Neoplasms

Blood and Lymph Conditions

All Conditions

Immune System Diseases

Symptoms and General Pathology

Rare Diseases

Leukemia

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Neoplasm, Residual

Lymphoma

Neoplasms by Histologic Type

Hematologic Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Acute Graft Versus Host Disease

Lymphosarcoma

Antineoplastic Agents

All Drugs and Chemicals

Tisagenlecleucel

Antineoplastic Agents, Immunological

Based on the subject's weight one of two possible dose ranges will be prepared for the subject:

Subjects ≤ 50 kg: 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight

OR

Subjects \> 50 kg: 0.1 to 2.5 x 10(8) CAR-positive viable T cells

Based on the subject's weight, one of two possible dose ranges will be prepared for the subject:

Subjects ≤ 50 kg: 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight

OR

Subjects \> 50 kg: 0.1 to 2.5 x 10(8) CAR-positive viable T cells

CTL019

Novartis Pharmaceuticals

Colleen Quinn

Matthew Kutny

Dresden Whitehead

Dana Salzberg

Anna Pawlowska

Lee Chen

Emily Hsieh

Eunice Kim

Satiro De Oliveira

Tina Templeman

Van Huynh

Alexandria Lim

Kara Davis

Reuven Schore

Joanna Gallacher

Deepakbabu Chellapandian

Sarah Votaw

Muna Qayed

Jennifer Pencek

April Rahrig

Clare Twist

Eileen Phifer

Timothy A Driscoll

Adam Nelson

Christine Phillips

Nycole Ferguson

Bill Chang

Aakruti Sharma

Shannon Maude

Taryn Harris

Samuel John

Mark De Hoyos

Amanda Lipsitt

Jenny Weiland

Christian Capitini

Tara Murphy

Julie-An Talano

R. Pieters

This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment \& follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up safety will continue under a separate protocol per health authority guidelines.

Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

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.

DFS is defined as the time from the date of tisagenlecleucel infusion to the date of the first documented morphological relapse, occurrence of secondary malignancy or death due to any cause.

OS is defined as the time from date of first tisagenlecleucel infusion to the date of death due to any reason.

Minimal Residual Disease (MRD) negative remission (complete remission (CR) or Complete remission with incomplete blood count recovery CRi)) at Month 12 without SCT after tisagenlecleucel infusion.

Assessing the effect of tisagenlecleucel on DFS if new anticancer therapy is not available.

Assessing the percentage of participants who achieved MRD negative complete response (CR) or Complete remission with incomplete blood count recovery (CRi) status as determined by central laboratory using multi-parameter flow cytometry.

Absolute lymphocyte CD19 count of \<50/uL

Success in manufacturing of tisagenlecleucel dose for patients who are ≥1 year and \<3 years as respective time points.

Patient reported outcome to assess quality of life in patients aged 8 and above. The 23 items PedQL (Pediatrics Quality of Life Inventory) measure core dimensions of health as delineated by the World Health Organization, as well as role (school) functioning.

Items measured include 1) Physical Functioning (8 items), 2) Emotional Functioning (5 items), 3) Social Functioning (5 items), and 4) School Functioning (5 items).

Each item is measured on a 5 point Likert scale with 0 indicating "never" and 4 indicating "almost always".

The Likert scores are reversed scored and linearly transform to a 0-100 scale with 0=100, 1-75, 2=50, 5=25, and 4=0. A higher score indicates better health-related quality of life (HRQoL).

Patient reported outcome to assess health status in patients aged 8 and above. The EQ-5D (European Quality of Life -5 Dimensions) measures a wide range of health conditions and treatments; it is composed of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and a visual analogue scale (EQ-visual analogue scale \[EQ-VAS\]) that records the patient's self-rated overall health state.

Respondents rate each of these 5 dimensions from "no problem", "some problem," or "extreme problem".

