CD19 CART LTFU study

Principal Investigator: Michael Boyer
Keywords: CD19 , Lentiviral , CART , Cell , Therapy Department: Hematology
IRB Number: 00079262 Co Investigator:  
Specialty: Pediatric Hematology and Oncology
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Luke Stone
Luke.Stone@hsc.utah.edu
801-213-3395

Brief Summary

This study is a prospective long term follow up of patients (adults and pediatrics) previously treated with CD19 CART therapy on other treatment protocols. No investigational treatment is involved with this study. 

Study rationale and purpose

Long term safety and efficacy follow up of patients treated with gene therapy products, or advanced therapy medicinal products that use integrating vectors (example: lentiviral vectors), is required per Health Authority guidelines (FDA, EMA) [see below]

The purpose of this study is to monitor all patients exposed to lentiviral vector based CD19 CART therapy for 15 years from the last CD19 CART infusion to assess the risk of delayed AEs suspected to be related to CD19 CART therapy, monitor for vectors persistence and replication competent lentivirus (RCL), and record the status of the primary malignancy (efficacy).

Collection of such long term effects of CD19 CART cell therapy will help to further define the risk-benefit profile of CD19 CART in patients with B cell malignancies. These observations will also serve to provide further guidance to patients, health care providers and investigators of such risks and their detection and management.

Patients will be monitored for safety and efficacy within the parameters of the primary treatment protocols. While every effort will be made to keep patients in follow-up within the respective treatment protocol itself, patients can withdraw or drop off treatment protocols at any time. Patients discontinuing from the primary treatment protocols (for any reason) will be enrolled in this long term follow up (LTFU). This will allow collecting data on long term safety and efficacy as mandated by the health authorities of all patients treated with CD19 CART therapy within the concept of a single “basket” protocol. Any data will be reported in the context of the original treatment protocol.

Primary Objective

Describe selected, delayed AEs suspected to be related to previous CD19 CART cell therapy as outlined in current Health Authority guidelines

Secondary Objective

1. Monitor the persistence of CD19 CAR transgene in peripheral blood

2. Monitor expression of RCL

3. Assess the long-term efficacy of CD19 CART

4. Monitor lymphocyte levels

5. Describe the growth, development, and female reproductive status for patients who were aged < 18 years at the time of initial CD19 CART infusion

 

 
 
 

 

Inclusion Criteria

The target population includes all patients who have received CD19 CAR T-cell therapy (murine and/or humanized) manufactured by Novartis or Penn and participated in a Novartis or Penn sponsored treatment protocol or a Novartis managed access program. All patients will be included upon completion of the core study or early discontinuation. The investigator or designee must ensure that only patients who meet all the inclusion criteria are enrolled in the study.

Patients eligible for inclusion in this study have to meet all of the following criteria:

1. All patients who have received CD19 CAR T-cell therapy, murine (CTL019) and/or humanized (CTL119) CD19-directed CAR T cell treatment, manufactured by Novartis or Penn and participated in a Novartis or Penn sponsored treatment protocol or a Novartis managed access program. All patients will be included upon completion of the core study or early discontinuation

2. Patients who have provided informed consent for the long term follow-up study prior to their study participation.

Exclusion Criteria

There are no specific exclusion criteria for this study.