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

Anti-Infective Agents

Blinatumomab

Antibodies

Inotuzumab Ozogamicin

Immunomodulating Agents

Immunoglobulins

Anti-Bacterial Agents

Antibiotics, Antitubercular

Immunotoxins

Immunoconjugates

Immunologic Factors

Patient to receive tisagenlecleucel after optional bridging therapy and lymphodepleting chemotherapy.

blinatumomab or inotuzumab per investigator's discretion after optional bridging chemotherapy

autologous cellular immunotherapy product

bispecific CD19-directed CD3 T-cell engager

Inotuzumab

This trial aims to compare the benefits and risks of tisagenlecleucel to blinatumomab or inotuzumab in adult patients with relapsed or refractory ALL. This trial investigates tisagenlecleucel as an additional treatment option for this patient population with high unmet medical need.

This is a phase III, open label, multinational, randomized trial. Eligible patients will be randomized 1:1 to Tisagenlecleucel treatment strategy (tisagenlecleucel after optional bridging chemotherapy and lymphodepleting chemotherapy) or control arm treatment strategy (blinatumomab or inotuzumab per investigator's discretion after optional bridging chemotherapy).

The randomization will be performed stratifying for the number of prior salvage therapies (0 vs. 1) and prior allogenic stem cell transplant (yes vs. no).

Tisagenlecleucel vs Blinatumomab or Inotuzumab for Patients With Relapsed/Refractory B-cell Precursor Acute Lymphoblastic Leukemia

Tisagenlecleucel Versus Blinatumomab or Inotuzumab for Adult Patients With Relapsed/Refractory B-cell Precursor Acute Lymphoblastic Leukemia: A Randomized Open Label, Multicenter, Phase III Trial

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

Time from randomization to death for any reason

EFS, assessed up to 48 months, is defined as the date from randomization to the earliest of (a) date of death due to any cause, (b) relapse after CR/CRi, or (c) treatment failure, which is defined as failure to achieve remission within 12 weeks of randomization.

Percentage of patients who achieved MRD negative CR/CRi at month 3 post randomization

ORR is defined as the proportion of subjects with best overall response (BOR) of CR or CRi, where the BOR is defined as the best response recorded from randomization until the start of new anticancer therapy or the data cut-off date, whichever is earlier

DOR is defined as the duration from the date when the response criteria of CR/CRi is first met to the date of relapse or death due to underlying cancer.

Probability of achieving CR/CRi based on all response assessments between randomization and month 12. This outcome measure will be based on all randomized patients and the assessment will be up to 48 months (from randomization of the first patient until 12 months after the randomization of the last patient).

Percentage of patients who have anti-tisagenlecleucel antibodies in the serum before randomization

Percentage of patients who develop anti-tisagenlecleucel antibodies in the serum after infusion of tisagenlecleucel

difference in response between patients with immunogenicity and patients without immunogenicity

Summary of qPCR detected tisagenlecleucel transgene concentrations

Summary of flow cytometry-detected tisagenlecleucel transgene concentrations

Relationship between the administered dose of tisagenlecleucel and response to treatment (complete response with or without hematological recovery). This assessment will be done for all patients for up to 48 months.

Describe the relationship between the cellular kinetics of tisagenlecleucel overtime and response.

Describe the relationship between the dose of tisagenlecleucel actually administered and cellular kinetics

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®)

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
Tisagenlecleucel vs Blinatumomab or Inotuzumab for Patients With Relapsed/Refractory B-cell Precursor Acute Lymphoblastic Leukemia	NCT03628053	Acute Lymphobla...	Tisagenlecleuce... Blinatumomab Inotuzumab	18 Years -	Novartis
CTL019 Out of Specification MAP for ALL or DLBCL Patients	NCT03601442	Acute Lymphobla... Diffuse Large B...	CTL019	1 Day - 25 Years	Novartis
Phase III B in Acute Lymphoblastic Leukemia	NCT03123939	Acute Lymphobla...	CTL019	- 25 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
Phase III B in Acute Lymphoblastic Leukemia	NCT03123939	Acute Lymphobla...	CTL019	- 25 Years	Novartis
Efficacy and Safety of Tisagenlecleucel in Adult Patients With Refractory or Relapsed Follicular Lymphoma	NCT03568461	Follicular Lymp...	tisagenlecleuce...	18 Years -	Novartis
Retreatment With CTL019/CTL119	NCT04419909	Lymphoma, B-Cel...	CD19 redirected...	18 Years -	University of Pennsylvania
CTL019 Out of Specification MAP for ALL or DLBCL Patients	NCT03601442	Acute Lymphobla... Diffuse Large B...	CTL019	1 Day - 25 Years	Novartis
TisaGenlecleucel in Elderly Patients With First-Relapsed or Primary Refractory Aggressive B-cell Non-Hodgkin Lymphoma	NCT04161118	Non Hodgkin Lym...	CTL019	60 Years - 80 Years	University of Cologne
Tisagenlecleucel in Adult Patients With Aggressive B-cell Non-Hodgkin Lymphoma	NCT03570892	Non-Hodgkin Lym...	Tisagenlecleuce... Platinum-based ...	18 Years -	Novartis
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 ctl019

Every month we try and update this database with for ctl019 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 ctl019

Every month we try and update this database with for ctl019 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 ctl019 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.