PARAMETER

DATA

Manufacturer

Novartis Pharmaceuticals Corporation

Transgene

CAR transgene

Indication

KYMRIAH is a CD19-directed genetically modified autologous T-cell immunotherapy indicated for the treatment of:

• Patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
• Adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

Virus and Serotype

CTL019 (murine) HIV-1 vector

Cell Substrate

1α (EF-1α) promoter

Manufacturing platform

Viral vector: It uses an upstream process (consisting of thawing of the working cell bank (WCB), expansion of the production cell bank, plasmid transfection, induction, and harvest), followed by a downstream purification process (consisting of filtration, chromatography, and nuclease treatment steps) to yield the ‘vector substance’ (purified bulk vector). The vector substance undergoes sterile filtration, concentration, and filling to obtain the vector product.

Transduced cells CTL019: The manufacture of CTL019 starts with the acceptance and thawing of the leukapheresis material and ends with the cryopreservation of the CAR-positive T-cell containing product. Washed leukapheresis cells are enriched and are then transduced with the vector. After static incubation, the cells are eventually expanded in a bioreactor. At the end of the culture period the cells are washed and cryopreserved.

Dose in vial/final container

• Pediatric and Young Adult B-cell ALL (up to 25 years of age)

A single dose of KYMRIAH contains 0.2 to 5.0 x 106 CAR-positive viable T cells per kg of body weight for patients 50 kg or less, or 0.1 to 2.5 x 108 CAR-positive viable T cells for patients more than 50 kg, suspended in a patient specific infusion bag for I.V. infusion. (3)

• Adult Relapsed or Refractory Diffuse Large B-cell Lymphoma

A single dose of KYMRIAH contains 0.6 to 6.0 x 108 CAR-positive viable T cells suspended in one or more patient-specific infusion bag(s) for I.V. infusion.

Dose/patient

Pediatric and Young Adult B-cell ALL (up to 25 years of age)

• For patients 50 kg or less, administer 0.2 to 5.0 x 106CAR-positive viable T cells per kg body weight intravenously.
• For patients above 50 kg, administer 0.1 to 2.5 x 108 total CAR-positive viable T cells (non-weight based) intravenously.

Adult Relapsed or Refractory Diffuse Large B-cell Lymphoma.

• Administer 0.6 to 6.0 x 108 CAR-positive viable T cells intravenously.

NCT

TRIAL PHASE

SUBJECTS ENROLLED

STUDY TITLE

COUNTRIES

NCT02435849

2

80

Determine Efficacy and Safety of CTL019 in Pediatric Patients with Relapsed and Refractory B-cell ALL and High-Risk B-cell ALL at First Relapse. Determine Feasibility and Safety of CTL019 Therapy in Pediatric Patients with High Risk B-cell ALL That Relapsed < 6 Months Post All-HSCT.

United States, Italy, Australia, Austria, Belgium, Canada, France, Germany, Japan, Norway, Spain