臨床研究等提出・公開システム

A Phase II, single arm, multicenter open label trial to determine the efficacy and safety of tisagenlecleucel (CTL019) in adult patients with refractory or relapsed (r/r) follicular lymphoma

Efficacy and safety of tisagenlecleucel in adult patients with refractory or relapsed follicular lymphoma

98

In this study, a total of 98 participants were enrolled. The mean age was 56.5 years (standard deviation, 10.34); there were 66 males and 32 females. The primary races were White (n=81) and Asian :Japanese (n=9).

A total of 98 patients were enrolled; 97 patients were infused with CTL019, 58 patients completed (ongoing/in follow-up), and 40 patients were not completed. The main reasons for discontinuation were death (n=22) and physician decision (n=7).

Adverse events were collected during the post-infusion period (starting at the day of infusion until the end of the study), up to maximum duration of 60 months for each patient. All-cause mortality was assessed from day of infusion up to 65.8 months. All-cause mortality - Total: 22/97 (22.68%) Serious adverse events (>=3%) - Total: 52/97 (53.61%) - Cytokine release syndrome: 19/97 (19.59%) - Pneumonia: 15/97 (15.46%) - Febrile neutropenia: 8/97 (8.25%) - COVID-19: 4/97 (4.12%) - Pyrexia: 3/97 (3.09%) Other (not including serious) adverse events (>=10%) - Total: 95/97 (97.94%) - Neutropenia: 43/97 (44.33%) - Cytokine release syndrome: 30/97 (30.93%) - Anaemia: 26/97 (26.80%) - Diarrhoea: 24/97 (24.74%) - Headache 23/97 (23.71%) - White blood cell count decreased: 22/97 (22.68%)

The primary endpoint was the complete response rate (CRR) per independent review committee (IRC). The CRR in the efficacy analysis set of 94 patients was 68.1% (95% confidence interval: 57.7 -77.3).

In heavily pretreated, high-risk patients with relapsed/refractory follicular lymphoma (r/r FL), CTL019 achieved high response rates. Safety was manageable, with no study treatment-related deaths as assessed by Novartis. No new safety signals or risks were identified, and the safety profile was consistent with the established CTL019 profile. Overall, CTL019 provided clinically meaningful benefit in r/r FL patients, including high-risk patients with limited treatment options.

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku2@novartis.com

Yamauchi Kyosuke

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku2@novartis.com

completed

Nov. 12, 2018

Interventional

Phase II, single-arm, multi-center, open-label study for r/r FL patients

treatment purpose

2

Refractory or relapsed Follicular Lymphoma (Grade 1, 2, 3A)
Radiographically measurable disease at screening

Evidence of histologic transformation
Follicular Lymphoma Grade 3B
Prior anti-CD19 therapy
Prior gene therapy
Prior adoptive T cell therapy
Prior allogeneic hematopoietic stem cell transplant
Active CNS involvement by malignancy

Both

Follicular Lymphoma

A single intravenous (i.v.) infusion of CTL019.

efficacy
CRR (Complete response rate)

Evaluate Overall Response Rate (ORR), duration of response (DOR) , relapse free survival (RFS), progression free survival (PFS), and overall survival (OS)
Safety
Immunogenicity and cellular kinetics

Oct. 23, 2018