Study Description
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.
Interventions
CTL019
Eligibility Criteria
Inclusion Criteria:
CD19 expressing B-cell Acute Lymphoblastic Leukemia
De novo NCI HR B-ALL who received first-line treatment and are MRD ≥ 0.01% at EOC. EOC bone marrow MRD will be collected prior to screening and will be assessed by multi-parameter flow cytometry using central laboratory analysis.
Age 1 to 25 years at the time of screening
Lansky (age < 16 years) or Karnofsky (age ≥ 16 years) performance status ≥ 60%
Adequate organ function during the screening period:
A. Renal function based on age/gender B. ALT ≤ 5 times ULN for age C. AST ≤ 5 times ULN for age D. Total bilirubin < 2 mg/dL (for Gilbert's Syndrome subjects total bilirubin < 4 mg/dL)
E. Adequate pulmonary function defined as:
no or mild dyspnea (≤ Grade 1)
oxygen saturation of > 90% on room air F. Adequate cardiac function defined as LVSF ≥ 28% confirmed by echocardiogram or LVEF ≥ 45% confirmed by echocardiogram or MUGA within 6 weeks of screening
Prior induction and consolidation chemotherapy allowed: 1st line subjects: ≤ 3 blocks of standard chemotherapy for first-line B-ALL, defined as 4-drug induction, Berlin-Frankfurt-Münster (BFM) consolidation or Phase 1b, and interim maintenance with high-dose methotrexate.
Exclusion Criteria:
M3 marrow at the completion of 1st line induction therapy
M2 or M3 marrow or persistent extramedullary disease at the completion of first-line consolidation therapy or evidence of disease progression in the peripheral blood or new extramedullary disease prior to enrollment. Patients with previous CNS disease are eligible if there is no active CNS involvement of leukemia at the time of screening.
Philadelphia chromosome positive ALL
Hypodiploid: less than 44 chromosomes and/or DNA index < 0.81, or other clear evidence of a hypodiploid clone
Prior tyrosine kinase inhibitor therapy
Subjects with concomitant genetic syndromes associated with bone marrow failure states: such as subjects with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Subjects with Down syndrome will not be excluded.
Subjects with Burkitt's lymphoma/leukemia (i.e. subjects with mature B-ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation)
Has had treatment with any prior anti-CD19 therapy 9. Treatment with any prior gene or engineered T cell therapy
Other protocol-defined inclusion/exclusion may apply.
Study Location
Novartis Investigative Site
Recruiting
Gent,9000,Belgium
Novartis Investigative Site
Recruiting
Montreal,Quebec,H3T 1C5,Canada
Novartis Investigative Site
Recruiting
Calgary,Alberta,T3B 6A8,Canada
Novartis Investigative Site
Recruiting
Roma,RM,00165,Italy
Novartis Investigative Site
Recruiting
Monza,MB,20900,Italy
Prinses Maxima Centrum voor Kinderoncologie
Recruiting
Utrecht,R. Pieters,3584 - CS,Netherlands
Novartis Investigative Site
Recruiting
Oslo,0424,Norway
Novartis Investigative Site
Recruiting
Esplugues De Llobregat,Barcelona,08950,Spain
Novartis Investigative Site
Recruiting
Goteborg,SE 416 85,Sweden
Novartis Investigative Site
Recruiting
Stockholm,141 86,Sweden
Novartis Investigative Site
Recruiting
London,WC1E 6HX,United Kingdom
Novartis Investigative Site
Recruiting
London,WC1N 3JH,United Kingdom
Rady Children s Hospital CTBM100C2401
Recruiting
San Diego,(858-966-8153) -- Deborah Schiff,92123 - California,United States
Children's Healthcare of Atlanta Emory University IRB
Recruiting
Atlanta,Sarah Votaw email: [email protected] -- Muna Qayed,30342 - Georgia,United States
Washington Univ School of Medicine CTBM100C2412
Recruiting
Saint Louis,Bethany Kellogg (314-454-2253) email: [email protected] -- Thomas Pfeiffer,63110 - Missouri,United States
Mattel Childrens Hospital UCLA
Recruiting
Los Angeles,Eunice Kim email: [email protected] -- Satiro De Oliveira,90095 - California,United States
Univ of Texas Southwest Med Center
Recruiting
Dallas,Taryn Harris (214-648-1390) email: [email protected] -- Samuel John,75390-9034 - Texas,United States
Cinn Children Hosp Medical Center
Recruiting
Cincinnati,Adam Nelson (800-344-2462) email: [email protected] -- Christine Phillips,45229-3039 - Ohio,United States
Roswell Park Cancer Institute Main Centre
Recruiting
Buffalo,(716-845-4485) -- Clare Twist,14263 - New York,United States
Children's Hospital of Richmond at VCU Pediatric Hematology Oncology
Recruiting
Richmond,Grace Garren (804-828-9605) email: [email protected] -- Elizabeth Krieger,23219 - Virginia,United States
Yale School of Medicine
Recruiting
New Haven,Aron Flagg,06519 - Connecticut,United States
Medical Uni of South Carolina Medical Univ of SC
Recruiting
Charleston,Matt Smith (843-876-8614) email: [email protected] -- Michelle Hudspeth,29425 - South Carolina,United States
University of Chicago Hospital .
