Tisagenlecleucel for Acute Lymphoblastic Leukemia (CASSIOPEIA Trial)

Recruiting in Palo Alto (17 mi)

+76 other locations

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Novartis Pharmaceuticals

No Placebo Group

Prior Safety Data

Approved in 3 jurisdictions

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for young people (1-25 years old) with high-risk B-cell Acute Lymphoblastic Leukemia who've had first-line treatment but still have detectable cancer cells. They need to be generally healthy, with good heart, kidney, liver function and no severe breathing problems.

Inclusion Criteria

My leukemia is CD19 positive.

My ALT levels are within 5 times the normal limit for my age.

My first treatment for B-ALL didn't clear all cancer cells, showing more than 0.01% MRD.

I can do most activities but may need help.

My AST levels are within 5 times the normal limit for my age.

I am between 1 and 25 years old.

My heart is strong enough for treatment, as confirmed by recent tests.

I experience little to no shortness of breath.

Exclusion Criteria

I have previously received treatments targeting CD19 or gene/T cell therapies.

My cancer cells have fewer than 44 chromosomes or a low DNA index.

My bone marrow is still highly affected by cancer after initial treatment.

I have been diagnosed with Burkitt's lymphoma/leukemia.

My leukemia has not fully responded to initial treatment or is progressing.

I have been treated with tyrosine kinase inhibitors before.

My leukemia is Philadelphia chromosome positive.

Participant Groups

The study tests the safety and effectiveness of a therapy called tisagenlecleucel (CTL019) in patients after initial leukemia treatments. It's an open-label phase II trial where participants are closely monitored over several phases including screening, pre-treatment, treatment & follow-up.

1Treatment groups

Experimental Treatment

Group I: Single dose of CTL019Experimental Treatment1 Intervention

Based on the subject's weight one of two possible dose ranges will be prepared for the subject: Subjects ≤ 50 kg: 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight OR Subjects \> 50 kg: 0.1 to 2.5 x 10(8) CAR-positive viable T cells

Tisagenlecleucel is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Kymriah for:

B-cell acute lymphoblastic leukemia (ALL) in patients up to 25 years old
Relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy
Relapsed or refractory follicular lymphoma after two or more lines of systemic therapy

🇪🇺 Approved in European Union as Kymriah for:

B-cell acute lymphoblastic leukemia (ALL) in patients up to 25 years old
Relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy
Relapsed or refractory follicular lymphoma after two or more lines of systemic therapy