Study of Tisagenlecleucel in Chinese Pediatric and Young Adult Subjects With Relapsed or Refractory B-cell ALL

A Phase II, Single Arm, Multi-center Trial to Evaluate the Efficacy and Safety of Tisagenlecleucel in Chinese Pediatric and Young Adult Patients With Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

ClinicalTrials.gov Identifier: NCT04156659

Novartis Reference Number: CCTL019B2210

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This is a single arm, multi-center, phase II study to evaluate the efficacy and safety of tisagenlecleucel in Chinese pediatric and young adult subjects with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL)

Condition 
B-cell Acute Lymphoblastic Leukemia
Phase 
Phase 2
Overall status 
Not yet recruiting
Enrollment count 
26 participants
Start date 
May 31, 2021
Completion date 
Jul 30, 2027
Gender 
All
Age(s)
25 Years and older (Child, Adult)

Interventions

Biological
Tisagenlecleucel
A single intravenous (i.v.) infusion of CAR-positive viable T cells.

Eligibility Criteria

Key Inclusion Criteria:

Chinese patients age ≤25 years at the time of informed consent form (ICF) signature.

Relapsed or refractory B-cell ALL

2nd or greater bone marrow (BM) relapse OR
Any BM relapse after allogeneic SCT and must be ≥ 3 months from SCT at the time of screening OR
Primary refractory as defined as not achieving a CR after 2 cycles of a standard first line chemotherapy regimen or chemorefractory as defined by not achieving a CR after 1 cycle of standard chemotherapy for relapsed leukemia OR
Subjects with Ph+ ALL are eligible if they are intolerant to or relapsed/refractory after two lines of tyrosine kinase inhibitor (TKI) therapy, or if TKI therapy is contraindicated OR
Ineligible for allogeneic SCT because of: comorbid disease; other contraindications to allogeneic SCT conditioning regimen; lack of suitable donor; prior SCT; subject declines allogeneic SCT as a therapeutic option after documented discussion about the role of SCT with a BMT physician not part of the study team
For relapsed patients, CD19 tumor expression demonstrated in bone marrow or peripheral blood by flow cytometry within 3 months of screening
Bone marrow with ≥ 5% lymphoblasts on local morphologic assessment at screening
Adequate performance status, cardiac, hepatic, renal and pulmonary function at screening
Must have a leukapheresis material of non-mobilized cells accepted for manufacturing
Once all other eligibility criteria are confirmed, must have a leukapheresis material of non-mobilized cells received and accepted for manufacturing. Note: Leukapheresis product will not be shipped to or assessed for acceptance by the manufacturing site until documented confirmation of all other clinical eligibility criteria is received

Key Exclusion Criteria:

Isolated extra-medullary disease relapse
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-cell ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation)
Prior anti-CD19 directed therapy, gene therapy or adoptive T cell therapy
Active central nervous system (CNS) involvement by ALL
Active neurological autoimmune or inflammatory disorders (e.g. Guillain-Barre syndrome)
History or presence of clinically relevant CNS pathology, e.g., epilepsy, paresis, aphasia, stroke, severe brain injuries, cerebellar disease, organic brain syndrome, or psychosis.
Investigational medicinal product within the last 30 days or five half-lives (whichever is longer) prior to screening NOTE: Investigational therapies must not be used at any time while on study until the first progression following tisagenlecleucel infusion.
Previous or concurrent malignancy except for curatively treated non-melanoma skin cancers, in situ carcinoma (e.g. cervix, skin), and cancers in complete remission for at least 3 years and without evidence of recurrence

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Name: 
Novartis Pharmaceuticals
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]