The EQ-VAS records the respondents' self-related health on a vertical, visual analogue scale. The range for EQ-VAS is from 0 (=the worst health you can imagine) to 100 (=the best health you can imagine).

Speed of performance (mean of the log10 transformed reaction times for correct responses)

This test measures the speed of performance (mean of the log10 transformed reaction times for correct responses)

This test looks at the total number of errors made in attempting to learn the hidden pathways during a single session.

This test measures the accuracy of performance (arcsine transformation of the square root of the percentage of correct responses). The test also measures the speed of performance (mean of the log10 transformed reaction times for correct responses).

This test measures the accuracy of performance (arcsine transformation of the square root of the proportion of correct responses).

Impact of immunogenicity on clinical response

Transgene concentration as detected by qPCR in target tissue

Summary of tisagenlecleucel CAR-positive viable T cells measured by flow cytometry in target tissue

Relationship between B-cell recovery and transgene levels

The maximum (peak) observed in peripheral blood or other body fluid drug concentration after single dose administration (% or copies/μg)

The time to reach maximum (peak) peripheral blood or other body fluid drug concentration after single dose administration (days)

The AUC from time zero to day 29 and 84 or other disease assessment days, in peripheral blood (% or copies/μg x days )

The AUC from time zero to Tmax in peripheral blood (% or copies/μg x days)

The half-life associated with the elimination phase slope of a semi logarithmic concentration-time curve (days) in peripheral blood

The last observed quantifiable concentration in peripheral blood (% or copies/μg)

The time of last observed quantifiable concentration in peripheral blood (days)

Clinical response summarized by quartile of administered doses

Impact of tisagenlecleucel exposure on day 29 response; The AUC from time zero to day 29 in peripheral blood (% or copies/μg x days )

Impact of tisagenlecleucel exposure on day 29 response; The maximum (peak) observed in peripheral blood or other body fluid drug concentration after single dose administration (% or copies/μg)

Impact of tisagenlecleucel exposure on day 29 response; The time to reach maximum (peak) peripheral blood or other body fluid drug concentration after single dose administration (days)

Impact of tisagenlecleucel exposure on day 29 response; The half-life associated with the elimination phase slope of a semi logarithmic concentration-time curve (days) in peripheral blood

Children's Oncology Group

Lymphoma, Large B-Cell, Diffuse

Lymphoma, B-Cell

Lymphoma, Non-Hodgkin

B-cell Lymphoma

Diffuse Large B-Cell Lymphoma

MAP requests are initiated by a licensed physician.https:// www.novart is.com/healthcare-professionals/managed-access-programs

Sajad Khazal

Emmanuel Katsanis

Leslie Popplewell

Michael Pulsipher

James Chng

Josh Sasine

Liora Schultz

Dimitrios Tzachanis

Deborah Schiff

Michelle Hermiston

Charalambos Andreadis

Christopher Seet

Gauri Sunkersett

Pamela Allen

Jonathon Cohen

Amelia Langston

Reem Karmali

Peter Riedell

John Cunningham

Rayne Rouce

Aaron Rapoport

Nancy Hardy

Saurabh Dahiya

Patrick Brown

Andreas Klein

Matthew Frigault

Steven Margossian

Caron Jacobson

Gregory Yanik

Mark Vander Lugt

Monalisa Ghosh

Abinav Deol

Julio Chavez

Edward Peres

Heather Stefanski

Fiona He

Mohamed Kharfan Dabaja

Armin Ghobadi

Michele Donato

Andrew Evens

Mathew Barth

Philip McCarthy

Lia Palomba

Craig Sauter

Prakash Satwani

Michael Scordo

Natalie Sophia Grover

Jeffrey Huo

Kirsten Boughan

Rajinder Bajwa

Jonathan Brammer

Samantha Jaglowski

Theodore Laetsch

Brandon Hayes-Lattin Hayes-Lattin

Stephan Grupp

Stephen Schuster

Managed Access Program (MAP) to provide access to CTL019, for acute lymphoblastic leukemia (ALL) or diffuse large b-cell lymphoma (DLBCL) patients with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release.

