Clinical trial NCT03601442: CTL019 Out of Specification MAP for ALL or DLBCL Patients
Official title: 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 type: EXPANDED_ACCESS
Overall status: AVAILABLE
Conditions: Acute Lymphoblastic Leukemia (ALL)
Diffuse Large B-cell Lymphoma (DLBCL)
Keywords: Acute Lymphoblastic Leukemia
ALL
Pediatric
Diffuse Large b-cell Lymphoma
DLBCL
Adult
Relapse
Refractory
CTL019
tisagenlecleucel
Kymriah
CART19
CART
CAR T cells
Chimeric antigen receptor
Manufacturing
Expanded Access
Out of Specification
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2018-07-26
Sex: ALL
Minimum age (years): 1
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

Patients eligible for inclusion in this Treatment Plan have to meet all of the following criteria:

1. Patients must be treated at a healthcare facility that has been certified /qualified by Novartis to dispense and administer tisagenlecleucel in line with the tisagenlecleucel Risk Evaluation and Mitigation Strategy (REMS) in the United States (US) or the local Risk Management Plan (RMP).

   - Note that tisagenlecleucel treatment should be initiated under the direction of and supervised by a HCP experienced in the treatment of hematological malignancies and trained for administration and management of patients treated with tisagenlecleucel. The healthcare facility must have tocilizumab for use in the event of cytokine release syndrome and emergency equipment per patient prior to infusion on site and ensure timely access to additional doses of tocilizumab; for detailed requirements refer to the approved local label.
2. Patients must be prescribed tisagenlecleucel in line with the locally approved indications (for the precise indication statements see approved local product label). These may include:

   * pediatric and young adult patients up to and (including) 25 years of age with refractory/relapsed (r/r) acute lymphoblastic leukemia (B-ALL)
   * adult patients with r/r diffuse large B-cell lymphoma (DLBCL)
3. Informed consent must be obtained prior to treatment
4. The incoming apheresis material and/or the final manufactured product is out of specification due to failure to meet acceptance or release specifications

Exclusion Criteria:

Patients eligible for this Treatment Plan must not meet any of the following criteria:

