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Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)

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ClinicalTrials.gov Identifier: NCT04225676
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : August 11, 2021
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
ALL Biological: Tisagenlecleucel Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 49 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 19, 2020
Estimated Primary Completion Date : February 28, 2022
Estimated Study Completion Date : October 27, 2022


Arm Intervention/treatment
Experimental: Tisagenlecleucel

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.

Biological: Tisagenlecleucel

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.





Primary Outcome Measures :
  1. 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 ]
    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 :
  1. Percentage of patients with overall remission rate (ORR= CR + CRi) per Investigator assessment [ Time Frame: reinfusion up to 12 months ]
    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

  2. Event free survival (EFS) [ Time Frame: reinfusion up to 12 months ]
    Time from reinfusion to the earliest of relapse (defined as morphologic evidence of lumphoblasts >=5%), treatment failure, or death

  3. Overall Survival (OS) [ Time Frame: reinfusion up to 12 months ]
    Time from date of re-infusion to the date of death due to any reason



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04225676


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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United States, California
Childrens Hospital Los Angeles Divisionof Hematology/Oncology Recruiting
Los Angeles, California, United States, 90027
Contact    323-361-2121      
Principal Investigator: Michael Pulsipher         
UCSF Medical Center Recruiting
San Francisco, California, United States, 94143
Principal Investigator: Michelle Hermiston         
Stanford Universtiy Medical Center Recruiting
Stanford, California, United States, 94304
Contact    650-723-4000      
Principal Investigator: Kara Davis         
United States, Colorado
Childrens Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact    720-777-5379      
Principal Investigator: Amy K Keating         
United States, Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact    312-227-4811      
Principal Investigator: Sonali Chaudhury         
United States, Missouri
Children s Mercy Hospital Recruiting
Kansas City, Missouri, United States, 64108
Principal Investigator: Doug Myers         
United States, Ohio
Cincinnati Children s Hospital Medical Center Cin. Children's Hosp Med Ctr Recruiting
Cincinnati, Ohio, United States, 45229-3039
Principal Investigator: Christine Phillips         
United States, Pennsylvania
Childrens Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Principal Investigator: Stephan Grupp         
United States, Texas
UT Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75235
Principal Investigator: Samuel John         
United States, Utah
University of Utah Clinical Trials Office Recruiting
Salt Lake City, Utah, United States, 84108
Contact    801-587-5711      
Principal Investigator: Michael Boyer         
United States, Wisconsin
Childrens Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Principal Investigator: Julie-An Talano         
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04225676    
Other Study ID Numbers: CCTL019BUS03
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: August 11, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
CTL019
Kymriah
Tisagenlecleucel
B-Cell Acute Lymphoblastic Leukemia
Leukemia
ALL
Pediatric
Young Adult
Reinfusion
Additional relevant MeSH terms:
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Leukemia
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases