A clinical study to provide the opportunity to be treated with CTL019, an investigational gene therapy, for children and adolescent patients with a recurrent form of B-cell acute lymphoblastic leukaemia after the closure of the Novartis single-arm phase II pivotal registration trial (Study CCTL019B2202) and to collect additional safety information.

Phase IIIb study for relapsed/refractory pediatric/young adult acute lymphoblastic leukemia patients to be treated with CTL019 - Phase IIIb study for CTL019.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-001991-31-AT

Enrollment

Registered

2016-12-21

Start date

2017-03-02

Completion date

Unknown

Last updated

2020-11-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paediatric/young adult patients with B-cell acute lymphoblastic leukaemia who are chemo-refractory, relapsed after allogeneic SCT, or are otherwise ineligible for allogeneic SCT. MedDRA version: 21.0 Level: LLT Classification code 10063625 Term: Acute lymphoblastic leukemia recurrent System Organ Class: 100000004864 MedDRA version: 21.0 Level: LLT Classification code 10063621 Term: Acute lymphoblastic leukaemia recurrent System Organ Class: 100000004864

Interventions

Trade Name: Kymriah Product Name: Tisagenlecleucel Product Code: CTL019 Pharmaceutical Form: Suspension for injection INN or Proposed INN: Tisagenlecleucel Current Sponsor code: CTL019 Other descripti

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients eligible for inclusion in this program have to meet all of the following criteria: 1. Relapsed or refractory B-cell ALL in pediatric or young adult patients 2. For relapsed patients, CD19 tumor expression demonstrated in bone marrow or peripheral blood by flow cytometry within 3 months of study entry with adequate organ function. 3. Adequate organ function as defined in the protocol. 4. Life expectancy > 12 weeks. 5. Age =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: EExclusion Criteria: 1. Isolated extra-medullary disease relapse. 2. Patients with concomitant genetic syndromes associated with bone marrow failure states: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Patients with Down Syndrome will not be excluded. 3. Patients with Burkitt's lymphoma/leukemia. 4. Prior malignancy, except carcinoma in situ of the skin or cervix treated with curative intent and with no evidence of active disease. 5. Prior treatment with any gene therapy product. 6. Prior treatment with any anti CD19/anti-CD3 therapy, or any other anti-CD19 therapy, except for patients pre-treated with blinatumomab who fulfill inclusion criterion no. 8. 7. Presence of active replication of hepatitis B or hepatitis C (for detailed criteria see Appendix 2 of main protocol). Serology must be repeated, if the interval between testing at Screening and CTL019 infusion exceeds 8 weeks. 8. HIV positivity as indicated by serology. Serology must be repeated, if the interval between testing at Screening and CTL019 infusion exceeds 8 weeks. 9. Presence of grade 2 to 4 acute or extensive chronic graft versus host disease (GVHD). 10. Uncontrolled acute life threatening bacterial, viral or fungal infection at Screening. 11. Previous or concurrent malignancy (exceptions defined in the protocol) 12. Intolerance to the excipients of the CTL019 cell product (i.e. dimethyl sulfoxide). 13. Cardiac or cardiac repolarization abnormality. 14. Patients enrolled in this study are not permitted to participate in additional parallel investigational drug or device studies. 15. Patient has an investigational medicinal product within the last 30 days prior to screening. 16. The following medications are excluded: a. Steroids, b. Allogeneic cellular therapy, c. GVHD therapies, d. Chemotherapy, e. CNS disease prophylaxis, f. Radiotherapy, g. Anti-T cell antibodies. 17. Pregnant or nursing (lactating) women. 18. Women of child-bearing potential 19. Sexually active males must use a condom during intercourse from enrollment and for at least 12 months after the CTL019 infusion and until CAR T cells are no longer present by qPCR on 2 consecutive tests. Other protocol-defined exclusion criteria may apply.

Design outcomes

Primary

Measure	Time frame
Main Objective: This expanded treatment protocol (ETP) will provide pediatric/young adult patients with relapsed or refractory B-cell ALL the opportunity to be treated with CTL019 after the closure of the Novartis single-arm phase II clinical trial (Study CCTL019B2202) and to collect additional safety information.;Secondary Objective: - Evaluate the efficacy of CTL019 therapy as measured by complete remission rate, which includes CR and CR with incomplete blood count recovery (CRi). - Evaluate the percentage of patients who achieve CR or CRi at Month 6 without SCT between CTL019 infusion and Month 6 response assessment. - Evaluate the percentage of patients who achieve CR or CRi and then proceed to SCT while in remission before Month 6 response assessment. - Evaluate the duration of remission. - Evaluate the relapse-free survival. - Evaluate the event-free survival. - Evaluate the overall survival. - Evaluate the response at Day 28 +/- 4 days. - Evaluate the impact of baseline tumor burden on response. - Evaluate the quality of response using minimal residue disease assessments. - Describe the prevalence and incidence of immunogenicity to CTL019. - Characterize the in vivo cellular kinetic profile of CTL019 cells in the blood. - Evaluate the relationship between exposure to CTL019 with CRS grades. ;Primary end point(s): Evaluate the safety of CTL019 therapy.;Timepoint(s) of evaluation of this end point: The endpoint is assessed throughout the 12 months study duration.	—

