood (copies per micrograms deoxyribose nucleic acid \[DNA\]) over time following the first CTX110 infusion.

Day 1 pre-infusion
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)13.43± 21.067
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)3.29± 0.872
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)2.90± 0.000
Day 1 post-infusion
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)781.86± 1395.907
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)1983.57± 1677.746
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)602.75± 521.357
Day 2
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)4.20± 2.252
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)93.47± 149.220
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)18.13± 17.532
Day 3
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)13.46± 14.905
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)20.47± 25.356
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)7.95± 4.705
Day 5
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)28.65± 36.011
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)42.37± 47.444
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)62.85± 124.743
Day 8
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)71.30± 118.215
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)16846.17± 33459.507
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)15941.89± 30200.959
Day 10
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)226.32± 290.508
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)26.17± 41.517
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)14177.59± 23256.089
Day 14
GroupValue95% CI
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)1040.13± 1466.869
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)2.90± 0.000
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)380.29± 924.424
Dose Escalation and Expansion: Number of Participants With Serious Adverse Events (SAEs) in NHL and B Cell ALL Population Secondary · Up to 5 years

An AE had to be classified as a serious adverse event (SAE) if it resulted in death, was life-threatening, required or prolonged hospitalization, caused persistent or significant disability or incapacity, led to a congenital anomaly or birth defect in a newborn, or was deemed a significant medical event by the investigator based on medical judgment.

GroupValue95% CI
Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)2
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)5
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)8
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)0
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)1
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)4
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)3
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)3
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)1

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 5 years. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Phase 1: NHL: Cohort A Dose Level (DL) 1 (3×10^7 CAR+ T Cells)
Serious: 3/3 (100%)
Deaths: 3/3
Phase 1: NHL: Cohort A: DL 2 (1x10^8 CAR+ T Cells)
Serious: 2/3 (67%)
Deaths: 2/3
Phase 1: NHL: Cohort A: DL 3 (3x10^8 CAR+ T Cells)
Serious: 1/6 (17%)
Deaths: 4/11
Phase 1: NHL: Cohort A: DL 3b (4.5x10^8 CAR+ T Cells)
Serious: 1/6 (17%)
Deaths: 3/6
Phase 1: NHL: Cohort A: DL 4a (6x10^8 CAR+ T Cells)
Serious: 5/10 (50%)
Deaths: 9/10
Phase 1/2: NHL: Cohort A: DL 4b (6x10^8 CAR+ T Cells)
Serious: 8/35 (23%)
Deaths: 21/35
Phase 1: NHL: Cohort B: DL 4 (6x10^8 CAR+ T Cells)
Serious: 0/1 (0%)
Deaths: 1/1
Phase 1: NHL: Cohort C: DL 3 (3x10^8 CAR+ T Cells)
Serious: 1/5 (20%)
Deaths: 3/5
Phase 1: NHL: Cohort C: DL 4 (6x10^8 CAR+ T Cells)
Serious: 4/5 (80%)
Deaths: 4/5
Phase 1: B Cell ALL: Cohort D: DL 2 (1x10^8 CAR+ T Cells)
Serious: 3/4 (75%)
Deaths: 4/4
Phase 1: B Cell ALL: Cohort D: DL 3 (3x10^8 CAR+ T Cells)
Serious: 3/5 (60%)
Deaths: 2/5
Phase 1: B Cell ALL: Cohort D: DL 4 (6x10^8 CAR+ T Cells)
Serious: 1/7 (14%)
Deaths: 1/7

Serious adverse events (47 terms)

ReactionSystemPhase 1: NHL: Cohort A Dos…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1/2: NHL: Cohort A: …Phase 1: NHL: Cohort B: DL…Phase 1: NHL: Cohort C: DL…Phase 1: NHL: Cohort C: DL…Phase 1: B Cell ALL: Cohor…Phase 1: B Cell ALL: Cohor…Phase 1: B Cell ALL: Cohor…
Myelodysplastic syndromeNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Cytokine release syndromeImmune system disorders
Immune effector cell-associated neurotoxicity syndromeNervous system disorders
COVID-19Infections and infestations
PneumoniaInfections and infestations
Deep vein thrombosisVascular disorders
HypotensionVascular disorders
NeurotoxicityNervous system disorders
Hypertensive encephalopathyNervous system disorders
Febrile neutropeniaBlood and lymphatic system disorders
NeutropeniaBlood and lymphatic system disorders
PyrexiaGeneral disorders
PainGeneral disorders
Haemophagocytic lymphohistiocytosisImmune system disorders
Abdominal painGastrointestinal disorders
Rectal haemorrhageGastrointestinal disorders
CholecystitisHepatobiliary disorders
Pulmonary embolismRespiratory, thoracic and mediastinal disorders
CoughRespiratory, thoracic and mediastinal disorders
Urinary retentionRenal and urinary disorders
HaematuriaRenal and urinary disorders
Renal vein thrombosisRenal and urinary disorders
Lower respiratory tract infectionInfections and infestations
Respiratory syncytial virus infectionInfections and infestations
Bronchopulmonary aspergillosisInfections and infestations
Other adverse events (212 terms — click to expand)