Recruiting
Chicago,James Labelle,60637 - Illinois,United States
City of Hope National Medical
Recruiting
Duarte,(+16262564673#85013) -- Anna Pawlowska,91010 - California,United States
Childrens Hospital Colorado .
Recruiting
Aurora,Darren Drake (720-777-5379) email: [email protected] -- Kelly Maloney,80045 - Colorado,United States
Stanford University Medical Center .
Recruiting
Stanford,Alexandria Lim (650-723-4000) email: [email protected] -- Kara Davis,94304 - California,United States
Methodist Children's Hospital .
Recruiting
San Antonio,Mark De Hoyos email: [email protected] -- Amanda Lipsitt,78229 - Texas,United States
Cleveland Clinic Foundation Main Centre
Recruiting
Cleveland,Nicole Previte (216-442-5232) email: [email protected] -- Rabi Hanna,44195 - Ohio,United States
University of Michigan
Recruiting
Ann Arbor,(734-936-8538),48109-2800 - Michigan,United States
Childrens Hospital Los Angeles SC CTL019
Recruiting
Los Angeles,Lee Chen (323-361-8670) email: [email protected] -- Emily Hsieh,90027 - California,United States
Texas Children's Cancer and Hematology Center
Recruiting
Houston,Grace Anand (832-824-1000) email: [email protected] -- Rayne Rouce,77030 - Texas,United States
Monroe Carell Jr Childrens Hospital at Vanderbilt
Recruiting
Nashville,(615-321-5247) -- Carrie Kitko,37232 - Tennessee,United States
Oregon Health and Science University .
Recruiting
Portland,Nycole Ferguson (503-494-5893) email: [email protected] -- Bill Chang,97239-3098 - Oregon,United States
Duke University Medical Center .
Recruiting
Durham,Eileen Phifer (919-681-6898) email: [email protected] -- Timothy A Driscoll,27710 - North Carolina,United States
University of Minnesota CAEB071B2201
Recruiting
Minneapolis,Mollie Koppes (612-273-6212) email: [email protected] -- Peter Gordon,55455 - Minnesota,United States
Childrens Hospital of Orange County CHOC Children's
Recruiting
Orange,Tina Templeman email: [email protected] -- Van Huynh,92868-3874 - California,United States
The Childrens Hosp of Philadelphia Div Gastroint., Hepat. & Nutr.
Recruiting
Philadelphia,Aakruti Sharma (215-590-2985) email: [email protected] -- Shannon Maude,19104 - Pennsylvania,United States
Nationwide Childrens Hospital suite T6B
Recruiting
Columbus,Trittnee Robinson (614-722-2556) email: [email protected] -- Mark Ranalli,43205 - Ohio,United States
Phoenix Children's Hospital
Recruiting
Phoenix,Dresden Whitehead (602-546-0985) email: [email protected] -- Dana Salzberg,85016 - Arizona,United States
Ann and Robert H Lurie Childrens Hospital of Chicago .
Recruiting
Chicago,Eric Brown (312-227-4811) email: [email protected] -- Sonali Chaudhury,60611 - Illinois,United States
James Whitcomb Riley Hospital for Children
Recruiting
Indianapolis,Jennifer Pencek (317-274-4281) email: [email protected] -- April Rahrig,46202 - Indiana,United States
University of Utah Clinical Trials Office .
Recruiting
Salt Lake City,Keeley Best (801-587-5711) email: [email protected] -- Luke Maese,84108 - Utah,United States
UCSF Medical Center .
Recruiting
San Francisco,Emily Theobald (415-353-1351) email: [email protected] -- Michelle Hermiston,94143 - California,United States
Childrens Hospital of Wisconsin
Recruiting
Milwaukee,Tara Murphy email: [email protected] -- Julie-An Talano,53226 - Wisconsin,United States
Children s Hospital of Alabama
Recruiting
Birmingham,Colleen Quinn email: [email protected] -- Matthew Kutny,35233 - Alabama,United States
University of Wisconsin Hospital and Clinics Pharmacy/Drug Shipping Address
Recruiting
Madison,Jenny Weiland email: [email protected] -- Christian Capitini,53792 - Wisconsin,United States
Johns Hopkins All Childrens
Recruiting
Saint Petersburg,Joanna Gallacher email: [email protected] -- Deepakbabu Chellapandian,33701 - Florida,United States
Univ Hospital - Cleveland/Rainbow Babies and Children's Hosp Pediatric Nephrology
Recruiting
Cleveland,Jahnahn Taghiani (218-844-3681) email: [email protected] -- Duncan Stearns,44106 - Ohio,United States
Childrens National Hospital
Recruiting
Washington,(202-476-5000) -- Reuven Schore,20010 - District of Columbia,United States
Worldwide Contacts
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