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CAR T-cell Therapy

Tisagenlecleucel Safety for Cancer

Phase 3

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients for whom the final manufactured tisagenlecleucel product does not meet the commercial release specifications.

Not excluded from commercial manufacturing under the Health Authority-approved tisagenlecleucel prescribing information for their respective country/region.

Must not have

Patients with active replication of Hepatitis B virus (HBV) or Hepatitis C virus (HCV).

Patients with primary central nervous system (CNS) lymphoma.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial will study the safety of a drug that doesn't meet specification for release. It will specifically study the safety of the drug in Japanese patients in two parts. Part one will also study the efficacy of the drug.

Who is the study for?

This trial is for patients with certain types of B-cell lymphoma or acute lymphoblastic leukemia whose tisagenlecleucel treatment doesn't meet commercial standards but isn't unsafe. They must understand the study, not have hepatitis B/C, CNS lymphoma, HIV, uncontrolled infections, be pregnant/nursing, or have conditions affecting safety assessment.

What is being tested?

The trial tests the safety and some efficacy of CTL019 (tisagenlecleucel) that's out of specification for commercial release in Japan. Part 1 looks at both safety and key effects; Part 2 focuses on safety within approved use.

What are the potential side effects?

While specific side effects aren't listed here, similar treatments can cause immune reactions, infusion-related symptoms, fatigue, blood abnormalities and increase infection risk. Patients with known hypersensitivity to related drugs are excluded.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My tisagenlecleucel treatment was not made to commercial standards.

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I am eligible for tisagenlecleucel treatment according to my country's health guidelines.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have active Hepatitis B or C.

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I have been diagnosed with lymphoma in my brain or spinal cord.

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I am HIV positive.

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I do not have any ongoing infections or inflammations that aren't under control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of participants with Adverse Events (AEs)

Secondary study objectives

Part 1: Overall Remission Rate in Group A (pALL)

Part 1: Overall Response Rate in Group B (LBCL)

Side effects data

From 2020 Phase 3 trial • 69 Patients • NCT03123939

43%

Cytokine release syndrome

35%

Pyrexia

30%

Hypogammaglobulinaemia

25%

Diarrhoea

23%

Headache

23%

Nausea

20%

White blood cell count decreased

20%

Hypokalaemia

20%

Cough

20%

Anaemia

19%

Vomiting

16%

Neutrophil count decreased

14%

Hypophosphataemia

14%

Neutropenia

14%

Platelet count decreased

14%

Rash

13%

Nasopharyngitis

12%

Hypoalbuminaemia

12%

Hypocalcaemia

12%

Tachycardia

12%

Arthralgia

12%

Abdominal pain

12%

Aspartate aminotransferase increased

12%

Epistaxis

12%

Decreased appetite

12%

Hypertension

12%

Pruritus

10%

Hypoxia

10%

Alanine aminotransferase increased

10%

Upper respiratory tract infection

10%

Fatigue

10%

Constipation

10%

Hypomagnesaemia

10%

Pain in extremity

Face oedema

Back pain

Oropharyngeal pain

Erythema

Hypotension

Myalgia

Petechiae

Pain

Oedema peripheral

Thrombocytopenia

Rhinitis

Immunoglobulins decreased

Lymphocyte count decreased

Insomnia

Dry skin

Nasal congestion

Febrile neutropenia

Sinus tachycardia

Chills

Allergy to immunoglobulin therapy

Abdominal pain upper

Blood fibrinogen decreased

Hyperglycaemia

Seizure

Anxiety

Haematuria

Hyperuricaemia

Herpes zoster

Sepsis

Acute lymphocytic leukaemia recurrent

Bacterial infection

Bone marrow failure

Pneumonia

Device related infection

Encephalopathy

B precursor type acute leukaemia

Tumour lysis syndrome

Central nervous system infection

Candida infection

Hepatosplenomegaly

Joint effusion

Cellulitis

Pneumonia haemophilus

Septic shock

Leukaemia

Depressed level of consciousness

Drug withdrawal syndrome

Neoplasm progression

Jugular vein thrombosis

Hyponatraemia

Periorbital cellulitis

Facial paralysis

Splinter

Aspergillus infection

Meningitis aseptic

Cellulitis orbital

Left ventricular dysfunction

Leukocytosis

Respiratory syncytial virus infection

Sinusitis

Hypernatraemia

Dyskinesia

Multiple organ dysfunction syndrome

Hepatocellular injury

Haemophagocytic lymphohistiocytosis

Dysarthria

Tremor

Completed suicide

Irritability

Alternaria infection

Cerebral fungal infection

Enterococcal infection

Infection

Influenza

Tonsillitis

Viral upper respiratory tract infection

Chest X-ray abnormal

Dehydration

Lactic acidosis

Somnolence

Agitation

Confusional state

Disorientation

Hallucination

100%

80%

60%

40%

20%

Study treatment Arm

All Subjects - CTL019

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Group C: r/r NHLExperimental Treatment1 Intervention

Adult patients with r/r NHL in consistent with the Health Authority approved indication in the package insert for CTL019 in Japan whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.

Group II: Group B: r/r LBCLExperimental Treatment1 Intervention

Adult patients with r/r LBCL including DLBCL not otherwise specified, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma, that is consistent with the Health Authority-approved indication in the package insert for CTL019 in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.

Group III: Group A: pALLExperimental Treatment1 Intervention

Pediatric/young adult patients with r/r pALL who meet the indication in the Health Authority-approved CTL019 package insert in the respective country/region whose final manufactured product is OOS for commercial release, but it is considered that the benefit-risk profile may remain favorable and the usual expected benefits of infusing such a product outweigh the potential risks for the patient.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CTL019

2017

Completed Phase 3

~150

0 / 6Eligibility criteria met