ReactionSystemPhase 1: NHL: Cohort A Dos…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1: NHL: Cohort A: DL…Phase 1/2: NHL: Cohort A: …Phase 1: NHL: Cohort B: DL…Phase 1: NHL: Cohort C: DL…Phase 1: NHL: Cohort C: DL…Phase 1: B Cell ALL: Cohor…Phase 1: B Cell ALL: Cohor…Phase 1: B Cell ALL: Cohor…
NeutropeniaBlood and lymphatic system disorders
Cytokine release syndromeImmune system disorders
AnaemiaBlood and lymphatic system disorders
ThrombocytopeniaBlood and lymphatic system disorders
LeukopeniaBlood and lymphatic system disorders
ConstipationGastrointestinal disorders
FatigueGeneral disorders
DiarrhoeaGastrointestinal disorders
NauseaGastrointestinal disorders
Oedema peripheralGeneral disorders
CoughRespiratory, thoracic and mediastinal disorders
Blood fibrinogen decreasedInvestigations
DizzinessNervous system disorders
HeadacheNervous system disorders
ArthralgiaMusculoskeletal and connective tissue disorders
HypokalaemiaMetabolism and nutrition disorders
AstheniaGeneral disorders
PyrexiaGeneral disorders
Atrial fibrillationCardiac disorders
PruritusSkin and subcutaneous tissue disorders
RashSkin and subcutaneous tissue disorders
Decreased appetiteMetabolism and nutrition disorders
HypophosphataemiaMetabolism and nutrition disorders
LymphopeniaBlood and lymphatic system disorders
Blood magnesium decreasedInvestigations
HypotensionVascular disorders
HypertensionVascular disorders
Catheter site painGeneral disorders
PainGeneral disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
RhinorrhoeaRespiratory, thoracic and mediastinal disorders
InsomniaPsychiatric disorders
Alanine aminotransferase increasedInvestigations
Aspartate aminotransferase increasedInvestigations
ContusionInjury, poisoning and procedural complications
TachycardiaCardiac disorders
Immune effector cell-associated neurotoxicity syndromeNervous system disorders
LethargyNervous system disorders
Febrile neutropeniaBlood and lymphatic system disorders
Abdominal painGastrointestinal disorders

Most-reported serious reactions: Myelodysplastic syndrome, Cytokine release syndrome, Immune effector cell-associated neurotoxicity syndrome, COVID-19, Pneumonia, Deep vein thrombosis, Hypotension, Neurotoxicity.

Data from ClinicalTrials.gov NCT04035434 adverse events section.

Sponsor's own description

This is an open-label, multicenter, Phase 1/2 study evaluating the safety and efficacy of CTX110 in subjects with relapsed or refractory B-cell malignancies.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Engineering strategies to overcome the current roadblocks in CAR T cell therapy.
    Rafiq S, Hackett CS, Brentjens RJ. · · 2020 · cited 1111× · PMID 31848460 · DOI 10.1038/s41571-019-0297-y
  2. CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors.
    Wang D, Zhang F, Gao G. · · 2020 · cited 405× · PMID 32243786 · DOI 10.1016/j.cell.2020.03.023
  3. CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future.
    Uddin F, Rudin CM, Sen T. · · 2020 · cited 329× · PMID 32850447 · DOI 10.3389/fonc.2020.01387
  4. Current applications and future perspective of CRISPR/Cas9 gene editing in cancer.
    Wang SW, Gao C, Zheng YM, Yi L, et al · · 2022 · cited 305× · PMID 35189910 · DOI 10.1186/s12943-022-01518-8
  5. Engineering the next generation of cell-based therapeutics.
    Bashor CJ, Hilton IB, Bandukwala H, Smith DM, et al · · 2022 · cited 271× · PMID 35637318 · DOI 10.1038/s41573-022-00476-6
  6. RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.
    Yu AM, Choi YH, Tu MJ. · · 2020 · cited 271× · PMID 32929000 · DOI 10.1124/pr.120.019554
  7. Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing.
    Zhang S, Shen J, Li D, Cheng Y. · · 2021 · cited 266× · PMID 33391496 · DOI 10.7150/thno.47007
  8. CRISPR-Cas systems: Overview, innovations and applications in human disease research and gene therapy.
    Xu Y, Li Z. · · 2020 · cited 202× · PMID 33005303 · DOI 10.1016/j.csbj.2020.08.031

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04035434.

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