Measure

Time frame

Main Objective: This expanded treatment protocol (ETP) will provide pediatric/young adult patients with relapsed or refractory B-cell ALL the opportunity to be treated with CTL019 after the closure of the Novartis single-arm phase II clinical trial (Study CCTL019B2202) and to collect additional safety information.;Secondary Objective: - Evaluate the efficacy of CTL019 therapy as measured by complete remission rate, which includes CR and CR with incomplete blood count recovery (CRi). - Evaluate the percentage of patients who achieve CR or CRi at Month 6 without SCT between CTL019 infusion and Month 6 response assessment. - Evaluate the percentage of patients who achieve CR or CRi and then proceed to SCT while in remission before Month 6 response assessment. - Evaluate the duration of remission. - Evaluate the relapse-free survival. - Evaluate the event-free survival. - Evaluate the overall survival. - Evaluate the response at Day 28 +/- 4 days. - Evaluate the impact of baseline tumor burden on response. - Evaluate the quality of response using minimal residue disease assessments. - Describe the prevalence and incidence of immunogenicity to CTL019. - Characterize the in vivo cellular kinetic profile of CTL019 cells in the blood. - Evaluate the relationship between exposure to CTL019 with CRS grades. ;Primary end point(s): Evaluate the safety of CTL019 therapy.;Timepoint(s) of evaluation of this end point: The endpoint is assessed throughout the 12 months study duration.

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Secondary

Measure	Time frame
Secondary end point(s): - Percentage of patients who achieve complete remission or complete remission with incomplete blood count recovery (i.e. CR or CRi) during the 6 months after CTL019 infusion. - Percentage of patients who achieve CR or CRi at Month 6 without SCT between CTL019 infusion and Month 6 response assessment. - Percentage of patients who achieve CR or CRi and then proceed to SCT while in remission prior to Month 6 response assessment. - Duration of remission. - Relapse-free survival. - Event-free survival. - Overall survival. - Response at Day 28 +/- 4 days. - Impact of baseline tumor burden on response. - Quality of response using MRD assessments before treatment and at Day 28 ± 4 days after treatment and before SCT by local assessment (flow cytometry +/- quantitative polymerase chain reaction (q-PCR)). - Describe the prevalence and incidence of immunogenicity to CTL019. - Characterize the in vivo cellular kinetic profile (levels, persistence, trafficking) of CTL019 cells in the blood. - Evaluate the relationship between exposure to CTL019 with CRS grades.;Timepoint(s) of evaluation of this end point: The endpoints are assessed throughout the 12 months study duration.	—

Measure

Time frame

Secondary end point(s): - Percentage of patients who achieve complete remission or complete remission with incomplete blood count recovery (i.e. CR or CRi) during the 6 months after CTL019 infusion. - Percentage of patients who achieve CR or CRi at Month 6 without SCT between CTL019 infusion and Month 6 response assessment. - Percentage of patients who achieve CR or CRi and then proceed to SCT while in remission prior to Month 6 response assessment. - Duration of remission. - Relapse-free survival. - Event-free survival. - Overall survival. - Response at Day 28 +/- 4 days. - Impact of baseline tumor burden on response. - Quality of response using MRD assessments before treatment and at Day 28 ± 4 days after treatment and before SCT by local assessment (flow cytometry +/- quantitative polymerase chain reaction (q-PCR)). - Describe the prevalence and incidence of immunogenicity to CTL019. - Characterize the in vivo cellular kinetic profile (levels, persistence, trafficking) of CTL019 cells in the blood. - Evaluate the relationship between exposure to CTL019 with CRS grades.;Timepoint(s) of evaluation of this end point: The endpoints are assessed throughout the 12 months study duration.

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Countries

Austria, Belgium, Canada, France, Germany, Italy, Japan, Norway, Spain

Contacts

Public ContactDrug Regulatory Affairs

Novartis Pharma GmbH

austria.dra@novartis.com+431 86657 0

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026