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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 : Terminated (Terminated due to slow enrollment)
First Posted : January 13, 2020
Results First Posted : July 15, 2022
Last Update Posted : July 15, 2022
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
This was a multi-center Phase II study investigating the efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with acute lymphoblastic leukemia (ALL) who were treated with tisagenlecleucel and experience B cell recovery.

Condition or disease Intervention/treatment Phase
Acute Lymphoblastic Leukemia Biological: Tisagenlecleucel Phase 2

Detailed Description:

This trial was 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. B-cell aplasia is defined as PB absolute B lymphocyte count <50/µL.

The study had the following phases for all patients: Screening, Treatment and Follow-up. The total duration of the study was about 12 months. After tisagenlecleucel re-infusion, efficacy was assessed at months 1, 3, 6, and End of Study at which time blood samples were obtained.

The study stopped early due to slow enrollment into the trial. The rate of enrollment made the trial no longer feasible to continue.

The patients were able to voluntarily withdraw from the study for any reason, at any time. Patients who received commercial tisagenlecleucel had to be followed for up to 15 years post-infusion. Patients could have been 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, adverse events were to be reported for 15 years or until the patient enrolls in the registry.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 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
Actual Primary Completion Date : October 19, 2021
Actual Study Completion Date : October 19, 2021


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 9 Months of Reinfusion [ Time Frame: Post-reinfusion up to 9 months (Day 1 is excluded) ]
    Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count <50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel.


Secondary Outcome Measures :
  1. Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day [ Time Frame: Post-reinfusion up to 9 months ]
    Overall remission rate (ORR) was defined as the percentage of participants with a best overall disease response of complete remission (CR) or CR with incomplete blood count recovery (CRi). However, the rate was not calculated due to low enrollment. Participants' best responses have been listed by day and participants may be counted more than once.

  2. Participants With an Event [ Time Frame: Reinfusion up to 9 months ]
    An event was defined as one of the following: death from any cause after remission, relapse, treatment failure (defined as no response in the study or discontinuation from the study for death, adverse event, lack of efficacy or progressive disease or new cancer therapy).

  3. Overall Survival (OS) [ Time Frame: Reinfusion up to 9 months ]
    Deaths 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 unexpired, commercial 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

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


Locations
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United States, California
Childrens Hospital Los Angeles Divisionof Hematology/Oncology
Los Angeles, California, United States, 90027
United States, Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Missouri
Children s Mercy Hospital
Kansas City, Missouri, United States, 64108
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75235
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Novartis ( Novartis Pharmaceuticals ):
Study Protocol  [PDF] June 23, 2020
Statistical Analysis Plan  [PDF] November 29, 2021

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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
Results First Posted: July 15, 2022
Last Update Posted: July 15, 2022
Last Verified: July 2022
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