Novartis Pharmaceuticals

CTL019 Out of Specification MAP for ALL or DLBCL Patients

Official Title: 
Managed Access Program (MAP) to Provide Access to CTL019, for ALL or DLBCL Patients With Out of Specification Leukapheresis Product and/or Manufactured Tisagenlecleucel Out of Specification for Commercial Release
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.

EA Type
Intermediate-size Population
Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-cell Lymphoma (DLBCL)

Biological: CTL019
Other Name: tisagenlecleucel
Other Name: Kymriah

Eligibility Criteria: 

Inclusion Criteria:

- Written informed consent must be obtained prior to any screening procedures or treatment assignment.

- Has a patient specific batch of CTL019 which is out of specification either due to out of specification incoming apheresis or final product not meeting commercial release.

- Not excluded from commercial manufacturing under the prescribing guidelines for their country

- Out of specification material has not been deemed to pose an undue safety risk to the patient

- Is suffering from a serious or life-threatening disease or condition

- Repeat leukapheresis is not feasible per the treating physician assessment

- Does not have access to a comparable or satisfactory alternative treatment

- Is not eligible for participation in any of the IMP's ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options, the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient

- Meets any other relevant medical criteria for compassionate use of the investigational product

- Is not being transferred from an ongoing clinical trial for which they are still eligible

Exclusion Criteria:

- Product can be commercially manufactured per the specification of the country in which treatment will occur.

- Patients who are able to repeat leukapheresis.

- Evidence of CD19 negative disease

- HIV positive patients

- Patients with active replication of Hep B or active or latent Hep C

- History of hypersensitivity to any drugs or metabolites of similar chemical classes as tisagenlecleucel.

- Uncontrolled active infection or inflammation

- History of unstable angina or MI within 6 months prior to screening

- Any medical condition identified by the investigator that may impact the assessment of the safety or efficacy outcomes in relation to study treatment

Eligibility Gender: 
United States

Children's Hospital Alabama
Birmingham, Alabama, 35233

Banner University Medical Center of Arizona -Tucson
Tucson, Arizona, 85724

Los Angeles, California, 90027

Los Angeles, California, 90095

Stanford University, Lucile Packard Children's Hospital
Palo Alto, California, 94304

Palo Alto, California, 94304

Rady Childrens - UCSD
San Diego, California, 92123

University of Colorado - Children's Hospital Colorado
Aurora, Colorado, 80045

University of Colorado
Aurora, Colorado, 80045

University of Miami
Coral Gables, Florida, 33146

Jacksonville, Florida, 32224

Johns Hopkins - All Children's Hospital
Saint Petersburg, Florida, 33701

Tampa, Florida, 33612

Northside Hospital Atlanta
Atlanta, Georgia, 30042

Atlanta, Georgia, 30322

Atlanta, Georgia, 30322

University of Chicago
Chicago, Illinois, 60637

Indiana University Health - Riley Hospital for Children - Indiana University
Indianapolis, Indiana, 46202

University of Iowa
Iowa City, Iowa, 52242

Johns Hopkins University
Baltimore, Maryland, 21287

Dana Farber Cancer Institute - DFCI/MGH/BWH/Boston Childrens
Boston, Massachusetts, 02494

Boston, Massachusetts, 62111

University of Michigan
Ann Arbor, Michigan, 48109

Henry Ford Health System/University Hospital
Detroit, Michigan, 48202

University of Minnesota
Minneapolis, Minnesota, 55455

University of Nebraska
Omaha, Nebraska, 68198

Hackensack University Hospital
Hackensack, New Jersey, 07601

Buffalo, New York, 14203

Columbia University and New York and Presbyterian Hospital
New York, New York, 10032

New York, New York, 10065

Cincinnati Children's Hospital
Cincinnati, Ohio, 45229

Jewish Mercy Hospital
Cincinnati, Ohio, 45236

University Hospital - Case Western Reserve/Seidman Cancer Center
Cleveland, Ohio, 44106

University Hospital - Rainbow Babies
Cleveland, Ohio, 44106

The Ohio State University
Columbus, Ohio, 43210

Portland, Oregon, 97239

Philadelphia, Pennsylvania, 19104

University of Pennsylvania
Philadelphia, Pennsylvania, 19104

Medical University of South Carolina
Charleston, South Carolina, 29425

Avera McKennan
Sioux Falls, South Dakota, 57105

Methodist Healthcare-Memphis Hospital
Memphis, Tennessee, 38174

Sarah Cannon Cancer Center - HCA/Tristar/Centennial Medical Center
Nashville, Tennessee, 37203

St. David's South Medical Center Austin Texas
Austin, Texas, 78704

Medical City Dallas Texas
Dallas, Texas, 57230

Dallas, Texas, 75390

Houston Methodist Hospital - Baylor College of Medicine
Houston, Texas, 77030

Houston, Texas, 77030

Houston, Texas, 77030

Huntsman Cancer Institute - University of Utah
Salt Lake City, Utah, 84112

University of Virginia
Charlottesville, Virginia, 22903

Virginia Commonwealth University
Richmond, Virginia, 23124

Morgantown, West Virginia, 26506

University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792

Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226


Novartis Pharmaceuticals

Novartis Pharmaceuticals

Novartis Pharmaceuticals
EA Number: 
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
MeSH Terms: 
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Lymphoid, Lymphoma, B-Cell, Lymphoma, Large B-Cell, Diffuse
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