1. Contraindications as per the approved local label or the IB.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Gilbert
country: United States
facility: Banner MDACC
state: Arizona
city: Phoenix
country: United States
facility: University of Arizona Cancer Center
state: Arizona
city: Phoenix
country: United States
facility: Banner University of Arizona Medical Center
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Childrens Hospital
state: Arizona
city: Tucson
country: United States
facility: Banner Uni Medical Center Tucson
state: Arizona
city: Duarte
country: United States
facility: City of Hope National Medical Center Hematology&HematCellTransplant
state: California
city: Duarte
country: United States
facility: City of Hope
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles CRFB002H2301
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: Mattel Childrens Hospital UCLA
state: California
city: Los Angeles
country: United States
facility: University of California at Los Angeles UCLA
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Children s Hospital Stanford University
state: California
city: Palo Alto
country: United States
facility: Lucile Packard Childrens Hospital
state: California
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: San Diego
country: United States
facility: UC San Diego
state: California
city: San Diego
country: United States
facility: University of California at San Diego, Moores Cancer Ctr UCSD
state: California
city: San Diego
country: United States
facility: Rady Children s Hospital Dept of Oncology
state: California
city: San Diego
country: United States
facility: Rady Childrens Hospital
state: California
city: San Francisco
country: United States
facility: Ucsf Benioff Childrens Hospital Pediatric Hematology Oncology
state: California
city: San Francisco
country: United States
facility: University of Califronia San Francisco
state: California
city: Santa Monica
country: United States
facility: University of California, Los Angeles
state: California
city: Stanford
country: United States
facility: Stanford Healthcare
state: California
city: Denver
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: New Haven
country: United States
facility: Yale Cancer Center
state: Connecticut
city: Miami
country: United States
facility: University of Miami Hospital
state: Florida
city: Saint Petersburg
country: United States
facility: JHMI All Children s Hospital
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine Emory University (12)
state: Georgia
city: Atlanta
country: United States
facility: Emory University School of Medicine/Winship Cancer Institute Winship Cancer Center
state: Georgia
city: Atlanta
country: United States
facility: Emory University School of Medicine/Winship Cancer Institute
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Cellular Therapies Lab
state: Georgia
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H Lurie Childrens Hospital of Chicago
state: Illinois
city: Chicago
country: United States
facility: Northwestern
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
city: Chicago
country: United States
facility: University of CHicago Comer Children's Hospital Regulatory
state: Illinois
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Louisville
country: United States
facility: Healthwatch Plaza Research Associates
state: Kentucky
city: Baltimore
country: United States
facility: University of Maryland Medical Center UMMC
state: Maryland
city: Baltimore
country: United States
facility: University of Maryland Medical Center
state: Maryland
city: Baltimore
country: United States
facility: University of Maryland
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins Outpatient Center
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins University Hospital
state: Maryland
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital Cancer Center
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital DF/HCC institutions
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: MGH Cancer Center MGH Cancer Center
state: Massachusetts
city: Boston
country: United States
facility: MGH Cancer Center
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber Cancer Institute Dana Farber Cancer Institute
state: Massachusetts
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan IRB
state: Michigan
city: Ann Arbor
country: United States
facility: University of Michigan Medical Center
state: Michigan
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Detroit
country: United States
facility: Moffitt Cancer Center
state: Michigan
city: Detroit
country: United States
facility: Wayne State University - Karmanos Cancer Institute
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Hospital Henry Ford
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota Masonic Childrens Hospital
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: Mayo CLinic
state: Minnesota
city: Saint Louis
country: United States
facility: WUSTL Jewish Barnes
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center Hackensack UMC
state: New Jersey
city: Hackensack
country: United States
facility: Hackensack University Medical Center Onc Dept
state: New Jersey
city: New Brunswick
country: United States
facility: Robert Wood Johnson Medical School
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute Rosewell
state: New York
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center MSKCC
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center Oncology
state: New York
city: New York
country: United States
facility: Columbia University Medical Center New York Presbyterian CUMC Research Pharmacy
state: New York
city: New York
country: United States
facility: Columbia University Medical Center New York Presbyterian Neuroendocrine Unit
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center MSKCC (9)
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina Chapel Hill Physician Office Building
state: North Carolina
city: Chapel Hill
country: United States
facility: University of North Carolina Health System
state: North Carolina
city: Charlotte
country: United States
facility: Atrium Heath
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center Dept. of DUMC (4)
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children s Hospital Medical Center
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital
state: Ohio
city: Cincinnati
country: United States
facility: Oncology Hematology Care Inc Drug Shipment
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center Univ. Hospitals of Cleveland
state: Ohio
city: Columbus
country: United States
facility: Nationwide Childrens Hospital Center
state: Ohio
city: Columbus
country: United States
facility: OSU Wexner MC James CH
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Hospitals Clinic The Ohio State University
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Hospitals Clinic
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Regulatory
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Comprehensive Cancer Center Ohio State UMC James Cancer Ct
state: Ohio
city: Elyria
country: United States
facility: Hematology Oncology Center, Inc.
state: Ohio
city: Portland
country: United States
facility: Oregon Health Sciences University
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Portland
country: United States
facility: OHSU
state: Oregon
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Childrens Hospital of Philadelphia CTRB 3006
state: Pennsylvania
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia Children's Hospital Of Phil(2)
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children s Hospital of Philadelphia Patient Treatment
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children s Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Childrens Hospital of Philadelphia Children's Hosp Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Childrens Hospital of Philadelphia Pediatric Oncology/Stem Cell T
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Childrens Hospital of Philadelphia Regulatory
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania Health System CERL080AUS90
state: Pennsylvania
city: Philadelphia
country: United States
facility: Pennsylvania Oncology/Hemotology Dept.ofPenn. Onc./Hem.
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: Fox Chase Cancer Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Cancer Institute
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina MUSC
state: South Carolina
city: Greenville
country: United States
facility: Prisma Health Cancer Institute
state: South Carolina
city: Sioux Falls
country: United States
facility: Avera McKennan Hospital and University Health Center
state: South Dakota
city: Memphis
country: United States
facility: Methodist University Hospital Blood and Marrow Transplant
state: Tennessee
city: Dallas
country: United States
facility: Texas Cancer Center ( Medical City Dallas Hospital) TX Cancer Center
state: Texas
city: Dallas
country: United States
facility: Univof Texas Southwestern Medical Center, Dept of Pediatrics
state: Texas
city: Dallas
country: United States
facility: U of TX Southwestern Medical Center - SimmonsCompCancerCtr Satellite
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center Pediatric Hematology/Onc
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center UT SW Medical Ctr of Dallas
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Houston Methodist Cancer Center Methodist Cancer Center (2)
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center/University of Texas MDACC
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center/University of Texas StudyCoordinator:CTKI258A2102
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Houston
country: United States
facility: Methodist Hospital / Methodist Cancer Center Houston Methodist Hospital
state: Texas
city: San Antonio
country: United States
facility: Methodist Healthcare System
state: Texas
city: San Antonio
country: United States
facility: Methodist Hospital
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah / Huntsman Cancer Institute Huntsman Cancer Cente
state: Utah
city: Salt Lake City
country: United States
facility: Primary Childrens Medical Center Oncology
state: Utah
city: Salt Lake City
country: United States
facility: LDS Hospital
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia Health Systems UVA
state: Virginia
city: Richmond
country: United States
facility: VCU Health Systems, MCV
state: Virginia
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center Dept. of FHCRC
state: Washington
city: Madison
country: United States
facility: University of Wisconsin Hospital and Clinics
state: Wisconsin
city: Madison
country: United States
facility: University of Wisconsin Hospital and Clinics Hematopoietic Stem Cell Lab
state: Wisconsin
city: Madison
country: United States
facility: University of Wisconsin Hospital and Clinics
state: Wisconsin
city: Madison
country: United States
facility: University Wisconsin Children's Hospital
state: Wisconsin
city: Milwaukee
country: United States
facility: Childrens Hospital of Wisconsin
state: Wisconsin
city: Milwaukee
country: United States
facility: Medical College of Wisconsin Med College of WI (29)
state: Wisconsin
city: Murdoch
country: Australia
facility: Novartis Investigative Site
state: Western Australia
city: Toronto
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Montreal
country: Canada
facility: Novartis Investigative Site
state: Quebec
Study officials:
References: