Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell Therapy

Long-term Follow-up Study to Evaluate the Safety and Efficacy in Patients Who Have Ever Received Lentiviral-based Gene-edited Immune Cell Therapy

According to health authorities guidances (FDA 2006, EMA(European Medicines Agency) 2009) for gene therapy clinical trials, observing subjects for delayed adverse events for 15 years is recommended. This purpose of this long-term follow-up study is to evaluate the safety and efficacy in patients who have ever received lentiviral-based gene-edited immune cells which are manufactured by Pell Bio-Med Technology Co. Ltd.

Study Overview

• Status: Recruiting
• Conditions: Diffuse Large B Cell Lymphoma; Primary Mediastinal Large B Cell Lymphoma; Large B-cell Lymphoma; Follicular Lymphoma Grade 3B; Follicular Lymphoma Grade 3A
• Intervention / Treatment: Genetic: Pell's lentiviral-based gene-edited immune cell therapy

Detailed Description

After completion or early withdraw from the other treatment protocol, patients should be enrolled into this long-term follow-up study. If patients do not enter this study right after leaving the treatment protocol, they may have the option to enter this long-term follow-up study at any time within 15 years after the last lentiviral-based gene-edited immune cell infusion.

• Study Type: Observational
• Enrollment (Estimated): 49

Contacts and Locations

• Study Contact: Name: Cherry Lo, MSC; Phone Number: 3111 886-2-8791-1789; Email: cherry.lo@pellbmt.com

Study Locations

• Taiwan
  • Kaohsiung, Taiwan, 807377
    • Recruiting
    • Kaohsiung Medical University Chung-Ho Memorial Hospital
    • Principal Investigator: Shyh-Shin Chiou
  • Taipei, Taiwan, 112201
    • Not yet recruiting
    • Taipei Veterans General Hospital
    • Principal Investigator: Jyh-Pyng Gau
  • Taipei, Taiwan, 10025
    • Recruiting
    • National Taiwan University Hospital
    • Principal Investigator: Shang-Ju Wu
  • Taipei city, Taiwan, 11031
    • Not yet recruiting
    • Taipei Medical University - Taipei Medical University Hospital
    • Contact: Jyh-Pyng Gau

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population includes all patients who have ever received Pell's lentiviral-based gene-edited immune cell therapy.

Description

Inclusion Criteria:

1. Patients must have ever received Pell's lentiviral-based gene-edited immune cell as monotherapy or as combination therapy in clinical trials.
2. The last lentiviral-based gene-edited immune cell infusion within 15 years.
3. Patient/patient's parent/legal guardian is capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria:

There are no specific exclusion criteria for this study.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Group A; After completion or early withdraw from the treatment protocol, patients will be enrolled into this long-term follow-up study. If patients do not enter this study right after leaving the treatment protocol, they may have the option to enter this study at any time within 15 years after the last lentiviral-based gene-edited immune cell infusion.	Genetic: Pell's lentiviral-based gene-edited immune cell therapy; No study drug or other planned treatment will be administered. Subjects who previously received Pell's lentiviral-based gene-edited immune cell therapy will be evaluated the safety and efficacy.
Group B; Some patients may require joining other Pell's gene-edited immune cell therapy study during participating in this long-term follow-up study. For such case, the patient could be enrolled into the new treatment protocol. Meanwhile, the patient can be remaining in this long-term follow-up protocol as an inactive participant.	Genetic: Pell's lentiviral-based gene-edited immune cell therapy; No study drug or other planned treatment will be administered. Subjects who previously received Pell's lentiviral-based gene-edited immune cell therapy will be evaluated the safety and efficacy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To assess delayed adverse events which are suspected related to previous gene-edited immune cell therapy	• Proportion of patients with any events of the following items which are suspected related to previous gene-edited immune cell therapy.; New malignancies; New incidence or exacerbation of a pre-existing neurologic disorder; New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder; New incidence of a hematologic disorder, including hypogammaglobulinemia; New incidence of infection (potentially product-related); Other than the above adverse events, which are suspected related to gene-edited immune cell therapy judged by the investigator	15 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Monitor for Replication Competent of Lentivirus (RCL)	Proportion of patients with detectable RCL in peripheral blood by VSV-G(Vesicular stomatitis virus G) qPCR	15 years
Monitor the persistence of gene-edited immune cells in peripheral blood(By qPCR)	Proportion of patients with detectable transgene level in peripheral blood by qPCR	15 years
Monitor the persistence of gene-edited immune cells in peripheral blood(By Flowcytometry)	Persistence of gene-edited immune cells in peripheral blood using flow cytometry	5 years
To assess the long-term efficacy of gene-edited immune cells	Proportion of patients with relapse or progress among patients who didn't progress or relapse at study entry/reentry; Incidence of death	15 years

Collaborators and Investigators

• Sponsor: Pell Bio-Med Technology Co., Ltd.
• Investigators: Study Chair: Chen-Lung Lin, MD, Pell Bio-Med Technology Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 29, 2022
• Primary Completion (Estimated): December 1, 2037
• Study Completion (Estimated): December 1, 2037

Study Registration Dates

• First Submitted: May 12, 2022
• First Submitted That Met QC Criteria: May 12, 2022
• First Posted (Actual): May 17, 2022

Study Record Updates

• Last Update Posted (Actual): April 3, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: DLBCL(Diffuse Large B Cell Lymphoma); PMLBCL(Primary Mediastinal Large B Cell Lymphoma); FL(Follicular Lymphoma)
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Lymphoma; Lymphoma, Follicular; Lymphoma, B-Cell; Lymphoma, Large B-Cell, Diffuse; Lymphoma, Non-Hodgkin
• Other Study ID Numbers: PLLV-LTFU-401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No