Description:
This is a multi-center Phase II study investigating the efficacy and safety of reinfusion of
tisagenlecleucel in pediatric and young adult patients with ALL who were treated with
tisagenlecleucel and experience B cell recovery.
Title
- Brief Title: Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)
- Official Title: A Phase II, Open Label, Multi-center Trial to Determine the Efficacy and Safety of Tisagenlecleucel Re-infusion in Pediatric and Adolescent Young Adult (AYA) Patients With Acute Lymphoblastic Leukemia Experiencing Loss of B Cell Aplasia
Clinical Trial IDs
- ORG STUDY ID:
CCTL019BUS03
- NCT ID:
NCT04225676
Conditions
Interventions
| Drug | Synonyms | Arms |
|---|
| Tisagenlecleucel | | Tisagenlecleucel |
Purpose
This is a multi-center Phase II study investigating the efficacy and safety of reinfusion of
tisagenlecleucel in pediatric and young adult patients with ALL who were treated with
tisagenlecleucel and experience B cell recovery.
Detailed Description
This trial is a phase II, open label, multi-center trial to determine the efficacy and safety
of tisagenlecleucel re-infusion in pediatric and adolescent young adult (AYA) patients with
acute lymphoblastic leukemia (ALL) experiencing loss of B cell aplasia. Loss of B-cell
aplasia is defined as: peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B
lymphocyte ≥ 10% of the total lymphocytes.
The study will have the following phases for all patients: Screening, Treatment and
Follow-up. The total duration of the study is about 12 months. After tisagenlecleucel
re-infusion, efficacy will be assessed at months 1, 3, 6, and 12 at which time blood samples
will be obtained to measure circulating B lymphocytes (< 50/µL) and presence of CTL019 cells
by qPCR in the peripheral blood. Safety will be assessed throughout the study.
The end of the study will be when all patients complete a month 12 visit unless discontinuing
prior. The patient may voluntarily withdraw from the study for any reason, at any time.
Patients who receive commercial tisagenlecleucel must be followed for up to 15 years
post-infusion. Patients can be followed under the Center for International Blood and Marrow
Transplant Research (CIBMTR) cellular therapy registry if consented for participation. For
patients who do not provide consent for participation in the Center for International Blood
and Marrow Transplant Research (CIBMTR) registry, AEs will need to be reported for 15 years
or until the patient enrolls in the registry.
Trial Arms
| Name | Type | Description | Interventions |
|---|
| Tisagenlecleucel | Experimental | Tisagenlecleucel Cell Dispersion for Infusion given once during the study.
The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight. | |
Eligibility Criteria
Inclusion Criteria:
- Signed informed consent must be obtained prior to participation in the study
- Must have an additional dose of tisagenlecleucel available and prescribed by a
physician in the course of medical practice
- Age up to and including 25 years
- Patients must have CD-19+ Leukemia
- Patients who were previously treated with tisagenlecleucel and present with evidence
of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count ≥
50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes
Exclusion Criteria:
- Prior gene therapy other than tisagenlecleucel
- Prior adoptive T cell therapy other than tisagenlecleucel
- Active CNS involvement by malignancy
- Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at
screening
- HIV positive test within 8 weeks of screening
Other protocol-defined Inclusion/Exclusion may apply.
| Maximum Eligible Age: | 25 Years |
| Minimum Eligible Age: | 2 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | Percentage of patients who establish B cell aplasia within 12 months of reinfusion, as measured by circulating B lymphocytes (< 50/µL) and presence of CTL019 cells by qPCR in the peripheral blood |
| Time Frame: | reinfusion up to 1 year |
| Safety Issue: | |
| Description: | Evaluate the incidence of B cell aplasia after reinfusion of tisagenlecleucel, as measured by circulating B lymphocytes (< 50/µL) and presence of CTL019 cells by Quantitative Polymerase Chain Reaction (qPCR) in the peripheral blood |
Secondary Outcome Measures
| Measure: | Percentage of patients with overall remission rate (ORR= CR + CRi) per Investigator assessment |
| Time Frame: | reinfusion up to 12 months |
| Safety Issue: | |
| Description: | Measure loss of B cell aplasia by overall remission rate ORR which includes CR and CR with incomplete blood count recovery (CRi) as determined by investigator assessment |
| Measure: | Event free survival (EFS) |
| Time Frame: | reinfusion up to 12 months |
| Safety Issue: | |
| Description: | Time from reinfusion to the earliest of relapse (defined as morphologic evidence of lumphoblasts >=5%), treatment failure, or death |
| Measure: | Overall Survival (OS) |
| Time Frame: | reinfusion up to 12 months |
| Safety Issue: | |
| Description: | Time from date of re-infusion to the date of death due to any reason |
Details
| Phase: | Phase 2 |
| Primary Purpose: | Interventional |
| Overall Status: | Recruiting |
| Lead Sponsor: | Novartis Pharmaceuticals |
Trial Keywords
- CTL019
- Kymriah
- Tisagenlecleucel
- B-Cell Acute Lymphoblastic Leukemia
- Leukemia
- ALL
- Pediatric
- Young Adult
- Reinfusion
Last Updated
August 11, 2021