The purpose of this Managed Access Program (MAP) Treatment Plan is to provide access to CTL019, for acute lymphoblastic leukemia (ALL) or diffuse large b-cell lymphoma (DLBCL) patients with out of specification leukapheresis product and/or manufactured tisagenlecleucel out of specification for commercial release where no overwhelming safety concerns has been identified for manufacture and release of the out of specification product.

CTL019 Out of Specification MAP for ALL or DLBCL Patients

Managed Access Program (MAP) Cohort Treatment Plan CCTL019B2003I to Provide Access for Patients With Out of Specification Leukapheresis Product and/or Out of Specification Manufactured Tisagenlecleucel (CTL019; Kymriah®)

Axicabtagene ciloleucel

Patients (≥18 years of age) with (relapsed/refractory diffuse large B cell lymphoma \[R/R DLBCL\])

Pediatric and young adult patients (≤25 years of age) with B cell acute lymphoblastic leukemia (ALL) ( refractory, in relapse post transplant or in second or later relapse

Patients (≥18 years of age) with (relapsed/refractory diffuse large B cell lymphoma \[R/R DLBCL\]) or Pediatric and young adult patients (≤25 years of age) with B cell acute lymphoblastic leukemia (ALL) refractory, in relapse post transplant or in second or later relapse

KYMRIAH

YESCARTA

This was a Retrospective cohort study based on the PMSI data source

A retrospective database analysis was performed using the French national hospital claims database (Medicalized Information System Program - PMSI, 2015-2019), which includes discharge summaries for all hospital admissions in France (\~99% of French residents).

The patients were identified based on the CAR-T administration hospital stay, between 2017 and 2019. Based on the exhaustivity of the database, all patients treated with CAR-T (since 2018) were identified.

The study design included multiple periods of analysis based on the CAR-T process. Three main periods were defined: the historical period, the CAR-T period, and the post CAR-T period. The CAR-T period was divided in 2 sub-periods: pre CAR-T (including the apheresis procedure and 15 days before this procedure) and per CAR-T (including the lymphodepletion and CAR-T cell injection hospital stay until the end at the discharge date related to CAR-T cell injection hospital stay). The follow-up period started at the end of CAR-T hospital stay.

CAR-T populations:

KYMRIAH® DLBCL cohort:

* Adult patients (≥18 years of age) with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.
* Patient with a CAR-T administration hospital stay of Kymriah between 2017 and 2019

YESCARTA®DLBCL cohort:

* Adult patients (≥18 years of age) with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy:
* Patient with a CAR-T administration hospital stay of Yescarta between 2017 and 2019

KYMRIAH® ALL cohort:

* Pediatric and young adult patients (≤ 25 years of age) with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse:
* Patient with a CAR-T administration hospital stay of Kymriah between 2017 and 2019

CAR T Cells Real World Evidence Study Based on the French Hospital Claims Data Source (PMSI)

CAR-T Cells - Real Life Study Of Care Pathway And Total Cost Of Care For Patients Under Car T-Cell Treatment Based On The PMSI French Database

Overall survival indicator was defined as all cause death recorded at the hospital (MSO, HAD, SSR). Patients were censored at the date of last hospitalization observed (MSO/HAD/SSR/ACE).

Time to next treatment indicator was defined as the time between CAR-T injection and the date of the hospitalization or palliative care.

Time to next treatment or death indicator was defined as the first event occurring between TNTT or death presented during the hospitalization or palliative care.

Follow-up is the time between the CAR-T injection (index date) and the last hospital stay observed.

The CAR-T hospitalization included Medical, Surgical, Obstetrics (MSO) hospitalization tariff and extra tariff linked to CAR-T treatment reimbursed by the health care insurance

Behaviors and Mental Disorders

Lymphoma, Follicular

Aggression

Follicular Lymphoma

Antirheumatic Agents

Cyclophosphamide

Fludarabine

Fludarabine phosphate

Bendamustine Hydrochloride

Immunomodulating Agents

Lenalidomide

Ibrutinib

Patients will receive investigator's choice of optional platinum-based immunochemotherapy followed by lymphodepleting chemotherapy and a single dose of tisagenlecleucel

Patients will receive investigator's choice of platinum-based immunochemotherapy followed in responding patients by high dose chemotherapy and autologous hematopoietic stem cell transplant (HSCT)

Investigator's choice of optional platinum-based immunochemotherapy (ie. R-ICE, R-GemOx, R-GDP, R-DHAP) + Lymphodepleting chemotherapy (fludarabine with cyclophosphamide or bendamustine) + Tisagenlecleucel (a second generation CAR-T composed of a CD19 antigen-binding domain, a 4-1BB costimulatory domain and a CD3-ζ signaling domain)

Tisagenlecleucel after optional bridging and lymphodepleting chemotherapy

Investigator's choice of platinum-based immunochemotherapy (ie. R-ICE, R-GemOx, R-GDP, R-DHAP)+ High dose chemotherapy (ie. BEAM) + autologous HSCT.

\*Ibrutinib or lenalidomide may be used in patients who are no longer eligible for autologous HSCT after 2 cycles of immunochemotherapy

Platinum-based immunochemotherapy followed in responding patients with high dose chemotherapy and autologous hematopoietic stem cell transplant (HSCT)

This is a randomized, open label, multicenter phase III trial comparing the efficacy, safety, and tolerability of tisagenlecleucel to Standard Of Care in adult patients with aggressive B-cell Non-Hodgkin Lymphoma after failure of rituximab and anthracycline containing frontline immunochemotherapy.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients	NCT03876769	B-Cell Acute Ly...	CTL019	1 Year - 25 Years	Novartis
CTL019 Out of Specification MAP for ALL or DLBCL Patients	NCT03601442	Acute Lymphobla... Diffuse Large B...	CTL019	1 Day - 25 Years	Novartis
CAR T Cells Real World Evidence Study Based on the French Hospital Claims Data Source (PMSI)	NCT05349201	Diffuse Large B... Acute Lymphobla...	KYMRIAH YESCARTA	18 Years - 30 Years	Novartis
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients	NCT03876769	B-Cell Acute Ly...	CTL019	1 Year - 25 Years	Novartis
CAR T Cells Real World Evidence Study Based on the French Hospital Claims Data Source (PMSI)	NCT05349201	Diffuse Large B... Acute Lymphobla...	KYMRIAH YESCARTA	18 Years - 30 Years	Novartis
CTL019 Out of Specification MAP for ALL or DLBCL Patients	NCT03601442	Acute Lymphobla... Diffuse Large B...	CTL019	1 Day - 25 Years	Novartis
CAR T Cells Real World Evidence Study Based on the French Hospital Claims Data Source (PMSI)	NCT05349201	Diffuse Large B... Acute Lymphobla...	KYMRIAH YESCARTA	18 Years - 30 Years	Novartis
Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma	NCT03570892	Non-Hodgkin Lym...	Tisagenlecleuce... Platinum-based ...	18 Years -	Novartis
Autologous CD22 CAR T Cells Following Commercial CD19 CAR T Cells in B Cell Malignancies	NCT06408194	Leukemia Acute Lymphobla...	CD22CART infusi... Tisagenlecleuce...	1 Year - 25 Years	Stanford University
Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)	NCT04225676	Acute Lymphobla...	Tisagenlecleuce...	2 Years - 25 Years	Novartis

Spots Global Cancer Trial Database for kymriah

Every month we try and update this database with for kymriah cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for kymriah

Every month we try and update this database with for kymriah cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for kymriah cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.